Orphan Drug Pricing And Reimbursement: Challenges To Patient Access

 
• By Joshua Cohen
  
Generate a summary of this article
Listen to this article
Beta

The drug industry and patient advocacy groups have been leery about the imposition of price controls or restrictions on reimbursement for drugs that treat orphan diseases. A review of formulary data from the beginning of the century shows that there has been some pushback from payers about these high-priced drugs, but patient access has not been severely hampered to date.

Orphan Drug_1200

In 1983, the US government passed the Orphan Drug Act (ODA), which fosters development of treatments for rare diseases affecting fewer than 200,000 individuals. Available through the act are special research grants, a 50% tax credit on clinical trial costs, shorter Food and Drug Administration (FDA) approval times and a guaranteed seven years of patent exclusivity. In terms of spurring orphan drug development, the ODA has been an unequivocal success.

In the decade prior to 1983 only 34 orphan drugs were licensed, whereas since 1983 the Food and Drug Administration...

In Vivo Podcast

Candid conversations with C-suite leaders.

Keep up with strategy, innovation and evolving trends across life science industries.

SpotifyApple PodcastsWebsite

More from Market Access

How To Decarbonize Pharma R&D

As the planet heats up and sea levels rise, the need to reduce carbon emissions is becoming ever more urgent. The life sciences industry is just one of many that are looking to cut the carbon it produces.

IV-MarketAccess (Premium-1): Stimulant Medications Don’t Cause Psychosis, New Study Finds

 
• By Nabil

Instead, the new study found children with more severe attention and hyperactivity issues or other mental health conditions such as anxiety were more likely to be medicated for ADHD.

[Test]With 75 Pipeline Candidates, TIL Therapies Gain Momentum in Solid Tumor Space

 
• By Nabil

Tumor infiltrating lymphocyte therapies show promise for solid tumors, with a first FDA approval, but face manufacturing and access challenges as development continues.

In Search Of Rare Disease Treatments, Nonprofits Can Lead the Charge

 
• By David Wild

Nonprofits are finding new ways to address market gaps and develop treatments for rare diseases with little commercial attraction.

More from In Vivo

XXXXtesting

 
• By Saba Afreen

XXXXtesting

In Vivo’s 2026 Rising Leaders At A Glance

 
• By Saba Afreen

Top 30 leaders: see the list of industry influencers highlighted in the sixth edition of In Vivo's Rising Leaders series.

InVivo-Innovation-Free: ‘Interview’ article that includes different Composer content

 
• By Nabil, Olga Zviezdicheva, Reed Miller, Mary Jo Laffler, Ian Schofield, João Andrés-Ruíz de la Fontaine-LeBlanc, Chloë Anastasía-Stojanović del Castillo-Ribeirão, and Nabil

To make your document look professionally produced, Word provides header, footer, cover page, and text box designs that complement each other. For example, you can add a matching cover page, header, and sidebar. Click Insert and then choose the elements you want from the different galleries.