In the United States, a rare disease is defined as one that affects less than 200,000 patients. Today there are approximately 7,000 known rare diseases affecting more than 30 million Americans.
Between 1973 and 1983, there were fewer than ten drugs on the market that treated rare diseases. In 1983 the U.S. passed the Orphan Drug Act, which incentivized companies to create products for rare diseases.
This has led to the development and marketing of more than 600 drugs and biologics for rare diseases. In 2019, orphan drug revenue topped $127 billion.
Orphan Drugs and CDMOs
Those numbers will continue to rise, as will the need for Contract Development and Manufacturing Organizations (CDMOs) with orphan drug expertise.
Orphan drugs may not require large volume manufacturing like many other drugs, but they do require a certain level of skill and quality.
While many CDMOs can handle the development of an orphan drug project, many do not end up on board for the entire process. That’s why it’s critical that an orphan drug project is matched with a right-sized partner.
In some cases, the CDMO may lack the necessary expertise when it comes to commercialization. The bigger issue, however, is business related. In many cases, the client – and the maximum potential commercial drug volumes – are not sufficiently attractive for the CDMO once development work has been completed.
Whatever the reason, the client may be left to find a new CDMO – frequently without enough time to adequately vet the new partner. This is not an ideal situation given the shorter timelines unique to the orphan drug designation.
Choosing the Right Contract Development and Manufacturing Organization Partner
So, how can an orphan drug company ensure they find the right CDMO that will work with them from development through commercialization?
Here are the 4 criteria orphan drug companies should consider in selecting a CDMO:
When you’re in the orphan drug space, you will need to have a contract manufacturing partner with mutual interests who can handle the development, the scale-up, and the commercialization while being flexible to your project’s unique needs. Selecting a CDMO with proven experience in all of these areas means you won’t have to worry about the next step.
CDMOs which possess both the scale-up and commercial expertise in the orphan drug space – and share a mutual economic interest with the client at those scales – are a rarity. These factors have been key drivers for Regis’ reputation as a vital partner in the orphan drug space.