Gaining Orphan Drug Status: Complexities Removed

orphandrugsOrphan drug legislation and incentives have been introduced in several countries to encourage the development of treatments for rare diseases.  While the legislation brings opportunities, it also comes with numerous complex regulations and procedures.  To successfully navigate these muddy waters, it requires expertise and deep understanding of the processes and procedures involved to successfully gain the opportunities and incentives available with orphan drug development.

Usually considered one of the most important incentives of the orphan drug legislation in the EU and the U.S. is market exclusivity – a 10-year moratorium on market competition.  This comes into effect, however, only after marketing authorization (MA) has been granted in the EU and after the new drug application (NDA) has been approved by the Food and Drug Administration (FDA) in the U.S.  and that’s where the knowledge and expertise of a CRO comes into play!

The process for orphan drugs generally mimics the same regulatory approval path as non-orphan drugs and can provide exciting and innovative paths forward in rare disease studies.  The introduction in the U.S. of the Orphan Drug Act of 1982 has spurred a rise in the number of orphan drugs being successfully brought to market.  With this success, the European Union has learned from U.S. incentives and introduced similar legislation that offers, fee reductions or waivers, free Scientific Advice and marketing exclusivity to those developing drugs to treat rare diseases.  An EU orphan designation also opens up access to the Centralized procedure and accelerated review programs with EMA.  A challenge often faced by sponsors however, is the differing definitions of an orphan in the U.S. and EU, which is where a CRO experienced in both regions can help.

Updating their guidance in 2015, the FDA offered sponsors further support in addressing the complex issues associated with the development of orphan drugs.  In fact, the guidance highlights the need for sponsors to gain greater biological, clinical, and epidemiological knowledge about the specific rare diseases under investigation, and suggests conducting specific history studies that look at not just the end disease, but also the progression of the disease through initial symptoms, diagnosis and other clinical metrics that could help with the design of a more efficient drug development program.

The recognition in the U.S. and E.U. as well as other areas of the world, is exciting for developers as well as CROs who, because of their specific expertise in regulatory processes can provide great benefits to programs due to their awareness of the best ways to navigate challenges and interact with regulatory authorities.   For sponsors, working with scientific and regulatory professionals who have experience interacting with authorities in both regions can offer significant benefits to advancing programs.

All indicators would seem to point to a continuing increase in the number of orphan drugs being brought to market.  Securing regulatory approvals, however, require partners that are experts in study design, are on top of the ever-changing regulatory mandates and have demonstrated exceptional knowledge of the scope and detail of the drug development process.  By working with these proven strategic partners who have the expertise and experience in designing and delivering orphan drug trials, sponsors can realize better outcomes in their efforts to provide successful relief to those who are fighting rare diseases.