Trends And Challenges In Rare Disease Clinical Development

Trends in pharmaceutical development come and go. However, looking back over the past few years and projecting forward, one of the strongest trends in the pharmaceutical industry centers on rare diseases.

Datapoints:

• According to the FDA, “There are an estimated 7,000 rare diseases, with a public health impact that affects more than 25 million Americans and many millions more of family members. Between 85 and 90 percent of these cases are serious or life-threatening.”¹
• According to FDA, the number of requests for orphan designation has quintupled since 2000.¹
• PharmaIntelligence notes that “The number of drugs in development for rare diseases has now reached 5,287 – almost one in three, up from 4,953 in 2019. Therapeutics are under development now for 608 individual rare diseases. Concomitantly, the number of orphan drug and expedited review designations granted has also risen (both are associated with rare disease development).”²
• EvaluatePharma predicts that “By 2024, orphan drugs are expected to capture a fifth of worldwide prescription sales and to reach $262bn” and “orphan products currently in R&D will account for 35-38% of sales generated from pipeline products from 2018 to 2024.”³