Orphan drugs are indicated in very low prevalence conditions. Most health authorities promote the development of orphan drugs by offering a series of incentives to companies developing treatments or diagnostics for rare diseases – extended market exclusivity, facilitated regulatory processes and financial incentives. Grounded on proven medical plausibility of the orphan condition and potentially significant benefits of the proposed treatment, Orphan Drugs Designations facilitate the access to such incentives.
From the recruitment of a sufficient number of patients with a rare condition to conduct clinical studies to the access to limited number of patients, orphan drugs present many challenges for drug developers. Key to commercial success for orphan indications will be to understand the criteria by which payers will make their assessment. With a growing fraction of the cost burden passed to patients, affordability remains a key limitation to the treatment of rare diseases.
Led by the U.S. National Organization for Rare Disorders (NORD) and the European Organization for Rare Diseases (EURORDIS), patients and patient advocates are heavily involved in orphan drug development. With several hundred drugs under development for rare diseases, the orphan market is expected to experience a two-digit annual growth rate in the coming years.
Key challenges encompass:
- Often no directly applicable guideline: collaborate with experts (Key Opinion Leaders), patients’ advocates, regulators to define path to market
- Positioning of orphan indications within broader development programs
- Compress time-to-market and cost: Orphan Drug Designation, Breakthrough Therapy Status, Fast track / Accelerated Assessment
- Need for adapted clinical design: potentially a single pivotal, statistically significant trial
- Market access strategies tailored to orphan markets’ specificities
- Management of post-approval measures for conditional authorizations/accelerated approvals