Santiago, October 2010 Sir Michael Rawlins, Chairman, NICE

1. Social participation for sustainable and legitimate prioritisation processes: NICE’s experience Santiago, October 2010 Sir Michael Rawlins, Chairman, NICE KalipsoChalkidou, Director, NICE International

2. The NHS in 2010 Tax-revenue allocation by government with limited co-pays and out-of-pocket UK parliament NHS vote every 2 years Secretary of State for Health/Department of Health Other bodies (e.g. Care Quality Commission) NICE 152 regions across the country Primary Care Trusts guidance 10 care doctors Hospitals Hospital doctors

3. 1997: Announcing the establishment of NICE • The National Institute's membership will be drawn from the health professions, the NHS, academics, health economists and patient interests. It will need to have access to an appropriate range of skills, including economic and managerial expertise as well as specialist input on specific issues. • NICE will create a new partnership between the Government, the NHS and clinical professionals…NICE will offer doctors, nurses and midwives more support than they have had before in making the complex decisions about individual patient care often required in modern health care. • It will also inform the decisions of those commissioning care

4. NICE’s features Our objectives Our principles Reduce inappropriate geographical variation Set quality standards for the practice of medicine Encourage innovation in health technologies Promote efficiency in resource allocation • Independence from government, industry and professionals • Transparency • Inclusiveness and public consultation • Scientific basis for decisions

5. NHS Evidence NICE structure Information systems Topic selection Communications Implementation Developing NICE guidance R&D Patient and public involvement Centre for Public Health Excellence - public health interventions - public health programmes • Centre for • Health Technology Evaluation • - technology appraisals • surgery • diagnostics • medical devices • Centre for • Clinical Practice • clinical guidelines • quality standards • QOF

6. Patients and Citizens Professionals Industry Payers and Providers Stakeholders EU and UK legislators Popular Media More than 3,000 external experts, including patients, health professionals, academics, researchers, industry representatives and lay members of the public, offer their time and experience to NICE every year… Ministries of Public Health and of Finance ParliamentPolitical Parties

7. Stakeholder input Topic referred to NICE* Scoping* Development Draft Guideline Public Consultation* Independent Review of response to public comment* appeal Publication* legal challenge Dissemination and Implementation * Published on NICE web site

8. Working with the country’s professionals: the National Collaborating Centres

9. How does NICE engage with patients and the public? • Lay and patient membership of committees • Public consultation • Dedicated patient and public involvement unit • Engagement in methods and processes development • Citizens’ Council and social value guidance • Regular opinion polls • Lay versions of all NICE products

10. All technology appraisals produced in versions for patients and the public, known as “Understanding NICE Guidance” Plain-English translation of guidance, not detailed patient information Includes details of key patient support organisations for further information Communicating with patients “If you are taking a beta-blocker (either on its own or with some other medicine), then you should continue taking it. Your GP may discuss other treatments with you at your next regular visit. If treatment with the beta-blocker is not working, then your GP may suggest trying one of the treatments described above.” “If it appears that you are suitable for the treatment described in this booklet, but it is not available, you should talk to your local Patient Advice and Liaison Service. If they are not able to help you, they should refer you to your local Independent Complaints Advocacy Service.” “It’s often possible to get less expensive non-branded medicines that are just as good as the branded versions. GPs are recommended to use the non-branded versions when this is possible, to get the best value for money”.

11. Seeking Patient Input: which outcomes matter?Case study - Psoriasis Clinical research and the specialists told us the amount of psoriasis was what most affected the quality of life. Patients told us that the location of the flare-up (e.g. face or joints) was more significant.

12. Assessing value is context-specific

13. Rawlins, Lancet Oncology, 2007 NICE’s Citizens’ Council • Our legal responsibilities on human rights and discrimination • The innovative nature of the technology and value added compared to alternatives, especially at the end of life • The implications of our guidance on equity and distributive justice

14. Industry as a partner • Engagement in development of methods and processes • Topic selection and scoping workshops • Submission of evidence – increasingly reliant on industry reviews for new products • Expert testimonies by professionals • Consultation • Appeal and judicial challenge

15. developed and provided by NICE • Technology information, with one record per indication, including mode of action, route of administration, formulation, dose, BNF class, likely comparators and whether the product has been selected for NICE review. • Clinical Trial information, with one record per study, including patient population, study design, primary objectives and outcomes. • Regulatory information such as status, date of submission, estimated license date, estimated UK availability. • Costs and budget impact, including proposed average dose, estimated length of treatment, drug cost range per patient per year/per episode, budget impact.

