Top Three Challenges Facing Orphan Drug Candidates

May 17, 2017 9:00:00 AM / by John Kouten, CEO
orphan-drugs.jpgPrior to the passage of the 1983 Orphan Drug Act, just 34 drugs were approved to treat rare diseases, leaving small patients populations with few options for their rare diseases. That number has since skyrocketed with the implementation of the Orphan Drug Act. In 2016 alone, the FDA designated 333 drugs in development as orphans and approved 39 products, including both novel medicines and new orphan uses of approved drugs. Since the Orphan Drug Act took effect, The FDA has designated 3,600 orphan drugs and approved 552 orphan drugs.

This landmark bill provides life science companies incentives — including 7-year market exclusivity on a single treatment, tax credits for half the cost of clinical development and waived FDA user fees—to develop treatments for rare diseases. Both biotech and pharmaceutical companies have responded, bringing life-saving medicines to patients, once neglected, while making the orphan drug business flourish.

In 2016, orphan sales reached $114 billion, a 12% increase versus 2015, and this market is on pace to double over the next six years, Evaluate Pharma reports.

While there has been significant progress in the development and approval of orphan drugs, challenges still lie ahead for orphan drug candidates.

  1. Scrutiny of drug pricing –Patient advocates and lawmakers voice concerns over orphan drug costs. Critics say pharmaceutical companies are unfairly pricing orphan drugs. And sometimes the scrutiny can lead to investigations or a delayed product launch, as seen in case with Marathon’s Emflaza, an orphan medication approved to treat Duchenne muscular dystrophy.
  2. Poor disease understanding –Unlike diseases affecting large patient populations, the natural history of rare diseases is difficult to fully understand. This fact makes development programs for rare diseases a fundamental challenge. In addition, typically very little scientific literature is published about rare diseases and experts on these diseases are few and far between.
  3. Clinical trial recruitment – Patient enrollment and retention is challenging in most rare disease clinical trials. From patient populations that are both small and geographically diverse to the fact that more half of rare diseases affect the pediatric population, most researchers find it difficult to register patients for trials.

Topics: drug development, rare diseases, orphan drugs, Precision medicine, FDA approval process

John Kouten, CEO

Written by John Kouten, CEO