Globally applicable facilitated regulatory pathways to improve equitable access to medicines

Abstract

Equitable access to medicines is a right of all patients and they may have great expectations of rapid and efficient regulatory processes that contribute to accelerated access to safe and effective innovative new medicines. A variety of approaches have been developed to accelerate the regulatory review of medicines. We characterise these expedited pathways as facilitated regulatory pathways (FRPs): designed to accelerate product development, the submission of market authorisation applications, and regulatory reviews. The goal of FRPs is to speed the assessment of new drugs often for serious diseases or where there is an unmet medical need. But FRPs may be applicable to a broader group of products, including generics, biologics and vaccines. In spite of the on-going trend towards global regulatory convergence, no internationally relevant guidelines or best practices have been promulgated that describe the elements or conditions needed to implement an accelerated regulatory review pathway. The diversity of FRPs found across high-, middle- and low-income countries creates confusion for stakeholders, with uncertainty about the accelerated review requirements and processes across jurisdictions; this results in patients questioning the timing or divergences in access to important medicines. Therefore, this research was conducted to identify and characterise the key building blocks that provide context and support for the efficient use of FRPs. We hypothesised that through the methodical assessment of four key themes (stakeholder support and the regulatory environment; processes that contribute to predictability in regulatory decision making; use and interpretation of evidence associated with regulatory outcomes; post-authorisation assessments) we would be able to characterise a globally applicable pragmatic framework for the use of a diverse set of currently available FRPs. This thesis presents our observations that support our proposal for a globally applicable approach to using FRPs. Using the findings from our specific building block research and developed a 4-step pragmatic framework approach designed to help agencies of all maturity levels determine how best to address the use of FRPs. Step 1 assesses four domains of the environment preparedness, Step 2 offers process criteria that should be in place to effectively use an FRP, Step 3 tiers agencies through a self-assessment of readiness and capacity, and Step 4 provides a pathway for agencies to determine the most relevant FRP for their use. This framework represents the first endeavour to holistically address the multifaceted aspects that should be considered for the effective use of an FRP. It offers process transparency to address the needs of sponsors and suggests timelines that address the practical considerations of sponsors and agencies and the expectations of patients. By providing supportive evidence for the building blocks of FRP best practices, we believe these approaches can form the basis for aligning the wide variety of review programmes that are in place or that are being developed for the accelerated assessment of important medicines. We trust that the work presented here can serve as the basis for international policies for efficient medicines reviews that can contribute to the equitable access to medicines worldwide.