Curing a rare disease is something I’m passionate about and, given my role in clinical supply chain planning, it’s something I can contribute to on a daily basis. But dealing with therapies for rare diseases is complicated and costly. That is why, in 1983, the US FDA established the Office of Orphan Products Development (OOPD) program to incent sponsors to develop products for rare diseases. Similar legislation has been enacted in Japan and the EU. These programs which provide tax credits and/or market exclusivity have resulted in an exponential increase in development, increasingly including big pharma/biotech as well as specialists. Clinical supply strategy has required significant adjustments because planning for these trials is different than for therapies associated with common diseases.
At last week’s Global Clinical Supplies Group 2018 European Knowledge Forum I joined industry colleagues in a discussion of the challenges associated with clinical supply in the context of rare diseases.
Rare Disease Characteristics and Challenges
- It may take years to find patients and close out a study, making shelf life and repackaging an ongoing consideration that must be addressed, as well as retention.
- Because patient enrollment is challenging it can require a broader geography which introduces logistics challenges e.g. product delivery to remote locations, longer distance for patient to travel.
- When working with remote locations, it’s possible they may not have the required infrastructure both in terms of facilities and prior research skills, placing an increased burden on the Sponsor to provide additional support and training.
- As the drug is so rare and valuable, it might need to be conserved and only shipped when an eligible patient is identified, impacting storage and distribution strategy.
- Infectious disease or seasonality may require a rapid response and agility in the supply chain.
How do Rare Disease Trials Differ From Traditional Trials?
- These patients are often very well informed and eager to share experience, which can enable Sponsors to tap into a network yet be aware that news travels fast in these communities.
- There is often no comparator or gold standard. Measurements of treatment success can become more subjective. It’s possible only milestone data are captured which can inform other studies.
- Developers might have only 1 or 2 of these therapies in their pipeline, so they are less familiar with this population segment.
- There’s a larger number of pediatric studies. Dosing might require weight calculation and special delivery systems may be required.
- New gene therapies can be a once and done administration. Getting the therapy to the right place at the right time can be challenging.
- Manufacturing is in small quantity and at a limited number of sites, so supply is extremely limited. At the same time, forecasting is an uncertain process. Clinical supply must manage the delicate balancing act of limited supply and unclear forecasts while ensuring there isn’t a patient-impacting shortage.
Our collective brain trust that was in attendance at GCSG EU Knowledge Forum could have gone on for hours. Mercifully for all, we were restricted to one hour. Before we adjourned we had an enlightening exchange on strategies we can use to help address the unique challenges of these therapeutic areas. My next article will summarize these great insights: Successful Clinical Supply Strategies for Rare Diseases.