What could gene therapies learn from orphan drugs’ post-regulatory approval access in the EU?

ABSTRACT

Introduction: Gene therapy medicinal products (GTMPs) have the potential to dramatically change the therapeutic paradigm in various conditions. However, these therapies do come at significant costs and are expected to produce substantial budget offsets.

Areas covered: This article aims to explore the rationale of post-regulatory approval access to GTMPs in the EU. An analytical framework was developed to structure the review around three underlying issues – clinical effectiveness and safety, societal preferences, coverage modalities. Finally, parallels were drawn between GTMPs and orphan drugs.

Expert opinion: Post-regulatory approval access to GTMPs in the EU is now in a situation, where orphan drugs used to be ten years ago. Back then, the main challenge was not whether to prioritize rarity, but to create mechanisms for assessment and appraisal of orphan drugs’ value. The only reasonable way to accept a higher valuation of GTMPs is through real-world evidence. Companies need to establish patient access mechanisms in order to demonstrate a value for money. Patient registries and centers of expertise could complement this process. Such experience and expertise are already available in the field of rare diseases and orphan drugs. This could be a real head start for GTMPs’ post-regulatory approval access model.

KEYWORDS:

• Access
• coverage
• gene therapies
• health technology assessment
• orphan drugs
• rare diseases
• value

Article highlights

• Withdrawal of several authorized advanced therapy medicinal products (including one GTMP) in the EU highlights the specific settings of the post-regulatory approval access decision-making.

• Assessment of clinical effectiveness and safety of GTMPs is a central challenge. Monitoring of treatment response and identification of long-term risks would require continuous resources and efforts.

• Incorporation of patients’ and society’s preferences is essential to measure the added value of GTMPs, including their impact on families and caregivers, health infrastructure and innovation.

• GTMPs are a perfect candidate for performance-based coverage agreements. Amortization mechanisms could be added, so payments could correspond to the gradual realization of health benefits.

• Coverage decision-making for GTMPs could greatly benefit from the accumulated experience and expertise in the field of rare diseases and orphan drugs in the EU, with patient registries and centers of expertise being of paramount importance.

This box summarizes key points contained in the article.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was supported by Ministry of Education and Science, Republic of Bulgaria (National Program „Young scientists and postdoctoral students”). Responsibility for the information and views set out in this publication lies entirely with the authors.