Regulatory Access Pathways to Facilitate Early Access, HTA Synergies

Manon Cappadoro
Leader of Tomorrow Contributor

any patients with severe illnesses still have no treatment today. Disparities in regulations and timelines have proven to be a real obstacle to the access to medicines in the European Union. To facilitate the generation of robust and relevant evidence to support earlier decision making and access to medicines in Europe, specific regulatory tools have been established by the EMA, including:

• PRIME: The PRIority MEdicines scheme aims to foster the development of medicines with major public health interest and should be considered in case of very promising exploratory data results. Benefits include scientific and regulatory advice at key development milestones, optimization of development for robust data generation, and the possibility to receive accelerated assessment of the Marketing Authorization Application.
• Scientific Advice that can be requested from the EMA at any point in product development, plus the additional possibility of consultation with Health Technology Assessment (HTA) bodies and/or with US FDA.
• Conditional Marketing Authorization: Authorization before comprehensive data are available, when benefits of early access outweigh its risks, in order to address unmet medical needs.

Experience with these early access pathway shows that the main challenge is to align discrepant evidence needs between regulatory and HTA bodies. Collaboration across these and other healthcare decision makers is increasingly needed to facilitate patient access to innovative new products. One such collaboration, between EMA and EUnetHTA, has enabled an optimized evidence generation plan and has proven critical to improve access for patients.

It is also important to note that these early access pathways do not necessarily lead to faster patient access. Even after market authorization is granted for a product through the centralized procedure, pricing and reimbursement must still be evaluated at the national level. This continues to present a barrier to access that has been increasingly difficult to cross. It is hoped that better alignment among regulators, HTAs, payers and sponsors will gradually eliminate this critical barrier.