3rd Report, 2008 (Session 3)

Availability on the NHS of cancer treatment drugs

CONTENTS

Remit and membership

Report—
Introduction

Format of report
Background
Approach to inquiry

Consideration of Evidence

Defining Roles

Licensing
Key organisations
rocesses in Scotland and England & Wales for producing advice
Consideration of evidence
Conclusion to this section

Guidance

Conclusion to this section

Data gathering

Conclusion to this section
Quality-Adjusted Life Year (QALY)
Conclusion to this section

Availability (covering exceptional prescribing)

Conclusion to this section

Funding

Conclusion to this section

Other issues

Pharmaceutical price setting
Conclusion to this section

Overall Conclusions

ANNEXE A: MINUTES OF PROCEEDINGS

Scottish Parliament Public Petitions Committee. Minutes of Proceedings, 15 January 2008
Scottish Parliament Public Petitions Committee. Minutes of Proceedings, 19 February 2008
Scottish Parliament Public Petitions Committee. Minutes of Proceedings, 4 March 2008
Scottish Parliament Public Petitions Committee. Minutes of Proceedings, 29 April 2008
Scottish Parliament Public Petitions Committee. Minutes of Proceedings, 23 May 2008 (item 1)
Scottish Parliament Public Petitions Committee. Minutes of Proceedings, 20 May 2008

ANNEXE B: ORAL EVIDENCE

Scottish Parliament Public Petitions Committee. Official Report, 15 January 2008 (consideration of PE1108
Scottish Parliament Public Petitions Committee. Official Report, 19 February 2008 (consideration of PE1108)
Scottish Parliament Public Petitions Committee. Official Report, 29 April 2008 (inquiry oral evidence hearing)
Scottish Parliament Public Petitions Committee. Official Report, 13 May 2008 (inquiry oral evidence hearing)
Scottish Parliament Public Petitions Committee. Official Report, 20 May 2008 (inquiry oral evidence hearing)

ANNEXE C: WRITTEN EVIDENCE

NHS Quality Improvement Scotland
Scottish Medicines Consortium
National Institute for Health and Clinical Excellence
Cabinet Secretary for Health and Wellbeing, the Scottish Government
NHS Grampian
NHS Grampian Attachment
South East Scotland Cancer Network (on behalf of NHS Lothian, NHS Fife, NHS Borders and NHS Dumfries & Galloway)
West of Scotland Cancer Network on behalf of NHS Greater Glasgow and Clyde, NHS Ayrshire and Arran, NHS Forth Valley and NHS Lanarkshire)
North of Scotland Cancer Network (on behalf of NHS Grampian, NHS Highland, NHS Orkney, NHS Shetland, NHS Tayside and NHS Western Isles)
Scotland Patients Association
Cancer Research UK
Association of the British Pharmaceutical Industry (Scotland) and the Scottish Cancer Industry Group
Royal College of Radiologists
Royal College of General Practitioners Scotland Patient Group
Scottish Association of Prostate Cancer Support Groups
Professor Karol Sikora, Medical Director, Cancer Partners UK and Professor of Cancer Medicine, Imperial College, London
Trish Thomas
Lydia Reid
Bristol-Myers Squibb Pharmaceuticals Ltd
Northern Ireland Minister for Health, Social Services and Public Safety
Scotland Patients Association response to further written questions
South East Scotland Cancer Network response to further written questions
Cancer Research UK response to further written questions
NHS Quality Improvement Scotland response to further written questions
North of Scotland Cancer Network response to further written questions
West of Scotland Cancer Network response to further written questions
Scottish Medicines Consortium response to further written questions

Remit and membership

Remit:

To consider public petitions addressed to the Parliament in accordance with these Rules and, in particular, to—

(a) decide in a case of dispute whether a petition is admissible;

(b) decide what action should be taken upon an admissible public petition; and

(c) keep under review the operation of the petitions system.

(Standing Orders of the Scottish Parliament, Rule 6.10)

Membership:
Bashir Ahmed
Claire Baker
Angela Constance
Nigel Don (from 31 October 2007)
Rhoda Grant
Robin Harper
Tricia Marwick (until 31 October 2007)
Mr Frank McAveety (Convener)
Nanette Milne
John Farquhar Munro (Deputy Convener)

Committee Clerking Team:

Clerk to the Committee
Fergus Cochrane

Assistant Clerks
Franck David
Zoé Tough

Committee Assistant
Eileen Martin

Availability on the NHS of cancer treatment drugs

The Committee reports to the Parliament as follows—

Introduction

1. The Public Petitions Committee, at its meeting on 4 March 2008, agreed to undertake an inquiry into the availability on the NHS of cancer treatment drugs. The starting point for this inquiry is petition PE1108 by Tina McGeever, on behalf of her husband Michael Gray. It is important to first set out the background to this inquiry which has led to this report.

Format of report

2. Annexes A, B and C to this report are available in e-format only. These can be accessed via the Committee’s homepage on the Parliament’s website—
www.scottish.parliament.uk/s3/committees/petitions/reports.htm

Background

3. Petition PE1108 was lodged on 7 January 2008. It calls on the Scottish Parliament to urge the Scottish Government to consider the provision, on the NHS, of cancer treatment drugs, in particular cetuximab, to ensure equity across NHS boards on the appropriateness, effectiveness and availability of such treatments.

4. The petition was first considered by us at our meeting on 15 January 2008 when we heard oral evidence from the petitioners. At the conclusion of the meeting we agreed to seek responses to the issues raised in the petition from the Scottish Government, Scottish Medicines Consortium, Bowel Cancer UK, NHS Grampian, the Royal College of Physicians of Edinburgh and the Royal College of Radiologists.

5.We also agreed to contact the Health and Sport Committee inviting it to consider undertaking an inquiry into the issues raised in the petition. This we did on 16 January and the convener of that committee responded on 24 January 2008 stating that it was content for the Public Petitions Committee to undertake further work on the petition rather than for it to be referred to the Health and Sport Committee. The convener also indicated that the Health and Sport Committee agreed to take the issues raised by the petition into account in the context of its inquiry into health inequalities. That inquiry is now underway and we hope that this report can usefully feed into that investigation where appropriate.

6. We gave further consideration to the petition on 19 February 2008 when we agreed to take forward the issues raised through an inquiry, which would gather evidence from the Scottish Government and all NHS boards. At our meeting on 4 March 2008 we agreed our approach to this inquiry.

Approach to inquiry

7. At our meeting on 4 March 2008 we agreed the following remit for the inquiry—

‘An inquiry into the provision, on the NHS, of cancer treatment drugs and whether there is equity across NHS boards of the appropriateness, effectiveness and availability of such drugs and whether there is parity between the cancer drug treatment regime and other life threatening or other terminal conditions.’

