Social participation for sustainable and legitimate prioritisation processes: NICE’s experience

Santiago, October 2010Sir Michael Rawlins, Chairman, NICEKalipso Chalkidou, Director, NICE International

The NHS in 2010UK parliament

NHS vote every 2 years

Secretary of State for Health/Department of Health

Primary Care Trusts

Other bodies (e.g. Care Quality Commission)

NICE

10 care doctors

Hospitals

152 regions across the country

Tax-revenue allocation by government with limited co-

pays and out-of-pocket

guidance

Hospitaldoctors

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

NICE’s features

Our objectives• Independence from

government, industry and professionals

• Transparency• Inclusiveness and public

consultation• Scientific basis for

decisions

Our principles

• Reduce inappropriate geographical variation

• Set quality standards for the practice of medicine

• Encourage innovation in health technologies

• Promote efficiency in resource allocation

Centre for Public Health Excellence

- public health interventions- public health programmes

Developing NICE guidance

Centre forHealth Technology

Evaluation- technology appraisals

- surgery- diagnostics

- medical devices

Centre for Clinical Practice

- clinical guidelines- quality standards

- QOF

NICE structure

Communications

Patient and public

involvement

Implementation

R&D

Topic selection

Information systems

NHS Evidence

Stakeholders

Patients and Citizens

Professionals Industry

Payers and Providers

Popular Media

ParliamentPolitical Parties

Ministries of Public

Health and of Finance

EU and UK

legislators

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…

Stakeholder input

* Published on NICE web site

Topic referred to NICE*

Draft Guideline Public Consultation*

Scoping*

Dissemination and Implementation

Publication*

Development

Independent Review of response to public comment*

appeal

legal challenge

Working with the country’s professionals: the National

Collaborating Centres

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

Communicating with patients

• 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

“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”.

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.

Assessing value is context-specific

• 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 justiceRawlins, Lancet Oncology, 2007

NICE’s Citizens’ Council

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

• 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.

developed and provided by NICE

Not always easy!

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

On the Committee Day

ERG

Mem

bers

Experts Members Members

Lead Team NICE Chair

ERG = Evidence Review Group

Manufacturers

Public G

alleryS

cree

n

Observers

45 - 55 participants

Public Recruitment Process for Decision-Making Committees

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)

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.

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

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

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

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

AN APPEAL CASE STUDY: AZACITIDINE FOR THE TREATMENT OF MYELODYSPLASTIC SYNDROMES

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

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.”

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

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 QCPoint 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

Thank you!

kalipso.chalkidou@nice.org.uk