Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on this space are key factors driving continued growth in the number of approved and emerging orphan drugs. However, with many orphan drugs priced at high premiums, and given the increasing armamentarium of such agents, the EU5 healthcare agencies must find ways to manage their ever-tightening budgets to best serve the areas of high unmet medical need that orphan drugs seek to address. This study analyzes the health technology assessment (HTA) and pricing and reimbursement (P&R) policies that are used to regulate market access for orphan drugs, specifically exploring the evolving dynamics affecting reimbursement and uptake of current and emerging orphan drugs for cystic fibrosis (CF), idiopathic pulmonary fibrosis (IPF), Duchenne muscular dystrophy, (DMD), spinal muscular atrophy (SMA), immune thrombocytopenic purpura (ITP), and beta thalassemia (BT).


What factors influenced HTA and P&R outcomes for select current orphan agents, with a focus on agents for CF, IPF, DMD, SMA, ITP and BT?

What is the current market access status for Kalydeco (CF), Orkambi (CF), Esbriet (IPF), Ofev (IPF), Translarna (DMD), Spinraza (SMA), Promacta (ITP), and Nplate (ITP), that represent different price points, varied target population sizes, different levels of unmet need, and staggered approval times.

What funding mechanisms are used for orphan drugs, and what measures are used for controlling and monitoring their use across the EU5 markets?

How will market access barriers for orphan drugs evolve and what challenges are emerging orphan drugs likely to face in the near future?

Product Description:

Access & Reimbursement reports provide in-depth insight regarding the impact of payer policies on physician prescribing behavior so that clients can build their market access strategy and optimize their brand positioning.

Content Highlights:

Actionable recommendations to optimize market access, market access success and stumbles, market access roadblocks, reimbursement dynamics, the impact of pricing and reimbursement, policy, and coverage on prescribing.

Scope of Report:

  • Geography: France, Germany, Italy, Spain, and the United Kingdom (i.e., EU5).
  • Primary research: Survey of 300 physicians in the EU5 (60 per country) and interviews with 10 EU5 payers (2 per country).
  • Key drugs covered: Kalydeco, Orkambi, Esbriet, Ofev, Translarna, Spinraza, Revolade, Nplate

Table of contents

  • Rare Diseases And Orphan Drugs - Access & Reimbursement - Detailed, Expanded Analysis Orphan Drugs (EU)
    • Access and Reimbursement Rare Diseases and Orphan Drugs (EU5)

Author(s): Henrique Duarte; Akash Saini, Ph.D.

Henrique Duarte is an analyst on the Global Market Access Insights team at Decision Resources Group, primarily focusing on European market access. He is responsible for monitoring, analyzing, and reporting on global market access through the production of DRG’s Global Market Access Solution (GMAS) and Access & Reimbursement products. Henrique’s specific focus is on all aspects of market access in the EU5, as well as in select Latin American countries. Henrique holds a pharmaceutical science degree from the Universidade Lusófona and a postgraduate certificate in health economics from the University of York.

Akash is a Principal Analyst in the China In-Depth team at Decision Resources Group. Since 2016, he has specialized in a range of indications including rare disease indications such as DMD, ALS, ITP, IPF, JIA, and retinitis pigmentosa. He has authored Disease Landscape & Forecast reports, Access & Reimbursement reports, and Treatment Algorithm reports based on primary market research and real-world evidence. Prior to joining DRG, Akash was a post-doctoral fellow at the University of Massachusetts Medical School. He has a Ph.D. from the International Centre for Genetic Engineering and Biotechnology, New Delhi.

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