8. We also agreed, in order to set parameters for the inquiry and focus attention on precise issues, to invite written evidence in response to the following broad questions and issues—

• Whether the roles fulfilled by local area drug and therapeutic committees, the Scottish Medicines Consortium, NHS Quality Improvement Scotland and the National Institute for Health and Clinical Excellence are clearly defined when undertaking clinical, scientific and cost-effectiveness assessment on the use of cancer treatment drugs and the guidance that is subsequently issued to NHS Boards and clinicians?
• In terms of the number and roles that these bodies play—
  • do any anomalies or contradictions exist and is there any duplication of roles?
  • are resources being used constructively and efficiently and are there inefficiencies as a result of all these bodies?
  • how is effective communication maintained across and amongst these bodies?
• Do any anomalies exist in terms of the roles played by these bodies when considering drug treatments for other life threatening and terminal conditions and how can such anomalies be resolved?
• What action and steps need to be taken to streamline the roles played by these bodies and the processes involved and why are these not being taken?
• What requirement is there for an NHS Board to adhere to advice and guidance issued by the Scottish Medicines Consortium and NHS Quality Improvement Scotland (which recommends NICE guidance to the NHS in Scotland and which supersedes SMC guidance) on the approval and use of cancer treatment drugs?
• Is implementation of guidance across NHS Boards variable and if so, what checks and balances are in place to address this?
• What assessment is done of how individual clinicians apply such guidance?
• Do any anomalies exist in terms of the implementation of guidance on cancer treatment drugs and guidance on drug treatments for other life threatening or terminal illnesses and how can such anomalies be resolved?
• What views and suggestions have been offered by (a) clinicians and (b) patients on the procedures involved in making cancer treatment drugs available?
• Whether there are any cancer treatment drugs available in some NHS Boards but not in others?
• What criteria are applied by NHS Boards when considering whether to fund a cancer treatment drug not recommended by the SMC?
• What are the conditions and criteria set by NHS Boards to use ‘exceptional prescribing circumstances’ and is any guidance provided to patients?
• Whether it is ethical and appropriate that patients should self-fund and prove the merit of being prescribed a particular drug and to what extent does ECHR apply in the decision making process when a NHS Board is considering a funding request?
• What is the reasoning behind the position whereby a person cannot be treated as a private patient and a NHS patient for one condition during a single visit to an NHS organisation?
• Does the availability of cancer treatment drugs across NHS Boards fit in with the Scottish Government’s cancer strategy and are there any contradictions between availability and the overarching policy?
• Are there any particularly unique issues or considerations that apply in terms of the availability on the NHS of cancer treatment drugs that do not apply in relation to other drugs for terminal illnesses?
• The criteria and basis of the formula used to measure both the quality and the quantity of life lived, as a means of quantifying the benefit of a medical intervention (Quality-Adjusted Life Year (QALY));
• What consideration is being given to the availability of cancer treatment drugs as part of the Scottish Government’s national discussion on the future of cancer care?

9. Further to a general call for written evidence, we wrote specifically to the following bodies drawing attention to these questions—

• Scottish Government
• Scottish Medicines Consortium
• NHS Quality Improvement Scotland
• National Institute for Health and Clinical Excellence
• Scottish Health Council
• CAB Scotland
• Scotland Patients Association
• Cancer Research UK
• Cancerbackup
• Association of the British Pharmaceutical Industry Scotland
• Scottish Cancer Industry Group
• Royal College of General Practitioners Scotland
• Royal College of Radiologists
• British Medical Association
• All 14 NHS Boards
• UK Department of Health
• Welsh Assembly Government
• Northern Ireland Executive
• Petitioner

10. We are extremely grateful to each and every person and organisation who responded to us with written evidence. We found some of this exceptionally helpful in broadening our understanding of the issues and identifying areas for improvement.

11. We held three oral evidence sessions on the following dates—

29 April 2008: Panel 1: Scotland Patients Association, Cancer Research UK; Panel 2: Scottish Medicines Consortium, NHS Quality Improvement Scotland, National Institute for Health and Clinical Excellence; Panel 3: Greater Glasgow and Clyde, Grampian, and Lothian NHS Boards

13 May 2008: Cabinet Secretary for Health and Wellbeing, Scottish Government

20 May 2008: Tina McGeever

12. Sadly, before the commencement of our oral evidence hearings, Michael Gray died on 16 April 2008. We know that both he and his wife, Tina McGeever, wanted to give further oral evidence before us at the commencement of our inquiry but, unfortunately, his worsening condition prevented this. We again pay tribute to the dignity, strength and determination shown by them both in pursuing their petition and recognise their commitment and dedication under what we can only imagine must have been at times very difficult circumstances. Their petition highlights important issues that might not have been brought to the surface otherwise. We hope that those who will now consider this report and our conclusions will reflect on this and the circumstances, indeed the spirit, in which this inquiry came about.

13. The written questions above can be grouped into four broad themes of defining roles; guidance; availability (covering exceptional prescribing); and funding.

14. During the course of our evidence gathering, a fifth theme emerged, that of data collection. Accordingly, the report is broken down into each of these.

consideration of evidence

Defining Roles

Licensing

15. In the UK, all medicines and medical devices for human and animal use are subject to a system of licensing laid down in EC legislation, the Medicines Act 1968 and other subsequent regulations. The control of medicines starts as soon as they are first discovered and tested, all the way through to when a company wants to change the conditions its products are approved for e.g. changing the colour of the tablet, or what it is used for. Before a medicine gets to the stage of licensing it will, typically, have undergone 12 years of research and development. In this long process, the substances identified in basic research need to pass the pre-clinical and clinical tests. Pharmaceutical companies quite often research and test 10,000-30,000 different substances before one can be successfully licensed for use.

16. Following the development process the pharmaceutical company will seek a licence for the medicine. The purpose of the licensing process is to consider whether the medicine has a measurable effect against a comparator in a clinical trial (referred to as ‘efficacy’) and whether, on balance, the drug is likely to have an acceptable level of safety and quality. There are two ways of obtaining a licence—

• UK: through the Medicines and Healthcare Products Regulatory Agency (MHRA)

Established in April 2003 from a merger of the Medicines Control Agency and the Medical Devices Agency. It is an executive agency of the Department of Health and is responsible for ensuring that medicines and medical devices work and are acceptably safe. It regulates a wide range of materials from medicines and medical devices to blood and therapeutic products/services that are derived from tissue engineering. Its Commission on Human Medicines assesses applications and recommends whether the drug should get a licence or not. If it does, then the product will receive its marketing authorisation¹ and can be ‘launched’ i.e. the introduction of a new product or indication to the market.

• Europe: through the European Medicines Evaluation Agency which relates to all EU Member States (EMEA)

Established by the European Union in 1995. Its main responsibility is the protection and promotion of public health, through the evaluation and supervision of medicines for human use.² It coordinates the evaluation and supervision of medicinal products throughout the EU and is supported by the scientific resources of over 40 national competent authorities in 30 EU and European Economic Area/European Free Trade Area countries in a network of over 4,000 European experts. It began its activities when the European system for authorising medicinal products was introduced, providing for a centralised³ and a decentralised⁴ procedure. It has a role in both, but is primarily involved in the centralised procedure. There are some types of medicines that must be submitted for licensing, including those for cancer.

17. Once a medicine has been licensed many clinicians will prefer to await official advice/guidance before prescribing it. NHS advice/guidance is not the same as licensing and it has no effect on the licence status of the medicine. The licensing process considers quality, efficacy and safety, whilst NHS advice/guidance considers the clinical effectiveness i.e. how the drug will be used in practice, how it compares to the existing treatment and cost effectiveness of the medicine, and whether it is good value for money.

18. It is the process for producing this advice/guidance that can cause the most confusion and debate. While guidance over the use of licensed drugs in the NHS in Scotland is a devolved matter, there is a level of joint working that takes place between the organisations responsible for issuing this advice/guidance across the UK.

Key organisations

19. In Scotland, the principal organisations for producing advice/guidance are the Scottish Medicines Consortium (SMC) and NHS Quality Improvement Scotland (NHS QIS) whilst in England and Wales it is the National Institute for Health and Clinical Excellence (NICE).

