Orphan Drugs

Orphan Drugs Around the World – Current Situation and Future Perspective of New Markets

by Marietta Seuß

Orphan drugs (ODs) which are used for the treatment of rare diseases are often not developed or marketed because their extremely limited use makes them unprofitable. In 1983, the US took a first step to meet people’s needs: the Orphan Drug Act (ODA) was established. Based on this law, the developing company of an orphan drug can rely on numerous beneficial incentives when bringing an orphan drug to the market.

Marietta Seuß summarizes the situation of orphan drugs in the US and other countries and explains the incentives for developing orphan drugs such as the Rare Pediatric Priority Review Voucher in the US.

Regulatory Pathways for Orphan Drug Designation

To obtain orphan drug designation, drug developers need to understand how to manoeuvre through the regulatory frameworks and to prepare a sound justification for their drug candidate. The regulatory requirements and evaluation process may vary from country to country and also depend on the policy status and experience of regulators. The individual elements for an orphan drug designation are illustrated by the example of the EU regulatory framework.

Reach the Market Smarter and Faster: Breakthrough Therapies, Orphan Drug Designation, Fast Track ... and the Right Strategy

“Breakthrough” and “Fast Track” are frequently heard buzzwords in the pharmaceutical arena. Looking at development times of up to 17 years from early R&D to product approval with further two years for pricing and reimbursement, it appears that the biopharmaceutical industry is rather on a “Slow Track”. Despite significant advances in medical science, there are still numerous patients without appropriate treatment options.