16. Not always easy!

17. But parliament support is key “We conclude that NICE does a vital job in difficult circumstances. The development of more and more health technologies and procedures, alongside rising patient expectations and the ageing population, is going to make it even more difficult in the future. Healthcare budgets in England, as in other countries, are limited. Patients cannot expect to receive every possible treatment. NICE requires the backing of the Government. NICE must not be left to fight a lone battle to support cost- and clinical effectiveness in the NHS.” Jan 2008 – UK Parliament – Health Select Committee

18. On the Committee Day Manufacturers Observers Members Experts Members Members Public Gallery Screen ERG Lead Team Chair NICE ERG = Evidence Review Group 45 - 55 participants

19. Public Recruitment Process for Decision-Making Committees

20. Managing Vested Interests: Code of Practice for Declaring Interests (NICE 2007) • Applies to: • NICE employees, NICE Chairman & non-executive board members and their families • Chairs and members of the advisory bodies to NICE • Expert advisors testifying • Employees of organisations contracted by NICE (including academic and professional associations)

21. Is there a personal pecuniary interest? A personal pecuniary interest involves a current personal payment, which may either relate to the manufacturer or owner of a product or service being evaluated. Example: Any consultancy, directorship, position in or work for a healthcare industry that attracts regular or occasional payments in cash or in kind, both those which have been undertaken in the 12 months preceding the meeting at which the declaration is made and which are planned but have not taken place.

22. Grounds for Appeal • Ground one: The Institute has failed to act fairly • breach of NICE’s own process – reference to NICE process manual respective paragraph • not related to content • appellants need to show they took steps to address unfairness at the time this arose • Ground two: The Institute has formulated guidance which cannot reasonably be justified in the light of the evidence submitted • need to prove that the guidance is obviously and unarguably wrong, illogical, or so absurd that a reasonable Appraisal Committee could not have reached such conclusions • Ground three: The Institute has exceeded its powers

23. Right to Appeal • Patients and Carers: National groups representing patient and carers • Professionals: Healthcare professional organisations (Colleges and Associations) • Industry: Manufacturer(s) or sponsor(s) of the technology • Government: The Department of Health and the Welsh Assembly Government • Payers: Specialised commissioning groups, primary care trusts and local health boards

24. Appeals’ Panel • Non-executive NICE directors incl. vice-chair of NICE (chair of Appeals Panel) (x2) • NHS representative (x1) • Industry expert (x1) • Lay member (x1) • + NICE’s legal advisor

25. Appeals matter! • Appeals are genuinely helpful in improving the final guidance; getting stakeholder buy-in and reducing legal challenge • Half of NICE’s decisions get appealed and half of the appeals get upheld, but... • “NICE’s decision to issue the final guidance may be challenged by applying to the High Court for permission for a judicial review. Any such application must be made within 3 months of publishing the final guidance.” NICE Guide to Appeals, Aug 2010

26. An appeal case study: Azacitidine for the treatment of myelodysplastic syndromes

27. The Appellants • Celgene Ltd; • The MDS UK Patient Support Group (MDS UK), The Leukaemia Society, and the Rarer Cancers Society; • The Royal College of Pathologists and the British Society for Haematology; • The Royal College of Physicians, the National Cancer Research Institute Haematological Oncology Clinical Studies Group, the Royal College of Radiologists, the Association of Clinical Pathologists, and the Joint Collegiate Council for Oncology

28. Ground 2: perversity • “…Omitting chemotherapy as a comparator was perverse, since it is in widespread use. The Committee had accepted that the treatments in AZA-001 trial were ‘broadly representative’ of practice in the UK. In that trial, 41% were allocated to…chemotherapy groups. The Company had, in addition, provided data from a survey of leading haematologists, showing that chemotherapy was commonly used. The Committee took note of the survey, to the extent that they interpreted it to show that practice varied from centre to centre.” Celgene QC • Point Upheld • “The Appeal Panel requests that the Appraisal Committee reappraise azacitidine, ensuring that the comparators take into account both best supportive care and low-dose chemotherapy. If after reconsideration the Committee still considers that only best supportive care should form the basis of its recommendations, it should provide clear and detailed reasons for doing so.”

29. Ground 3: breach of NICE’s powers • “…the guidance contravened NICE's duties under the Human Rights Act. Specifically, they argued that the failure to recommend a life-extending treatment such as azacitidine contravened Articles 2, 3 and 8 of the European Convention on Human Rights, as well as amounting to discrimination in the enjoyment of those rights contrary to Article 14.” Celgene and MDS Patient Support Group, QCs • Article 2, the right to life • Article 3, the prohibition of torture, or inhuman or degrading treatment • Article 8, the right to respect for private and family life • Article 14, the prohibition on discrimination in the enjoyment of a convention right

30. Art 2: Right to Life • Negative obligation: “Art 2 may arguably require the state to make available an overall health service in some form, achieving at least a minimal level of healthcare benefit. The UK government by way of the NHS does provide such a health service.” NICE QC • Point Dismissed • “Even allowing that not every condition is so serious, and not every treatment so beneficial, as to engage either Article, very many conditions and treatments would engage these rights, and the ability of any signatory state to manage its public healthcare budget would seem to be gravely undermined if the appellant's arguments were correct. Not only would this be impractical, it would seem to remove an essentially political or policy decision about the priorities for public spending into the judicial arena, which seems incorrect in principle. No case decided to date seems to imply that this is the law.” Appeals Panel

31. Thank you!kalipso.chalkidou@nice.org.uk