20. The SMC, under the umbrella of NHS QIS, advises on the clinical and cost effectiveness of all newly licensed medicines. It is made up of representatives of all local Area Drug and Therapeutic Committees (ADTCs), other health professionals, the pharmaceutical industry and patient representatives (through its Patient and Public Involvement Group).

21. NHS QIS is a special health board. It provides a national service, but rather than managing health services directly, works with, and supports, other organisations that do. It is the lead organisation in NHS Scotland for improving the quality of healthcare delivered. Its roles include providing clear advice and guidance on effective clinical practice. It is also an umbrella for other organisations that work to improve the quality of healthcare.

22. NICE was first set up in 1999 and its role reviewed and expanded in 2005. It is responsible for providing national guidance on the promotion of good health and the prevention and treatment of ill health in England and Wales. As part of its role it produces technology appraisals, which offer guidance on the use of selected new and existing medicines and treatments within the NHS in England and Wales. The NHS is then obliged to adhere to this.

Processes in Scotland and England & Wales for producing advice

23. SMC, NHS QIS and NICE have their own processes for developing guidance although there is a level of co-operation between them.

24. The SMC advises NHS boards and their ADTCs on the use of all new medicines, new indications and new formulations as soon as possible after they are licensed by the MHRA or EMEA. It does not issue advice on medical devices, vaccines, branded generic drugs, non-prescription-only medicines, blood products, plasma substitutes and diagnostic drugs.

25. It was established in October 2001 and considers medicines licensed since January 2002. Prior to this decisions on which new medicines to approve were taken by the local ADTCs, which resulted in the possibility of wide variation in the provision of new medicines across Scotland and involved much duplication of effort. Thus, the SMC was created to help reduce ‘postcode prescribing’.

26. It first requires pharmaceutical companies to complete a New Product Submission form. So that advice can be published soon after the product becomes available for use the submission needs to be made before the product is licensed. It then undertakes a clinical, scientific and cost-effectiveness rapid assessment of the company’s submission and issues advice on its use.

27. NHS QIS considers that SMC advice on unique medicines accepted for use for specific conditions is obligatory and must be introduced in NHS Scotland to an agreed national programme. Medicines that are accepted for use for conditions where alternative drug treatments already exist are subject to local NHS board decisions.

28. In time, SMC advice may be superseded by the advice of a NICE multiple technology appraisal (MTA) if it is endorsed by NHS QIS. The Scottish Government expects NHS boards to implement advice from the SMC and guidance from NHS QIS.

29. NICE only considers medicines referred to it by UK Ministers. It produces initial guidance for selected medicines through its single technology appraisal (STA) process. This started in mid-2006. Prior to this, there was no such procedure, and this meant newly licensed medicines had to wait for the full evaluation process to be undertaken. Given the SMC remit in Scotland for dealing with newly licensed medicines this often led to advice being issued in Scotland a significant time before that in England.

30. Therefore NICE introduced its STA process, which is a more rapid process for assessing a small number of selected medicines and other treatments and sits alongside its standard process. The medicines are chosen and referred to NICE by the UK Department of Health. The process for evaluating drugs is similar to that of the SMC, covers all health technologies and enables single new drugs and existing drugs with new indications to be assessed more rapidly than its fuller evaluation process. This means that NICE can produce faster guidance for England and Wales on these medicines.

31. NICE STAs have no formal status in Scotland, though NHS QIS publishes them on its website for information. This was confirmed through Scottish Executive Health Department letter (NHS (HDL) 2007 26).⁵

32. In some circumstances SMC advice may be superseded if NHS QIS endorses a NICE MTA relevant to the product or condition to be treated as being valid for Scotland. The MTA process is a fuller evaluation process which NICE undertakes for new medicines, in addition to its STA process. The process itself can take up to 54 weeks to be completed though in some cases it has taken much longer depending on whether the decision of NICE is appealed.

33. Having set out the bodies that exist at a national level, it is useful to identify key bodies at the local level. Regional cancer advisory groups provide guidance on the structure and functions of regional cancer needs. They work with managed clinical networks which are linked groups of health professionals and organisations working in a co-ordinated manner, unconstrained by existing professional and NHS board boundaries, to ensure equitable provision of high quality clinically effective services.

34. Area Drug Therapeutic Committees, broadly, have responsibility for advising their local NHS board on all aspects of the use of medicines including rational, safe and cost-effective prescribing. They may have representation from general practice, hospital medicine, nursing and pharmacy, and take expert advice from clinical specialists.

Consideration of evidence

35. We turn now to the evidence we received on whether the roles of these bodies identified above are clearly defined. We were struck by their number and the tiers that exist in terms of appraising the clinical and cost effectiveness, which lead to determining the availability, of a cancer treatment drug. We wanted to consider whether there were any failings in this process that would have a detrimental impact on cancer patients in accessing drug treatments. For example, do all these bodies fulfil necessary and clearly defined roles, do clear lines of communication exist, is there any duplication of effort, does this process create an additional burden on cancer patients, are resources being used productively, that might otherwise be spent on other aspects of cancer care?

36. The written responses to our initial questions, on the whole, indicated that the system that exists is generally recognised to work well. Roles and responsibilities are clearly defined, overlap between these bodies is minimal and the system is specified and understood. We understand that these organisations must sometimes be faced with complex legal, medical, financial and ethical issues during the appraisal process and welcome the overriding opinion about how they operate.

37. Professor Peter Johnson, the Chief Clinician at Cancer Research UK, in his oral evidence stated that Scotland was a model of good practice in terms of the system that exists for appraising drugs and that the processes that have been put in place “have served patients interests well and dispassionately” and that “the appraisal process is probably as good as it can be”.⁶ We are encouraged that this view came from such a respected organisation.

38. However, we must also recognise that an appraisal system that is working well can always be improved, made more efficient and bring about greater benefits to cancer patients. This is certainly not about change for the sake of change or about meddling with something which is working well. For example, Dr Jean Turner of Scotland Patients Association in oral evidence⁷ felt that greater transparency was required in this appraisal process, probably at a more local level. Also, the SMC itself indicated that there was perhaps scope to clarify the role of ADTCs in relation to cancer drugs.⁸ These are particularly important points to draw out for further investigation.

39. We welcome the oral evidence from NHS QIS⁹ when it said that it continued to identify ways in which processes can be streamlined. That is to be encouraged and actively supported. What is not evident is how such streamlining to provide efficiencies and improvements can be delivered and who or what will make this happen. While we recognise that each individual organisation will no doubt introduce internal operating efficiency measures, and ultimately these should improve the overall process, we wonder whether some form of external monitoring requires to be put in place.

40. We have drawn attention to the tiers that exist in the appraisal process, the roles the bodies play and their interrelationship. We are not recommending that any further tier is added or that anything is done that redirects resources away from areas where they are needed most, that is delivering front line care to cancer patients. Indeed, we would hope to see where efficiency savings can be made that might free up resources for more front line care.

41. However, this may be an area for the Scottish Government to reflect on and consider whether it needs to take any action and, if so, what. For example, in what way could the process of appraisal at national and local level be made more transparent, is more patient involvement in the process needed, in particular within area drug therapeutic committees? We simply draw attention to this matter and the risk, albeit small, of a process that appears to be working well, becoming cumbersome and inefficient simply because no system of monitoring was in place.

42. We are aware¹⁰ that reviews of both the SMC and NHS QIS have been undertaken over the last few years and that an impact and effectiveness evaluation of the SMC is currently ongoing and is due to report later this year. This is examining the impact of SMC decisions on medicines utilization at local, regional and national levels across the health care system; its budget forecast with resource use within NHS Scotland over time, its engagement with key stakeholders and the linkage of clinical information and medicines utilisation data. The outcome of this evaluation we hope will lead to continued improvements for cancer patients.

