The Orphan Drug Act of 1983 encourages the development of drugs for rare diseases, a process with unique clinical and commercial challenges. Although manufacturers of orphan drugs generally enjoy greater pricing latitude in the small populations their products serve, the discussion about orphan drug value assessment is rapidly evolving as the number of rare disease approvals climbs. A key concern for developers is how payers will control the growing cost of orphan drugs and how this policy will affect prescribing of these clinically valuable medications. Here, we provide an analysis of payer policy in cystic fibrosis (CF), idiopathic pulmonary fibrosis (IPF), Huntington’s disease (HD), spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), beta-thalassemia, and chronic immune thrombocytopenia purpura (ITP), as well as stakeholder receptivity to key emerging and recently launched orphan agents, including Vertex Pharmaceuticals’ triple combination therapy for CF, Novartis/AveXis’s AVXS-101 for SMA, Bluebird Bio’s LentiGlobin for beta-thalassemia, Rigel’s Tavalisee for chronic ITP, and Sarepta’s golodirsen for DMD.


  • What impact do orphan drugs have on payer budgets? What measures do payers expect to implement to rein in costs associated with orphan drugs?
  • What pharmacoeconomic models do/will payers use to assess the value of orphan drugs and gene therapies?
  • What utilization management restrictions do physicians encounter when prescribing key orphan drugs, and how commonly?
  • How does payer policy influence orphan drug prescribing patterns?


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

Table of contents

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

Author(s): Claudia Dall'Osso

Claudia is a Senior Business Insights analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing on niche and rare indications.

Before joining DRG, she held a management and strategy consultant position at Precision Medicine Group, where she worked for clients in the biopharmaceutical, medical device and diagnostic industries. Claudia completed her Master’s in Management at Harvard University; she also holds a Ph.D. in medical genetics from Brescia University in Italy and a BS/MS degree in medical biotechnology from University of Milano in Italy.

Related Reports

Myelodysplastic Syndromes - Landscape & Forecast - Disease Landscape & Forecast

Myelodysplastic syndromes (MDS) are a heterogeneous collection of hematopoietic stem-cell disorders that result in cytopenias and the risk of progress...

View Details

Rare Diseases And Orphan Drugs - Landscape & Forecast - Disease Landscape & Forecast: Gene Therapies For Rare Diseases

Approximately 80% of rare diseases have a genetic etiology. Gene therapies hold the potential to transform the treatment of rare diseases and may provide a functional cure for some. However, these...

View Details

Myelodysplastic Syndromes - Epidemiology - Emerging Markets

DRG Epidemiology's coverage of myelodysplastic syndromes comprises epidemiological estimates of key patient populations across 45 countries worldwide. We report both the incidence and prevalenc...

View Details

Rare Diseases And Orphan Drugs - Access & Reimbursement - Detailed, Expanded Analysis - Rare Diseases And Orphan Drugs (US)

Renewed interest in the development of gene therapies has fueled commercial interest and investment into novel gene replacement technologies, which has resulted in a strong, diverse gene therapy pi...

View Details