Another issue of concern is the relationship between relative assessment of value, reimbursement, and the marketing authorization
process. You suggest that the process has been complicated by the growing demands of payers and health technology assessment
agencies for the same kind of evidence. This creates more bureaucracy and inhibits your mandate to speed innovation.
TL: Expectations around the R&D industry are very high today. Payers—and patients—want new treatments that represent a therapeutic
advance, but they don't wish to go bankrupt in paying for them. Yet the reality is that the industry is suffering from low
productivity, and the prices charged for products we do approve are quite high. The reality for us as regulators is that we
no longer have discretion in deciding whether patients have access to the medicines we approve. Our external stakeholders
are noting this discrepancy. It runs counter to our mandate, which is to promote the supply of safe and effective medicines
to all citizens in Europe.
Hence there is potential for confusion, ultimately leading to not one but two drug development programs, which will only increase
the cost and probably slow industry productivity even further.
What we are proposing is a twofold approach to limit the possibility that this will become a bureaucratic reality, not just
in Europe but elsewhere. First, we want to work together with Health Technology Assessment (HTA) agencies to refine the information
we produce and the evidence we require—on efficacy and safety against agreed endpoints—so that it can be extended to help
determine relative effectiveness against drugs and other healthcare interventions. One instrument is the European Public Assessment
Report (EPAR) we produce as part of the licensing evaluation. The goal is to adapt that document to better suit the information
needs of the national reimbursement and HTA agencies. A second instrument here is parallel scientific advice. A pilot project
is now under way with a few R&D companies to examine how this might work in practice, with particular emphasis on what kind
of data and information our two groups—regulators and HTA bodies—would need for their assessments.
The second plank in our proposal is to cooperate on the execution of post-authorization studies linked to risk management
plans. There is room to pool our resources to generate strong observational data and determine how a medicine is actually
being used in a patient population. Such evidence is highly relevant to the value determination that reimbursement authorities
and HTA bodies wish to make before granting access to patients. I note that under the new EU pharmaceutical legislation, the
defining principle of risk management has been widened to incorporate benefit, in addition to risk. This provides a better
measure to calculate value, at least from the clinical side.
Finally, the Agency has to take into account the political aspects of the discussion. DG-Sanco is interested in building a
common European terminology for HTA. There is now a Commission-financed network of national HTA agencies that some experts
believe might lead to a single point of access to establish whether the medicines we approve are actually worth paying for—in
all 27 member states. We are not advocating for this. Personally, I see it as taking place many years in the future, if at