Conclusion to this section

43. We invite the Scottish Government, in its written response to this report, to indicate—

• whether any form of monitoring is required of the roles of the national and local bodies in the drug appraisal process and what streamlining and efficiencies can be identified and introduced;
• how performance management arrangements, as referred to in its written evidence to us,¹¹ would be used to address any shortcomings and when it would consider it appropriate to use such arrangements; and
• how it will provide more openness and transparency in this process, with greater patient involvement, particularly at the local level.

Guidance

44. Having considered the roles and responsibilities of the various organisations that operate in the cancer drug appraisal arena, we gave consideration to how guidance that emerges from that process filters down to clinicians on the ground. Both themes are obviously intertwined. We were keen to clarify what role there was for patient involvement in the preparation of guidance, whether clinicians were operating in an atmosphere of clarity and understanding given the levels of organisation above them and whether guidance was being applied equitably across all NHS boards.

45. It is the expectation of the Scottish Government that NHS boards and clinicians take ‘full account’¹² of initial SMC advice following its appraisal of a drug and the guidance from NHS QIS in the light of a NICE multiple technology appraisal. We are unsure of how this ‘expectation’ is policed by the Scottish Government and it would be useful for it to clarify this in its response to this report.

46. We welcomed the helpful clarification given by Dr Kenneth Paterson, Chairman of the SMC in oral evidence that, as the SMC is effectively a consortium of ADTCs, NHS board chief executives will accept the advice that emanates from the SMC given “they have signed up to taking on board that advice”.¹³

47. Our attention was drawn to NHS circulars HDL(2003)60¹⁴ and HDL(2007)26¹⁵ which effectively require NHS boards to follow SMC advice. In the view of NHS Grampian, this helps to reduce postcode prescribing.¹⁶ However, we did inquire into whether the implementation of guidance across NHS boards was variable and, while there was nothing to suggest that this was a particularly serious problem that existed, we did note some remarks made in responses.

48. For example, the North of Scotland Cancer Network, of which NHS Grampian is part,¹⁷ in its response said that when it conducted an audit of implementation there did appear to be more variation than it would have expected.¹⁸ Further, in response to another question from us, it indicated that the application by clinicians of guidance varies between NHS boards within its area. Such a situation must clearly be addressed.

49. In addition, Cancer Research UK in its written evidence drew attention to the need for greater transparency in the decision making process, and we have referred to this earlier in the report. It stated it would like to see improved audit arrangements for NHS boards to record how recommendations of emerging SMC advice is considered and incorporated into future decisions.¹⁹

50. There may be scope for confusion here. While the guidance preparation and issuing process appears to work well, it is not conclusive that there is then equal and consistent application of it at ground level. While there is scope for a degree of acceptability about this in the sense that it is only guidance therefore clinicians have flexibility in terms of what information they use to substantiate clinical decisions, some further investigation is required into the extent of regional variations and whether this is causing difficulties for clinicians and, more importantly, patients. That is a matter for the Scottish Government to investigate further. It will wish to satisfy itself that there is no issue for concern here.

51. It is worth drawing attention here to our impression that there is no evidence to suggest that a system of postcode prescribing exists across NHS boards. While, until a few years ago, one particular drug may have been available in Edinburgh but not in Glasgow the position in terms of drug availability is now perhaps more subtle. For example, one NHS board may use a particular drug to treat a condition while another board will use a different drug. Importantly, the patient is not denied appropriate treatment, simply the local ADTC has factored in local needs and circumstances. To expand this example, an ADTC in a rural area might recommend a drug that can be taken orally as this will reduce the need for the patient to travel to a local health clinic for a medicine that must be administered intravenously while, in an urban setting, that consideration may not be relevant. The claim was made in evidence that no patient has been denied the appropriate therapy. The issuing of national guidance and advice has contributed largely to this. That is why it is important that our comments above about the universal implementation of guidance and advice are properly addressed so that difficulties in terms of postcode prescribing do not emerge.

52. We were particularly interested in the statement by the SMC in its written evidence that it was unable to assess the uptake of its advice across Scotland as a result of insufficient data gathering although, in oral evidence, it did remark that its advice is being followed²⁰ (although, as we have just stated, we are not convinced that there is conclusive evidence that there is equal and consistent application across NHS boards of SMC and NHS QIS advice and guidance). It expanded on this in its oral evidence.²¹ This was an interesting issue. Having created clear and accepted procedures in the appraisals process, with effective communication between key organisations, and for guidance and advice to then emerge from that process which filters down to clinicians on the ground, it seems strange that there is no informed data collected which indicates how the guidance is then being applied and what benefits it is bringing about in providing good cancer patient care. We turn to this in the next section of our report.

53. We were encouraged by the view of SMC, NHS QIS and cancer networks, in addressing one of the specific parts of our remit, that there appear to be no anomalies in terms of the implementation of guidance for cancer treatment drugs and that for other life threatening or terminal illnesses. However, we do draw attention to the views expressed by the Association of the British Pharmaceutical Industry (Scotland)/Scottish Cancer Industry Group²² and the Royal College of General Practitioners patient group²³ who consider that anomalies exist in terms of implementation. Clarity is required. Put simply, is advice and guidance being implemented equitably and consistently across all NHS boards and what impact is this having on access to cancer treatment drugs? We draw the attention of the Scottish Government to this for further consideration as part of its investigation referred to above.

Conclusion to this section

54. We invite the Scottish Government, in its written response to this report, to—

• clarify how it monitors the expectation that NHS boards and clinicians take ‘full account’ of initial SMC advice following its appraisal of a drug and guidance from NHS QIS in the light of a NICE multiple technology appraisal; and
• investigate the apparent regional variations in the implementation of guidance, whether such variations are of concern and, if so, what action it is taking as a result

Data gathering

55. We refer above to concerns expressed, particularly in oral evidence,²⁴ about a lack of proper, useful data. This is not an issue that we identified as a core topic of our inquiry but we are grateful that our inquiry has given rise to what appears to us to be a key missing element. Dr Andrew Walker, a health economist and member of the SMC’s New Drugs Committee, was particularly helpful in highlighting this matter. He stated that there was no national system of data collection that would show, for example, how many patients are getting one drug and how many patients another. While such information may be available locally, it was not nationally and we are unaware of any procedure in place whereby NHS boards are able to exchange data (or whether there is any system in place which indicates what data might usefully be collected, albeit locally). One area where data collection would be useful is that covered in the previous section, the implementation of advice and guidance across NHS boards.

56. On this issue of local collection, Professor Alan Rodger, medical director of the Beatson Oncology Unit, welcomed the ability to produce better data. He drew attention to work undertaken by pharmacy staff in the west of Scotland area in this regard although he did consider that the work was hampered as a result of a shortage of pharmacy staff. That is an issue for the Scottish Government to reflect on.

57. The production of better data was certainly something that would be welcomed by NHS boards and this was clear from their oral evidence.²⁵

58. It appeared to us from the oral evidence of the SMC that the lack of data was hampering health professionals at national level in identifying where difficulties perhaps exist and where improvements can be made. We welcome the recognition by the Cabinet Secretary for Health and Wellbeing in her oral evidence²⁶ of this issue and her commitment to consider this data gathering issue. The evidence submitted to us indicates that data needs to be captured and we consider it is for the Scottish Government to identify what data and how it is captured. It is obvious that a discussion needs to take place between it and key organisations such as the SMC to identify what data should be gathered, why, how this can be done, by whom and what purposes this data can then usefully fulfil. There will no doubt be a resource impact but if resources are allocated for this then it may, in the long term, identify where perhaps wastage is taking place and where saving can be made across NHS boards. Importantly, it can lead to improvements in cancer patient care.

