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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 (http://www.lshtm.ac.uk/hsru/vpdt/). 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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