By Ansis Helmanis
Ethics is a matter of trust, which in the regulatory space requires transparency and openness among all stakeholders, including patients and industry. This was the consensus of FDA Commissioner Margaret Hamburg and European Medicines Agency (EMA) Executive Director Guido Rasi, both of whom shared the podium with Nobel Laureate Professor Elie Wiesel at a plenary lunch panel on “Ethics of Innovation” at Tuesday’s Prix Galien Forum in New York. Rasi also emphasized the need for increased communication and collaboration within a regulatory community that is now global in scope and reach. “We must be more than just the sum of our parts.”
The regulators also identified certainty and predictability in the regulatory process as critical factors in fostering new medicines innovation, the more so for those companies that are seeking to provide complex biologic therapies for unmet medical needs, where agency guidance is often needed on how to proceed. Hamburg sees the role of FDA as a “gateway” to innovation in speeding medicines access for patients with few available treatments for their conditions. But to do so she underscored that regulatory agencies need to develop more tools to clarify scientific uncertainty as new technologies advance our knowledge of disease. Rasi added that in the European Union a key issue is the question of what is innovation, given that EMA has to act on the basis of collaboration with 27 member states, each with their own perspective.
Prompted by panel moderator William Looney of Pharm Exec, Hamburg and Rasi also addressed the current environment where patient access to medicines is in practice being defined by reimbursement authorities rather than through traditional registration approval. Both agency heads stressed that neither has a legal mandate to include reimbursement or cost effectiveness in their review and approval determinations. However, they did see a role for their respective agencies in consulting with cost containment entities – such as the Centers for Medicare and Medicaid (CMS) in the US and national health technology assessment institutions in Europe – on the safety and efficacy factors that led to the initial marketing authorization decision.
Bringing CMS into the FDA’s pre-approval drug review process on a trial basis is in part intended to facilitate CMS reimbursement decision-making by getting it into gear earlier, according to Hamburg. Rasi noted that in Europe, the EMA is engaged in a similar exercise bringing together, well prior to marketing approval, the drug sponsor and representatives from the major reimbursement agencies to facilitate what amounts to a parallel process of review. In addition, EMA has expanded the information that is included in the European Public Assessment Report (EPAR) as a way to assist national reimbursement decisions. The goal is to avoid situations where the EMA and local reimbursement agencies might be asking for the same information, which can strain the innovator’s resources and end up delaying access.
The moderator also noted the impact on FDA of a mandatory budget sequestration if there is no progress on avoiding the year-end tax and fiscal cliff. Hamburg expressed optimism on an eventual resolution of the impasse, but added the stakes were as high as they could be: sequestration could end up barring introduction of the new user fees that are critical to FDA’s ability to fulfill its mission in advancing innovation.