59. Further written evidence submitted to us following our oral evidence session on 29 April 2008 highlighted where some of this data gathering might be focussed. The SMC drew attention to an ongoing project within NHS Information Services Division to bring together data on drug utilisation.²⁷ There is an indication that some information is being gathered in respect of primary care but not in secondary. It is important that any procurement work currently ongoing or scoping for projects into data capture is reflective of the needs highlighted in the evidence to us and the Scottish Government should ensure that no opportunity is lost in this regard.

Conclusion to this section

60. We invite the Scottish Government, in its written response to this report, to—

• indicate how it will take forward discussions with key health professionals and others as appropriate on the provision of a national data gathering system; clarify what data will be gathered, why, how this will be done, by whom and what purposes this data will fulfil; and indicate the timeline for taking this forward; and
• give careful consideration to any procurement or scoping work for data gathering projects and how these might be adapted, if appropriate, to capture more extensive data as highlighted in the evidence to this Committee.

Quality-Adjusted Life Year (QALY)

61. Before moving to the next topic, it may be helpful to consider the evidence we received on the issue of quality-adjusted life year or QALY. This is a particularly complex area and one that inevitably gives rise to strong arguments, focused as it is on the measurement and valuation of health benefits. We are particularly indebted to Dr Andrew Walker for his very helpful written submission made available to us after his oral evidence on 29 April 2008.²⁸

62. In summary, a QALY seeks to capture, in a single measure, the fact that medicines aim to prolong life, improve the quality of life, or do both. From the written evidence we were informed that it is an internationally recognised comparator in cost utility analysis and a widely recognised methodology in health economics. Similar systems to that used in Scotland are used in England, Sweden, Canada and Australia. It is welcomed that it is this principle of prolonging the quantity and improving the quality of life that appears to be the overarching principle in the appraisal process.

63. We of course recognise that decisions on drug availability will at times be difficult and, inevitably, judgements and decisions will be taken that some patients and their family, relatives, friends will find difficult to accept. However, we start from the premise that a process such as QALY should feature in the overall decision making process on a drug’s availability. From that, we must all be satisfied that a QALY process is sound, fair, just, transparent and applies logical criteria. It must also be humane.

64. We were pleased to note from the written evidence from NICE that cost utility analysis is not the only factor in reaching decisions. Judgement must be exercised when considering the results of cost effectiveness analyses by setting them in the broader context of the disease or condition against which they are used, the availability of other treatments, the specificity of the results of the economic modeling and the attitudes and preferences of society to the social value judgements which they need to make. It is too easy for critics to say that appraising a drug is all about economics. It is not and the evidence we considered did not lend itself to any such conclusion. Dr Walker in his oral evidence on this matter stated—

“The cost of the drug is not the only issue – we consider the patient’s whole experience”.²⁹

65. This point was supported in oral evidence by Dr Harpreet Kohli, medical advisor to NHS QIS who said in his oral evidence—

“The QALY should be seen in the context of the other information that is before the SMC. As a mere public health physician, I would be worried if any assessment considered only the cost per QALY, but it does not; all the other information is taken into account. The QALY is not a technical answer but is a tool that helps us to assess and evaluate. In this case, it helps us to evaluate cancer drugs”.³⁰

66. At a local NHS board level, Professor Alan Rodger, medical director of the Beatson Oncology Unit stated—

“The decision is driven not by the cost of the drug but by the clinical judgement of a particular patient’s situation”.³¹

67. The written evidence from cancer networks, NHS QIS, SMC and others all leaned towards a belief that the QALY system was both fair and robust. There was also honesty that it has its limitations and that, reading into the evidence, there is call for an assessment to be undertaken into where improvements can be made. For example, the West of Scotland Cancer Network in its written evidence stated it would welcome further research into health economic methodologies to improve and refine how benefit is quantified. This is an issue we would invite the Scottish Government to respond to. We are aware that, from responses to other petitions, the Chief Scientist’s Office does not seek to outwardly invite research but, rather, to consider proposals for research submitted to it. However, this may be an area where the Scottish Government should proactively commission research if it was considered necessary.

68. It will, of course, wish to give careful consideration to what the scope of any research required would be and this should, we consider, be done in consultation with key health professionals such as the cancer networks and the SMC, cancer groups and patient representative bodies. Here we draw attention to our consideration of the evidence received on drug pricing and how issues arising from that might feature in this research (see paragraphs 103-109).

69. We should like to also draw attention to the view of Cancer Research UK where it alludes to ‘uncertainty’ amongst the health community (professionals and patients) on how cancer treatment drugs are judged. It advocates greater public input into discussions about ‘rationing of NHS resources’ and the QALY process. It will come as no surprise that we support such involvement. There are no doubt important views and considerations that public and patient bodies can bring to the table and we must not be too precious about protecting the current system by ignoring such views.

Conclusion to this section

70. We invite the Scottish Government, in its written response to this report, to indicate how it will take forward initiating research into the area of health economic methodologies, and the QALY process, and to take forward consideration of the scope of any such research with key health professionals and cancer groups and patient representative bodies.

Availability (covering exceptional prescribing)

71. We turn our attention now to the provision of cancer treatment drugs at the local level. As set out earlier in the report, the SMC will appraise a cancer treatment drug and then NHS QIS will issue guidance to NHS boards outlining the assessment and recommendation. It is then for the local ADTC to make a decision on the drugs formulary status. That is, decide whether the particular drug should be used locally. Our focus, obviously, is on the situation whereby the SMC or the ADTC has assessed that a drug is not made available on the NHS but a patient’s clinician does consider that particular drug would be beneficial to the patient and should be prescribed. It is at this point that the clinician can raise the matter, through the board’s ‘exceptional prescribing’ arrangements, and make the case for that drug to be funded by the board. For example, the clinician has assessed that the circumstances and the criteria applied to the individual case differ from those considered by the SMC or the ADTC.

72. It is argued that having exceptional prescribing panels is the only equitable method to ensure that all those with needs for different treatment and therapies, including children, the elderly, and those with acute and chronic diseases and conditions are guaranteed an opportunity to put forward the evidence available for an individual, exceptional decision. Further, equity requires that different decisions may be reached in different cases and circumstances on the basis of the available evidence. Guidance issued by the previous Scottish Executive in 2007³² states that NHS boards should have protocols and processes in place to consider non-NHS formulary treatment requests.

73. Having established that NHS boards are required to have such a regime in place, we were interested, particularly in light of the experience of the petitioners, to understand what this process was and whether it was fair, transparent, logical, easy to navigate through etc. Also that there is equity and consistency across every NHS board in its application and that there are agreed, identified minimum requirements applied. In essence, all the elements you would justifiably expect a process to include given the particular circumstances that may give rise to a patient seeking to go through it. That is, a patient having been told that they have a terminal condition and that the drug the clinician wishes to prescribe is not on the local NHS board’s formulary. It soon became apparent that the system appears to be anything but what expectations might be.

74. One of the issues we were keen to address, that came out of the process that Michael Gray went through, was that at no point should a patient, diagnosed with cancer, be burdened as a result of the procedures in place with undue, unnecessary stress or anxiety given the situation they are already facing. We wanted to know that the options available to a patient were readily identifiable and, upon investigation, the advice would be found to be clear, balanced and informed. That is not what can be said of the advice on co-funding of treatment.

