Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective

ABSTRACT

Introduction

The EU Orphan Regulation has successfully stimulated R&D of medicines for rare diseases, resulting in a substantial increase of orphan designations and authorized orphan medicinal products in the EU during last decade. Despite such advances, access to treatment across the 27 EU Member States is still highly variable.

Areas covered

We provide an overview of the current situation of patient access to orphan drugs in the EU. We discuss the EU policy landscape regarding joint assessment and pricing & reimbursement negotiations of medicinal products, price and sustainability of orphan drugs for health care systems, and the importance of Real-World Data and registry infrastructures for rare diseases. Additionally, we provide recommendations for areas of improvement throughout the lifecycle of orphan drugs, aiming to preserve a positive R&D climate for rare diseases in the EU and accelerate patient access.

Expert opinion

The EU needs to maintain a patient-centric pharmaceutical ecosystem that encourages long-term investments and rewards innovation in areas of high unmet medical need. Areas of potential improvement range from enhanced alignment of regulatory and HTA evidence requirements and use of specific value frameworks for the assessment of orphan drugs to the development of registry infrastructures and innovative performance-based pricing agreements.

KEYWORDS:

• Orphan drugs
• access
• orphan drug regulation
• health technology assessment
• pricing and reimbursement

Article highlights

• Several studies on access to orphan drugs highlight strong variations across EU Member States, despite a centralized Marketing Authorization. Several causes, including different HTA requirements and processes among Member States, have been identified.

• More homogeneous access to orphan drugs can be fostered by establishing pathways for joint assessment and negotiation of orphan drugs among Member States. While the new EU HTA Regulation may introduce some positive changes, the potential for joint negotiations still needs to be fully explored and its added value proven.

• A higher degree of alignment between regulatory and HTA evidence requirements can be achieved through iterative parallel consultations among EMA, HTA bodies, and orphan drug developers.

• Pricing considerations for orphan drugs should build upon a flexible HTA decision-making process and augmented value frameworks able to capture the full impact of rare diseases on patients, their families, and caregivers, as well as health systems, and society as a whole.

• Investing in registry infrastructure across rare diseases will give industry and authorities more insights into the course of diseases, enable faster trial recruitment, facilitate long term follow-up of patients, and create the basis for implementing Managed Entry Agreements.

Declaration of interest

G Tafuri and A Bracco are employed by Apellis Switzerland GmbH, which has a financial interest in orphan drugs. J Grueger is employed by Boston Consulting Group Switzerland and consults with clients in the pharmaceutical and medical devices industry. The authors have no other 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 apart from those disclosed.

Reviewer disclosures

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

Disclaimer

The views and opinions expressed in this article are those of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of Apellis Pharmaceuticals or Boston Consulting Group.

Notes

1. Throughout the text we refer to a prevalence of 5 in 10,000 people to define a rare disease, based on the EU definition, and to a prevalence of 1 in 50,000 people to define an ‘ultra-rare’ disease.

Additional information

Funding

This paper was not funded.