One interesting session at last week’s ISPOR annual European congress in Milan focused on the provision of parallel scientific advice by regulatory agencies and HTA agencies. Some of the insights from the panel discussion and ensuing Q&A session are worth sharing.
What is parallel advice?
The discussion in this session focused on seeking parallel advice – which means advice provided at the same time, at the same meeting – by a drug approval agency (for example, the EMA) and one or more HTA agencies. The idea of receiving parallel advice is that the pharmaceutical company could simultaneously benefit from the advice of the regulator and the HTA body on how to set its clinical trials for a new medicine so it can adequately address the needs of both. Historically, EMA provides scientific advice sessions – and the advice provided at those is binding. In contrast, HTA agencies can also provide advice – as does the UK’s NICE under its scientific advice programme – however, that advice is not binding for the manufacturers. In principle, obtaining both sets of advice at the same time should be beneficial.
Divergent views on the need for parallel advice
Indeed this is the view shared by the industry representative on the panel: bringing the regulator and HTA decision makers together can help optimise development resources, which would in turn serve the needs of patients. From the perspective of an HTA agency representative, the picture is somewhat more complicated. EMA and the US FDA are powerful organisations. “Do we want them to dictate to a small country’s HTA agency?” he asked. The issue is complicated further by the aim of regulators to approve drugs for marketing quicker under new mechanisms such as the Breakthrough Designation in the United States and the Adaptive Licensing procedure in Europe. As regulatory approval is granted earlier, it tends to be based on more limited clinical data. It is a tradeoff in terms of market access unless the company can generate additional clinical data. “There is a benefit to the US population from early access, but there is a cost to the German population if IQWiG does not see the data as sufficient,” the HTA representative said – it should not be a question of having to choose which set of data the manufacturer should generate, as in some cases both the data the regulator would have wanted and the data the HTA agency would have wanted are needed.
A case of “be careful what you wish for!”
One piece of advice for pharmaceutical companies that came out of the Q&A session is that if a company asks for parallel advice it should then follow through and incorporate that advice into its clinical programme and not ignore it. Another thing to keep in mind is that scheduling of parallel advice meetings is increasingly challenging as more companies are realising the benefits and asking for such meetings. The resources of EMA and HTA agencies are limited, and thus it is best to seek parallel advice in cases when the drugs in development bring about a true therapeutic innovation and not just a minor improvement over existing therapies. And small and medium-sized pharmaceutical companies should expect that their larger counterparts will be also lining up to receive parallel advice in many cases. Even if they have a lot of experience with HTA, the industry would also benefit from parallel advice in cases when the development work is in a therapeutic area they do not have much HTA experience in and also in cases when having early parallel advice would give them a view to the future and a sense of what they should expect the HTA agency to ask for when they apply for reimbursement.
And finally, they should keep in mind that having several HTA agencies at the same meeting – e.g., UK’s NICE, Germany’s IQWiG and France’s Transparency Commission – does not mean that simultaneous advice means uniform advice. The different HTA agencies can still insist on data that fit their national treatment guidelines and not be swayed by what other HTA agency representatives are asking for. Even if quasi-consensus is reached at the parallel scientific advice meeting, the company should still be prepared to engage early with the national level HTA agency in the country where it is planning to seek reimbursement as a follow-up to the early parallel advice discussion.
Milena Izmirlieva is head of the research team at IHS Life Sciences.
Posted 13 November 2015