75. A number of difficulties and clear areas for improvement were highlighted, starting with the name, ‘exceptional’. We quote Tina McGeever in her oral evidence³³ to us—

“Although the health board might not think that Michael is exceptional, I certainly do.”

76. However, this is more than just window dressing through changing the name of the process, as unacceptable and inappropriate as the term is. It is about the fundamental improvements that must be made to the process itself.

77. We did consider whether such a process must exist. Could the clinical judgement of a patient’s oncologist that their patient should be prescribed a particular drug not on the local formulary not be sufficient reason for the local NHS board to then fund that treatment? But that may place undue pressure on an individual clinician to take the collective decision that the NHS board should fund the treatment without recourse to a wider discussion and assessment.

78. That wider discussion and assessment would elaborate on the cost of the drug concerned and the QALY, safety issues, risk factors and possible legal considerations. Ultimately however, and witnesses were quite clear on this, it is a clinical decision as to whether the drug should be funded and we highlight this earlier in the report. We recognise that wider discussion by the clinician with others could also elaborate on particular clinical and treatment issues such as whether other drugs (perhaps on the local formulary) might be more suitable and what the likelihood is of the particular drug having the positive outcome desired. We agree that some ‘panel’ of clinical and health professionals, perhaps similar to that which exists at present, should exist. However, there are two important issues to be drawn out here that must be improved before the point this ‘panel’ meets to consider such applications from a clinician for a non-formulary prescription.

79. First, at the point that a patient receives news they have a terminal or life threatening form of cancer (and this should apply in the case of every terminal or life threatening condition), there must be information, guidance and advice made available to that patient indicating what options are available to them and what they can do to pursue each option. In the case of going down the ‘exceptional prescribing’ route, the guidance should indicate that such a process actually exists, and then explain its purpose, how it works, what it does and how the patient can access more information. A clear impression emerged from the inquiry of patients having to ‘fight their way’ through the system to access relevant information and the time that it can take to do this. Given the circumstances of patients who may have a terminal condition, and where time therefore is so very precious, that is simply not acceptable.

80. We are mindful of the point made by Dr Jean Turner of Scotland Patients Association in her oral evidence that some patients, on hearing news such as this, will simply want to turn and face the wall.³⁴ But here it is about choice and availability. Choice to seek the information, for the patient to quickly and simply broaden their understanding of what they can do and how, and then making the information readily available.

81. The information can be made available in simple formats. For example, the NHS board’s website, handout leaflets or an information pack which the patient could take away and read after the initial shock of their diagnosis has reduced. Information must have regard to standard, plain English (we did notice in some literature patients being referred to as ‘appellants’). It must be reviewed and updated as necessary. On too many occasions do we see outdated information stored on organisations’ websites. We consider that patient bodies should be involved in drawing up such information as well as cancer charities if appropriate.

82. Second, and a point which emerged from across our oral evidence sessions, in particular on 20 May 2008, was the weight that is attached to the view of the clinician throughout this process and how that person, and most importantly the patient, at every stage of the process, are actively involved in the consideration of whether the drug is funded. This leads us on to our most critical area, that of patient involvement, transparency and again lack of information.

83. Takingas the premise that the patient has stated he or she wishes to be involved in the process and the specific consideration of their particular case then, at each stage of that process, the local NHS board should inform the patient of precisely what will happen, when, where and why. The patient, and their clinician, must be given the option of attending every meeting at which their case is considered including the meeting at which a decision is made as to whether the particular drug is to be publicly funded. Minutes or some form of record should be taken of each meeting, indicating clear timed action points which should be made available to the patient and their clinician within 24 hours of the meeting.

84.We believe that it would be beneficial to the patient if they knew precisely how long this process will take from start to finish. If the starting point is the submission by the clinician of the evidence that this drug should be funded and the finishing point is the decision on whether to fund it, then a firm timetable should be produced by the board and made available to the patient and the clinician. We would consider two weeks to be a maximum time. Any timetable deviation should be justified and discussed tri-party. It would be helpful for the Scottish Government to indicate how this will be put into practice.

85. One suggestion worthy of consideration is whether an individual within the NHS board could be appointed to act as a liaison between the board, the patient and their clinician thereby establishing a clear, recognised, understood and direct line of communication. This person would also act as an information point to respond to any procedural questions that the patient or the clinician may have. It is essential that such an individual was trained and familiar in the processes that exist in their board area. We support the idea of an—

“… early follow-up appointment with somebody who can go through the information with the patient and explain the whole system

as suggested by George Darroch in oral evidence.³⁵

86. Further to this, in terms of health equality issues, we consider that support and guidance must be given to those who are not able to question decisions themselves and that independent advocacy services should be made available to those cancer patients who, for whatever reason, are disempowered.

87. We turn now to the point at which the ‘exceptional prescribing’ panel meets. We state above our views on the need for transparency. However, there is a further issue here and that is the level of public involvement on such panels. Some form of patient representation on the panel is required to present a non clinical, cost, legal, risk etc dimension to the decision. This is also the case with an ADTC where there needs to be greater public involvement in their work and representation on the committee itself.

88. A further area that concerned us was the inequity and lack of consistency by NHS boards in this area, even within regional cancer networks. While we support the notion of flexibility, there clearly needs to be a more consistent approach adopted across all NHS boards. Some boards felt their procedures work well and perhaps they do. However, we were unaware of any analysis that, at the very least, sought the views of patients who had gone through this ‘exceptional’ procedure and whether the experience was good or bad.

89. We were pleased to note that the West of Scotland Cancer Network³⁶ is taking forward work to develop a consistent approach to non-formulary requests across the local NHS boards in its area. That is to be welcomed and the Scottish Government should consider whether there are useful measures that can be rolled out across all boards.

Conclusion to this section

90. We invite the Scottish Government, in its written response to this report, to indicate—

• how it will put in place the production of information that can be made available to patients setting out options and directions for the ‘exceptional prescribing’ route’;
• how it will involve patient bodies and others in the preparation of this information;
• how it will develop a more participatory process for the patient and their clinician in the consideration of their ‘exceptional prescribing’ case, a process that will indicate a clear timeline for action that is no longer than two weeks;
• how it will improve communication between the NHS board, the patient and the clinician throughout this process, including the appointment of ‘liaison officers’;
• how the process will allow for participation by the patient and their clinician in all meetings of the ‘exceptional prescribing’ panel and that the panel itself has a patient representative body member on it; and
• how it will develop a consistent approach across all NHS boards in considering non-formulary requests.

Funding

91. Our consideration here focussed on the points made in oral evidence³⁷ by Michael Gray about the co-public and private funding of treatment. At that meeting we were concerned at the process that he and his wife had gone through in having to fund the cost of his treatment.

92. The starting point is the guidance³⁸ issued by the Scottish Executive’s Chief Medical Officer and Chief Pharmaceutical Officer to the NHS. The stated purpose of this letter is to set out ‘guidance on the legal position and the process that should be followed in such circumstances’. The letter runs to five paragraphs but we were astonished, in the oral evidence on 29 April 2008, at how much confusion it causes.

93. The views expressed in oral evidence are unambiguous. NHS Grampian stated³⁹—

“We would appreciate more clarity on mixing private and NHS treatment. We have tried to obtain clarity on that, but we are not getting it.”

And—

“because it is not practically possible to make the separation in the way the health department letter suggests—we have difficulty with it because it places us in the uncomfortable position of having to make judgments on a case-by-case basis.”

