Convergence and difference in HTA approaches in UK, Germany and France: reflections on recent and proposed changes

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1 Convergence and difference in HTA approaches in UK, Germany and France: reflections on recent and proposed changes Professor Ron Akehurst School of Health and Related Research

2 Content of Talk During this talk I will try to do two things: 1) explain how HTA works in the UK and the way that it may change with the introduction of Value Based Pricing 2) reflect on the similarities there are between France, Germany and UK despite some continuing differences

3 Commissioning In the UK Clinical Commissioning Groups (CCGs) commission (or buy) most services in the NHS, with exception of some specialised services; CCGs commission: Planned hospital care Urgent and emergency care Rehabilitation care Community health services

4 Provision of Care General Practitioners (GPs) are funded by a mixture of capitation payments and fees for performance (e.g. for vaccination or screening); they have to decide how to provide care within the funds, staff and time they have available Hospitals are funded by Commissioners to provide agreed mixes and levels of activity and may lose funding if they fail to deliver agreed levels or quality of service); they have to decide how to provide the care commissioned

5 Advice and Direction In making their decisions both Commissioners and Providers receive a mixture of advice, guidance and mandatory direction The direction is usually from NHS England and the Department of Health, though not exclusively, while most of the advice and guidance comes from NICE in England and Wales. NICE decisions on drugs have a mandatory force Scotland and Northern Ireland have their own devolved arrangements although NICE advice may also be influential there. I am concentrating my description on NICE activities

6 What is NICE? NICE, The National Institute for Health and Care Excellence, is a Special Health Authority created in 1999 by direction of the Secretary of State for Health for the purpose of providing advice to the NHS and now established as a statutory body under primary legislation It provides advice on Public Health interventions; interventional procedures; clinical guidelines and health technologies, including devices, diagnostics and pharmaceuticals Importantly it has a growing implementation role

7 Health Technology Assessment (HTA) NICE has separate processes for assessing the value of medical devices, diagnostics and drugs to the NHS (with separate consideration of Highly Specialised Technologies) All processes use a common HTA methodology

8 Drug Pricing Currently, manufacturers can set what price they wish for new drugs but their operation in the UK is subject to an upper limit on their total profits. They may only increase prices if profits fall below a lower limit and with permission from the DoH, under a scheme known as the Pharmaceutical Price Regulation Scheme (PPRS) At the moment NICE has no formal role in drug pricing. These arrangements change in Jan 2014 when Value Based Pricing, operated through NICE, will come into effect and a price will have to be agreed between the manufacturer and NICE and ratified by DoH

9 NICE Approval New drugs (as well as many other technologies) are reviewed by NICE around the time they are launched in the UK NICE has to advise whether drugs should be used in the NHS, given their price; in which patients and in what circumstances (e.g. only after failures of other, specified treatments and only until defined signs appear) Decision is therefore Yes/No and in whom Recently drug and device manufacturers have negotiated Patient Access Schemes which have had the effect of lowering price as a means of getting a favourable decision; the intention is that from 2014 there will be a Value Based Pricing scheme introduced, based on HTA by NICE, through which prices will be negotiated with companies

10 Process of Selecting Topics A high level committee (MTAC) looks at all new drugs and at topics suggested by any of NHS bodies, professional bodies (e.g.rcp) patient groups or manufacturers MTAC may make a decision on the technology if evaluation is relatively simple (as in the case of some devices), decide that it should not be evaluated at all or refer it to a relevant NICE committee (TA, Diagnostics, Public Health, HST or Guidelines) for evaluation All new drugs of any significance are referred to the TA Committee or (for drugs for very rare conditions) to HST

11 Scoping and definition of decision problem For all forms of technology appraisal much effort is put into defining the problem to be addressed through an initial scoping document produced by NICE and subsequent rounds of meeting and consultation with interested parties Highly consultative process with all stakeholders manufacturer of drug under review, manufacturers of comparator products, clinical groups, PCTs, hospitals and patient groups Starting point is current treatment Great efforts made to define relevant pathways of care and to assess where the new technology will fit within them This defines comparators, patient populations and the evidence which is desirable to inform a decision

12 Finding Evidence Once the evidence that is needed is clear it has to be found, extracted and synthesised in an agreed manner This has to be carried out in a systematic way according to NICE guidance Searching for other than RCT evidence and all aspects of conducting synthesis are areas where methods are developing quite quickly

13 Principles of Evaluation and Opportunity Cost At the core of the ethical principles guiding evaluation of the provision of all services and technologies in the NHS is the fact that the total resources available for the provision of health care are, within fairly narrow tolerances, fixed. The view is taken that nothing should be introduced into the health care system, however effective, if the value of the care it displaces is greater than the value of the care it brings. By capturing evidence on the costs of the technology we provide a bridge to the benefits foregone in using it. The more it costs the more we forego and the greater the benefits have to be to make it worthwhile to introduce it The economist s concept of Opportunity Cost underlies the NICE Threshold, an estimate of the value of benefits lost when the resources for a new intervention are removed from their best alternative use Much effort has been put in the last two years to estimate the threshold

14 Economic Evaluation Drug, device and diagnostic manufacturers have to present a clinical and economic case to NICE. They are asked to conform to a set way of presenting their economic arguments, known as the reference case. This ensures that all appraisals are handled in the same way NICE staff and advisors also have to conform to the reference case in their analyses

