Approve, Deny or Experiment: Innovations in Regulatory Science that Balance Risk and Benefit

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Background

Health regulatory and reimbursement systems have been challenged by therapeutic products and devices that do not easily fit within existing laws, regulations, policies, and practices, such as precision medicines, drugs for rare diseases, and advanced therapeutic products. The blockbuster model no longer dominates the drug development landscape; instead, many new health technologies target smaller patient populations. Sponsors of these health technologies may not be able to meet the clinical evidentiary thresholds on safety and efficacy that are typically expected by regulators and reimbursement decision-makers. In many clinical areas with unmet medical needs, such as oncology, rare diseases, and emergent infectious diseases, waiting for sufficient evidence to be generated pre-market may be outweighed by the potential benefits of earlier patient access.

In response, regulators have been shifting regulatory market authorization and reimbursement decision paradigms. Many regulators have introduced conditional or accelerated regulatory pathways that grant market authorization based on earlier clinical evidence, typically on the condition that additional evidence will be collected post-market to confirm clinical benefit. Additionally, regulators are beginning to introduce regulatory pathways specific to advanced therapeutic products. These products are so unique they cannot be regulated under existing pathways, and regulators are experimenting with novel approaches such as regulatory sandboxes. As reimbursement decisions are typically made separate from and following regulatory authorization, reimbursement paradigms have similarly had to adjust to changing industry and regulatory practices and trends. Rather than binary, fund, or don’t fund decision-making, many payers are experimenting with alternative reimbursement models, such as conditional reimbursement schemes and managed access agreements that provisionally fund new technologies, with long-term funding contingent on additional evidence confirming clinical and cost-effectiveness.

In this Research Topic, we draw on the experience of different jurisdictions to explore issues, challenges, experiences, and recommendations for implementing conditional regulatory and reimbursement frameworks to enable patient access to innovative health technologies.

We welcome articles analyzing or discussing regulatory and reimbursement experiences and/or reforms related to conditional approval/reimbursement mechanisms and pathways. Articles may be empirical, analyses, or commentaries. Authors will be invited to an international workshop to discuss these topics and a resulting discussion article will be one of the submitted manuscripts in the Research Topic.

Keywords: conditional regulatory pathways, accelerated regulatory pathways, managed access agreements, conditional reimbursement, expensive drugs for rare diseases, advanced therapeutic products, regulatory sandbox, clinical evidentiary thresholds

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

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