Background
As our understanding of the underlying biology of ALS has improved, multiple experimental treatment approaches have been identified. Currently, there are numerous interventional drug trials testing a diverse set of targets using multiple drug approaches (for example, small molecules, antibodies, stem cells, and gene interface/silencing technologies).
Drug regulators have also set up programs to provide special incentives for companies to develop drugs and biologicals for rare diseases that have a small market (fewer than 200,000 people in the United States, as with ALS). One such program is the orphan drug designation (or sometimes “orphan status”) administered by the US Food and Drug Administration (FDA).
Created in 1983, this program provides financial incentives and research subsidies, such as a partial tax credit for clinical trial expenditures, waived user fees, and eligibility for market exclusivity for the drug. The European Medicines Agency (EMA) of the European Union has a similar program and defines a drug as “orphan” for a rare disease affecting fewer than 5 in 10,000 people.
Benefit for patients
The National Organization for Rare Disorders, along with many other organizations, led the lobbying efforts for the passage and creation of this program. Companies will typically shy away from developing drugs for a rare disease area because of financial considerations, limited profit margins, and the scientific, ethical, and operational complexities of conducting clinical research in small niche patient populations. This program motivates, supports, and incentivizes drug companies to invest their research and development capital (both scientific and financial), with the hope that more medical breakthroughs will be made available for patients with rare diseases than would otherwise have been achieved.
Benefit for companies and sponsors
Companies benefit from this program because it gives them exclusive marketing and development rights and allows them to recover part of the cost of researching and developing the drug. In addition to cost reductions, the FDA provides streamlined regulatory processes and guidance for drugs with this designation. Investors in pharmaceutical companies also often view orphan designation as a signal of higher company value, which brings in more of the capital needed for drug discovery programs.
What orphan drug designation means and does not mean
While orphan drug designation can be an important milestone for a drug company, orphan drugs, like non-orphan drugs, are still required to show safety and efficacy before approval for use as a therapeutic. Orphan drug designations are typically given early in development, and orphan drugs must still follow the proper clinical development and regulatory process.
Orphan drug designation should also not be confused with other regulatory designations, such as fast-track designation. Fast-track designation does not provide drug companies with any explicit financial benefits for developing a drug. However, it does provide more frequent meetings with the FDA, as well as an expedited review when a company submits an application to bring a new drug to market.
Recommendation
The Scientific Advisory Council (SAC) hopes that drug companies will take advantage of incentives and expedited pathways set up by regulatory agencies, such as the orphan drug designation, to discover and rigorously test new treatments for people living with ALS/MND.
International Alliance of ALS/MND Associations
July 2020
The original language of communication is English, and any translation cannot be guaranteed for accuracy of messaging.