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16 Perspective on Costs and Benefits NICE currently takes into account costs and benefits that fall on the health service; benefits or costs from changes in production are only captured if they improve the individual s health related quality of life but not otherwise. This will likely change. Unlike now it is likely that both carer costs and benefits and net productivity effects will be counted in future for both index drug and comparator; this likely to be in a fixed manner depending on therapy area Process benefits such as greater convenience to patients are not currently directly counted in the base case. This is unlikely to change. Possible other benefits, for example from reduced crime resulting from detoxification treatment of illegal drug users, are currently only noted for consideration in the final decision rather than built into the formal process of estimating cost effectiveness. This is also unlikely to change

17 Valuing Benefits Knowing the results of clinical studies is not enough on it own to make it clear that a technology is good value for money For example, trial outcomes might be in the form of percentage of treated population that achieved a given target, say phosphate levels in End Stage Renal Disease patients How should that be valued? At NICE there is a process of calculating what the implications of the clinical trial results would be when applied in the context in which the treatment would be used. This is in terms of quality of life and survival, using the QALY

18 Quality Adjusted Life Years (QALYs) NICE describes the QALY as follows: The QALY is a measure of a person s length of life weighted by a valuation of their quality of life over that period. The HRQL weighting consists of two elements: the description of the changes in HRQL itself and the valuation of that change in HRQL. Standard instruments for estimating the weighting for Quality of Life, in particular the EQ5D, are preferred, although others such as HUI and SF6D are also used

19 Longer term costs and benefits of the technology: synthesis and modelling Often trials do not provide evidence about the relative effectiveness of the new technology against all (or even any) relevant comparators, meaning that further sources of evidence have to be sought NICE expects that getting to a decision rather than describing the evidence will take the use of a great deal of evidence from a variety of sources many of which would be non trial These considerations have lead to rapid growth in both evidence synthesis and modelling techniques Given the comparative nature of cost effectiveness studies, it is often the case that much if not most of the evidence required is on comparators. Thus NICE requires that this evidence be collected as systematically and comprehensively as on the index technology

20 Appraisal Committees Decisions are made by Appraisal Committees. The Committees are made up of independent minded volunteers who are unpaid, except the Chair Membership contains a mixture of physicians, HTA specialists, NHS managers and patient representatives. Topic expert clinicians are invited to Committee meetings Most of the Committee business is conducted in public

21 Decision Process Committees have to come to a decision, informed by the manufacturers submission as critiqued by Evidence Review Groups (ERGs) and other commentators, expert opinion and patient views. This is followed by further comment from the manufacturer and other stakeholders after the initial evaluation, and the evidence on which it has based, have been placed on the Institute website. In reaching their decision their first consideration is likely to be how important is the clinical innovation being introduced; other important considerations are likely to be whether the patient population in which the evidence was collected was relevant to the UK, whether the evidence of effect against relevant comparators is convincing; the QALY value and the uncertainty around the estimate Ultimately, the Committee has to decide on whether the technology is good value for money for the NHS, with the cost/qaly estimate being one of the pieces of evidence it takes into account (albeit a very influential one)

22 Implications of NICE decisions Negative decisions are implemented comprehensively Positive decisions have to be actively implemented by CCG s, GPs and Hospitals CCG s are required to act on NICE recommendations in England After VBP is introduced, the Appraisal will be the basis for price negotiations where NICE will be trying to ensure that price is set to give a cost/qaly below the Threshold this is not too different from what happens now with PAS We may see common use of initial prices which may be revised or supported following further collection of evidence, as we see in France If a price cannot be agreed, NICE will issue a not recommended decision

23 France, Germany and the UK different? In looking at HTA processes in these three countries it has been commonly thought that they are very different. The UK has been alone in a number of respects: it has been very explicit about the process by which a cost effectiveness case should be made; has sought a single metric for measuring cost effectiveness in the QALY; has adopted much formal modelling of CE and has set an explicit cost/qaly threshold for decisions. Post launch evidence has not been generally sought as part of planned reviews of prices in the UK Until recently, CEA had little role in France and Germany and the use of the QALY was rejected; in France, unlike in the UK, price negotiation was explicit and post marketing collection of evidence important for prices over time.

24 . or becoming more similar? However, there were always great similarities: Although the British have not used an ASMR system, the significance of an advance has always mattered to them; the issues of relevance of evidence locally, assessment of its quality and its synthesis have always mattered in all three systems and were the most likely hurdles at which drugs would fail And there is convergence in processes: the British are adopting negotiation approaches like the French and intend to look at post launch evidence in reviews of price; France has introduced CEA, considering cost/qaly, for drugs which achieve an ASMR of 3 or better and Germany has adopted strict evaluation processes at IQWIG which have much in common with those of NICE and which start from an evaluation of the importance of an advance which is similar to assessing an ASMR in France; techniques such as indirect comparison of RCT evidence and decision modelling, once accepted in the UK but rejected elsewhere, have been taken up in Germany and France Further, the more one talks to those who sit on Committees that make the final decisions the more it is clear that what drives those decisions is often very similar

25 Thank You for your Attention