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The Official News & Technical Journal Of The International Society For Pharmacoeconomics And Outcomes Research


Kalipso Chalkidou MD, Associate Director, Research and Development, National Institute for Health and Clinical Excellence (NICE), London, UK

Conditional Reimbursement Based on Future Research

The following was presented during the Third Plenary Session, “Conditional Reimbursement Based On Future Research,” at the ISPOR 10th Annual European Congress, 23 October 2007, Dublin, Ireland.

The option of recommending the use of a technology in the context of research or data collection has been part of NICE's statutory rights from the very beginning. In addition to the yes and no type decisions, NICE has always had the option of recommending further research particularly for promising interventions where the evidence base is weak; “only in research” OIR recommendations indicate the questions that the research should be addressing and advise clinicians that in the meantime the technologies should be used in the context of this research. Of course, it is a very challenging decision option to implement. There are important questions around who pays for this research: will it be public or private funding or mixed? What happens in the meantime, while this research or data collection is ongoing? That depends again on the type of the research, the extent to which patient access is restricted the type of uncertainty to be addressed. And finally what are the mechanisms to then use the research results to inform updates of the guidance?

We can explore some of these issues through examples of previous NICE decisions. In the case of photodynamic therapy NICE recommended the use of the technology only in the context of research. NHS R&D funded a prospective cohort study that is still ongoing; the study's findings will inform the next NICE update. Setting the study up has not been without challenges, however it is now up and running; the London School of Hygiene has set up an interesting website on this study that addresses some of the issues of OIR in the context of NICE ( This is a study with public funding in terms of participation and access. Almost all patients that are eligible to get this intervention do get it across the country in those centers that participate and almost every center is part of this work. As an example of private funding, NICE issued an OIR recommendation in 2002 for the use of CCBT technologies only in the context of research. Since then the guidance has been updated: for two of the technologies, “Beating the Blues” and “Fear Fighter”, additional RCT evidence was submitted by the manufacturers and NICE recommended that these technologies are reimbursed in the context of the NHS. For two more technologies, NICE recommended that more information is needed and this only in research recommendations still holds because of the existing uncertainty. To give an overview of what we have been doing so far, about one in 20 NICE's technology appraisal recommendations have been only in research. Almost half of recommendations around the use of surgical procedures have similarly been OIR. Finally, in NICE clinical and public health guidance, where there is a lot of uncertainty mainly around established and well-diffused interventions that may require the OIR option has been used extensively by our decision makers.

Risk sharing could well be thought of as another way of collecting the information to inform definitive decisions on coverage and multiple sclerosis is an example of such a scheme that was set up by the Department of Health and it is still ongoing. Most recently, the NICE decision on Velcade for multiple myeloma, it is an interesting example of risk sharing where manufacturers and decision makers agreed that the drug could be reimbursed as long as more evidence was collected and the evidence was used to inform reimbursement rates on a case-by-case basis. This evidence will also be used to inform the NICE update in three years time.

“Where sufficient information at the time of launch was not available to take an informed view…risk sharing arrangements can help coordinate the expectations of the payer and manufacturers…allow for more predictable uptake for manufacturers, and predictable health gains for a given expenditure [when] an agreement may not be able to be reached otherwise.”

– OFT Report, February 2007

We have so far been talking a lot about costs: some people have argued that NICE only cares about the cost per QALY and that the OIR option may be another way for restricting access to treatment to save money. This is far from true; NICE involves patients and the public in developing individual recommendations and, with the help of its Citizens Council, it involves the public in shaping its general social value principles that inform the broader decision making process. In this context we asked the Citizens' Council what they thought of the OIR option, of recommending a technology only in the context of research when we are not certain about its effects. The Council concluded that “patients would be reassured to know that clinicians and the system in general can face up to uncertainty and are confident enough to deal with it in a mature and scientific way, avoid wasting resources on unproven technologies.” In their report which can be found on the NICE website, the Citizens' Council has listed a number of key consideration decision makers should take into account before making an OIR recommendation, including whether the uncertainty can be reduced through further research, whether the research is feasible, whether patient access will be compromised during the research. Patient access to the research rather than patient access to the technology was their main concern, as were the timeliness and the value for money of the research.

There are three key conditions for the OIR to work: a) we have to develop and apply consistently clear decision criteria that can guide policy makers assess how much uncertainty is enough to warrant an OIR recommendation; b) the health care system, including professionals, commissioners, researchers, patients and industry have to work towards developing the right frameworks for implementing OIR recommendation in the real world; and finally c) We have to make sure that the right mechanisms are in place to allow prompt update of the guidance in light of the research evidence. In a way the third condition is the easier to meet in the context of the NICE regular review process. A lot of progress is made with regard to the second condition: we are developing, in the UK, an effective infrastructure through the efforts of the Clinical Research Collaboration, and, in the post-Cooksey world, the Office for Strategic Coordination of Health Research (OSCHR). Furthermore, the potential of Connecting for Health for generating outcomes data to inform OIR recommendations is considerable.

The Cooksey Report on health research in the UK, published in December 2006, recommended that NICE and NHS R&D work more closely together and that funding is allocated to direct implementation of NICE recommendations calling on the NHS to use technologies in the context of research. This report was endorsed by Gordon Brown in the pre-budget report and the recent comprehensive spending review report so it will be fully supported financially. In fact there has been an announcement recently for an increase in public HTA funding, for the National Institute of Health Research to support this sort of HTA, real world studies that seek to address decision makers' questions and inform policy and practice. Another fairly recent announcement in the Darzi Report of the Health Innovation Council was $100 million was given to support good value innovation. Reducing decision makers' uncertainty on the effectiveness and value of new and existing technologies the NHS pays for could and should be part of the remit of the Innovation Council and this broader initiative. Finally, the new critical path modeled along the lines of the FDA critical path raised also by Cooksey is another important development where NICE, NIHR and ABPI, and individual manufacturers can work together throughout the licensing process to inform decisions and fill evidence gaps.

NICE has been working closely with NIHR over the past two years with a specific focus on primary research coming out of NICE technology appraisals but also clinical guidelines and public health guidance. We hold regular joint prioritization meetings to look at NICE research recommendations and have set up what we call “direct access” to support specific questions that NICE decision makers have, head to head trials most of the time, which industry might not be willing to fund. The results of such research will directly inform NICE updates and be translated into policy and, hopefully, clinical and public health practice.

One of the biggest challenges facing NICE is how to deal with uncertainty. Should we say 'no' when we do not know? This is a very important question. It is a methodology question and it is a policy question. “yes/no' decisions are no longer adequate to deal with uncertainty in a pragmatic way. All different stakeholders need to come together to develop this third option that allows access while encouraging evidence generation, that promotes innovation while preventing waste and protecting patients from potentially harmful effects of untested treatments. I think it is no longer sustainable to say 'no' when the evidence is weak as it is no longer sustainable to say 'yes' and give technologies the benefit of the doubt particularly within a resource constrained system that is the NHS. Only in research is the third option that can address this dilemma.

On a different note, in the United States, millions of children are going uninsured because of President Bush's veto against a joint Democratic/ Republican proposal by Congress to expand coverage for poor children and their families. The reason: such expansions could lead to socialized medicine and universal coverage, which is what the case is in most European countries today.


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