While Professor Rodger said⁴⁰—

“The situation is a moral, ethical and logistical nightmare and minefield.”

94. We do agree with the Scottish Government in its written response to us⁴¹ that the guidance should ‘provide a framework within which NHS boards should operate in relation to concurrent treatment’ and that ‘there needs to be clarity’. Clearly, neither exists.

95. We have no doubt that there are, as the Cabinet Secretary for Health and Wellbeing clearly stated in her oral evidence,⁴² financial, ethical, moral and clinical considerations that must all be factored in. We accept that it is simply not possible to split pro rata a patient’s treatment costs between that received privately and that on the NHS. We are mindful of the points made about clinical accountability. We also note the point made about creating a two tier system within the NHS of those who can afford to pay and those who cannot. Here, we draw particular attention to the written evidence of NHS Grampian⁴³ and how the existing policy requires self funding of associated costs such as administration, monitoring and testing. We wonder whether, in some areas, the considerations surrounding co-funding might be easier resolved than in others and at least remove some financial burden from an individual who does wish to self fund treatment.

96. We must also recognise the situation that currently exists. Access to a drug treatment that is not available on the local formulary may, to a degree, be ‘relatively inexpensive’. The difficulty created is not just the cost of paying for that drug privately, which may be achievable to many people, but the rise in the cost of the treatment when the associated NHS treatment costs are added to this. It is this escalation that can then put the cost of the treatment beyond the patient. That does not seem an appropriate situation to create for an individual who is simply looking to receive a treatment that may prolong their life. We then wonder, in such a situation, whether access to treatment really does then come to money and how this squares with the principle of access to treatment being based on clinical judgement.

97. While the evidence in response to our questions referred to the complexities involved, it was in a way, a simple and swift conclusion to reach. There is quite clearly a need for a thorough review of the guidance and of the principle of co-funding of treatment itself. The written and oral evidence, particularly from cancer networks, was unambiguous and strongly put. We referred earlier to our desire to see a system that ultimately allows clinicians to operate in an atmosphere of clarity and understanding. To do so, allows them to provide optimum patient care and advice. The guidance is not allowing this. Rather it is putting them in awkward situations and causing unnecessary and unacceptable difficulty.

98. There was perhaps a perception that, through this process of ‘exceptional prescribing’, a patient was having to plead before a panel for a drug to be funded. Whether that is in fact the case, that such a view exists clearly shows that an important part of the process is not working properly. There was also a feeling, and Tina McGeever in her oral evidence amplified this, that there was an onus on the patient to prove the value of the drug in question. For example, she and her husband privately funded the treatment of the cetuximab drug recommended by his oncologist. After it was shown to be proving effective, the local NHS board agreed to publicly fund the treatment.

99. We wonder whether some sort of system could be put in place whereby, on the recommendation of the clinician, the NHS board agrees to fund an agreed initial ‘trial’ of the drug through a set number of treatments to generate initial and early feedback from the patient and, importantly the clinician, on whether the drug is proving to be clinically effective. If it is, then that should improve the case for a more long term funding arrangement. If the clinical analysis is that the drug is not proving effective, not improving the quality and quantity of the patient’s life, then it would then be up to clinicians to consider what the next most suitable course of action should be.

100. We certainly welcome the commitment by the Cabinet Secretary to “take a fresh look at” the guidance letter.⁴⁴ There is clearly a need for urgency in addressing this issue given there are many other patients daily facing the same issue that Michael Gray and Tina McGeever faced. We are not recommending that a new process be rushed in, this matter is far too important and serious for that. Rather, the Scottish Government should identify what the difficulties are that clinicians at NHS board level are facing, how these can be properly resolved and whether there are “fresh” and innovative ideas out there that are worthy of consideration in terms of co-funding. We cannot offer up any best practice example of this. Indeed, we asked our witnesses this very question at our 29 April 2008 meeting.⁴⁵

Conclusion to this section

101. We invite the Scottish Government, in its written response to this report, to clarify how it will take forward a review of its guidance to NHS boards on concurrent NHS and private treatment and the policy itself.

Other issues

Pharmaceutical price setting

102. The prices of branded prescription medicines and the profits that manufacturers are allowed to make on their sales to the NHS are regulated by the pharmaceutical price regulation scheme (PPRS) which is a voluntary agreement made between the Department of Health and the branded pharmaceutical industry (represented by the Association of the British Pharmaceutical Industry) under section 33 of the Health Act 1999. There have been a series of voluntary agreements with the industry since 1957 to limit branded medicine prices and profits, each lasting five years or so, although the details of these agreements have evolved over time.

103. According to the ninth report of the PPRS,⁴⁶ a new five-year scheme negotiated with the ABPI commenced on 1 January 2005 in succession to the 1999 scheme. It includes a price reduction of 7% in the prices of branded prescription medicines supplied to the NHS as part of a package of measures, which provide stability for the industry and reward innovation and research whilst keeping public expenditure under control. The objectives for the 2005 scheme are unchanged from those for the 1999 scheme and, as stated in the agreement, continue to be to—

• secure the provision of safe and effective medicines for the NHS at reasonable prices;
• promote a strong and profitable pharmaceutical industry capable of such sustained research and development as should lead to the future availability of new and improved medicines; and
• encourage the efficient and competitive development and supply of medicines to pharmaceutical markets in this and other countries.

104. We noted that a recommendation of the Office of Fair Trading (OFT), following an inquiry into the PPRS,⁴⁷ was that the scheme should be reformed to deliver better value for money from NHS expenditure on drugs and to focus business investment on drugs that have the greatest benefits for patients. It identified a number of drugs where prices were significantly out of line with patient benefits. It also recommended that the current 'profit-cap and price-cut' scheme be replaced with a patient-focussed, value-based pricing scheme in which the prices the NHS pays for medicines reflect the therapeutic benefits they bring to patients. This would enable the NHS to obtain greater value for money from its existing drug spend. It proposes two options under which the prices of on-patent branded prescription drugs could be set according to value-based principles—

• Ex-post value-based pricing: this would involve retaining upfrontfreedom of pricing for companies but would replace company-wide profit controls and price cuts with a series of reviews of the cost effectiveness of individual drugs or drug classes, conducted some years after launch.
• Ex-ante value-based pricing: this, in addition to the ex-post reviews, would involve a fast-track ex-ante assessment of a new drug's cost effectiveness before launch.

105. The Department for Business, Enterprise and Regulatory Reform published the UK Government’s interim response⁴⁸ to the OFT report in August 2007 and agreed that drug pricing arrangements should be updated.

106. There are clear issues to be taken forward in the light of the OFT report and the UK Government’s response. Again, in oral evidence, there was a call for greater transparancy and more open discussion on this matter and Cancer Research UK felt that a different approach to pricing would be helpful.⁴⁹ There are some important issues to be addressed in terms of the price set by the pharmaceutical company for a drug, for example in relation to an ‘orphan drug’ (a drug which perhaps has a limited target population or which treats a rare disease thereby limiting its financial potential), and the relationship with the QALY (quality-adjusted life year).

107. While the PPRS is largely a reserved area, and one for the UK Government to take the lead on as acknowledged by the Cabinet Secretary for Health and Wellbeing in her oral evidence,⁵⁰ there are clear impacts on devolved areas in terms of bringing about better cancer patient care which is what we are trying to achieve through this inquiry and report. We would support direct communication by the Scottish Government to the UK Government in the light of this report and the evidence given to us. This is particularly important given the difficult and sensitive issues that apply when a QALY analysis is being undertaken and how the price set for the drug concerned factors into that consideration.

Conclusion to this section

108. We have invited the Scottish Government (paragraph 71) to carefully consider the case for initiating research into the area of health economic methodologies. An important part of that research should be how the price of a cancer treatment drug is set and the impact of the price in the QALY analysis.

Overall conclusions

109. We asked witnesses during oral evidence gathering what procedures they would like to see in place in five years time. Essentially, what lessons can be learned here. We received some clear responses—

• Scotland Patients Association wanted a considerably shortened process when a NHS board is considering a non-NHS formulary application;⁵¹
• Cancer Research agreed and, in addition, wanted to see a lot more transparency in the process for implementing guidance. Also, different models of pharmaceutical pricing and a more liberal regime to allow people to help themselves though the process;⁵²
• the Scottish Medicines Consortium also wanted greater speed and transparency at local levels. On top of that was the need for consistency in the decision making process. It also referred to a value-based pricing structure;⁵³
• NHS Quality Improvement Scotland wanted greater data collection and evidence and identified the need for bodies involved in this area under constant review to identify any scope for streamlining;⁵⁴
• the National Institute for Health and Clinical Excellence hoped for advancements in science that generate greater health benefits for patients; it also drew attention to the drug pricing structure.⁵⁵

110. We support each of these which we recognise will all bring about improvements in procedures and ultimately patient care. There is no more important outcome. It is for the Scottish Government to reflect on these desires and to indicate how it will turn them into realities and when. When asked this question, the Cabinet Secretary stated her commitment to working towards improving procedures in the future.⁵⁶ We conclude on this point with what Tina McGeever said in her oral evidence⁵⁷—

“… it is important that when somebody is told that they are terminally ill, they go away with information that they can come back to later. There should also be a key person whom the patient can trust and who can take them through the minefield as they progress with clinicians. We must start with the basics. We need stepping-stones, so that people know the road that they can take. We do not have that at present, but it is important for the patient that it is put in place”.

111. This has been a particularly worthwhile inquiry and the Committee was privileged to have the opportunity, through the petition, to take it forward. There has of course been a particular emotional and human aspect to how the inquiry started and we have not lost sight of that. However, we believe we have been objective throughout in investigating wider public and policy issues. There are clear and compelling grounds for much needed improvements in this process, particularly in terms of data gathering, health economic methodologies and the QALY process, exceptional prescribing and the co-funding of treatment. A simple read of the oral evidence from 29 April and 20 May 2008 make that abundantly clear.

112. We hope, through this report, we have highlighted clear areas for further investigation and action. We appreciate the open approach of the Cabinet Secretary for Health and Wellbeing in her oral evidence to work with the Committee, to consider carefully our report and the issues raised through the written and oral evidence. In that spirit of participation, we look forward to clear action points and timelines from the Scottish Government. We would consider that some of the issues highlighted in this report can easily, readily and quickly be addressed with some imagination and commitment. Others, we accept will take longer but it is important that a timeline is identified for actions. We are aware that the Scottish Government will shortly announce, following its discussion on Better Cancer Care, how its cancer strategy will be developed. We hope that our inquiry can positively contribute to that strategy.

113. The issues that we have highlighted are not unique to Scotland. We are aware that similar concerns and questions are being asked in England on matters surrounding exceptional prescribing and co-funding of treatments. Indeed, we noted with interest the debate held in the House of Commons on 29 April 2008 on these very issues⁵⁸ and we would actively encourage the Scottish Government to liaise closely with the Department of Health in England on how the UK Government is addressing these matters. For example, what relevance and lessons can NHS boards and ADTCs in Scotland learn from the outcome of the review due later this year on the democratic accountability of primary care trusts (in England).

114. Finally, and most importantly, we hope we have done justice to the petition itself, to the work of both Tina McGeever, and her late husband Michael Gray, and to the trust they placed in this Committee in taking forward their petition. It is through them and their petition that we have highlighted clear areas for action. It will be their making that the improvements that we expect to emerge from this inquiry, and which the Scottish Government will amplify in its response, have come about. These improvements may have eventually come about later through some other process, but better they are sooner.

115. It is our intention to continue consideration of the petition on the back of the response from the Scottish Government. That said, the Committee hereby sets out its overall conclusions—

Defining roles (paragraphs 36-43)
We invite the Scottish Government, in its written response to this report, to indicate—

• whether any form of monitoring is required of the roles of the national and local bodies in the drug appraisal process and what streamlining and efficiencies can be identified and introduced;
• how performance management arrangements, as referred to in its written evidence to us,⁵⁹ would be used to address any shortcomings and when it would consider it appropriate to use such arrangements; and
• how it will provide more openness and transparency in this process, with greater patient involvement, particularly at the local level.

Guidance (paragraphs 45-54)
We invite the Scottish Government, in its written response to this report, to—

• clarify how it monitors the expectation that NHS boards and clinicians take ‘full account’ of initial SMC advice following its appraisal of a drug and guidance from NHS QIS in the light of a NICE multiple technology appraisal; and
• investigate the apparent regional variations in the implementation of guidance, whether such variations are of concern and, if so, what action it is taking as a result.

Data gathering (paragraphs 56-60)
We invite the Scottish Government, in its written response to this report, to—

• indicate how it will take forward discussions with key health professionals and others as appropriate on the provision of a national data gathering system; clarify what data will be gathered, why, how this will be done, by whom and what purposes this data will fulfil; and indicate the timeline for taking this forward; and
• give careful consideration to any procurement or scoping work for data gathering projects and how these might be adapted, if appropriate, to capture more extensive data as highlighted in the evidence to this Committee.

Quality-Adjusted Life Year (QALY) (paragraphs 62-70)
We invite the Scottish Government, in its written response to this report, to indicate how it will take forward initiating research into the area of health economic methodologies, and the QALY process, and to take forward consideration of the scope of any such research with key health professionals and cancer groups and patient representative bodies.

Availability (covering exceptional prescribing) (paragraphs 72-90)
We invite the Scottish Government, in its written response to this report, to indicate—

• how it will put in place the production of information that can be made available to patients setting out options and directions for the ‘exceptional prescribing’ route’;
• how it will involve patient bodies and others in the preparation of this information;
• how it will develop a more participatory process for the patient and their clinician in the consideration of their ‘exceptional prescribing’ case, a process that will indicate a clear timeline for action that is no longer than two weeks;
• how it will improve communication between the NHS board, the patient and the clinician throughout this process, including the appointment of ‘liaison officers’;
• how the process will allow for participation by the patient and their clinician in all meetings of the ‘exceptional prescribing’ panel and that the panel itself has a patient representative body member on it; and
• how it will develop a consistent approach across all NHS boards in considering non-formulary requests.

Funding (paragraphs 92-101)
We invite the Scottish Government, in its written response to this report, to clarify how it will take forward a review of its guidance to NHS boards on concurrent NHS and private treatment and the policy itself.

Other issues
Pharmaceutical price setting (paragraphs 103-108)
We have invited the Scottish Government (paragraph 71) to carefully consider the case for initiating research into the area of health economic methodologies. An important part of that research should be how the price of a cancer treatment drug is set and the impact of the price in the QALY analysis.

116. We consider that some of the issues highlighted in this report can easily, readily and quickly be addressed with some imagination and commitment. Others, we accept will take longer but it is important that a timeline is identified for actions. We therefore invite the Scottish Government, in its response, to give a timeline for each action point above.

Footnotes: