In recent years, there has been an increase in the number of new drugs and therapies developed for rare diseases or specific patient populations. The FDA’s Center for Drug Evaluation and Research approved 50 new drug therapies in 2021. Of those, 26 were considered orphan drugs—products to prevent, diagnose, or treat conditions or diseases that affect fewer than 200,000 people. Because this rate of growth is expected to continue, CDER created a rare drug hub.
Before 1983, orphan drugs did not exist. As explained in this post from Pearl Pathways, the imagination of Maurice Klugman, a writer and brother of Jack Klugman, who starred on the TV show Quincy, M.E., brought the issue to prime time and sparked an advocacy movement.
Though the conditions orphan drugs treat are rare, millions of people are affected by these cancers, autoimmune, inflammatory, cardiac, and kidney diseases. In response, companies and labs are testing not just medications, but also diagnostic tools, cell and gene therapies, and devices.
Working with unique patient populations can present unique challenges. Partnering with a contract research organization (or clinical research organization) can accelerate the timeline in bringing orphan drugs or related medical devices and diagnostics to market. A CRO can offer expertise in planning and conducting clinical trials, as well as in navigating the development and regulatory pathway to approval.
The biggest CROs have broad offerings. They have experience with and systems in place for a variety of treatments and technologies. And they have name recognition, which can go a long way in dealings with the FDA.
On the other hand, a CRO with a giant footprint might be less nimble. For individuals or companies focusing on specialized products, partnering with a smaller, “niche” CRO may be the right strategy. For example:
- Niche CROs often specialize in specific drug classes, therapeutic areas, or orphan drug submissions, unlike large CROs that handle more “cookie-cutter” drug development. This expertise means the niche CRO can design clinical trials that will work with a smaller patient pool.
- Small CROs are more cost effective. With a smaller team, communication is less likely to be filtered through layers of management. Thus, there are fewer chances for the unexpected, added expenses of extended timelines or new managers joining mid-project.
- Niche CROs are often started by individuals with highly specialized experience. They come to the table with knowledge of research in specific disease states and/or drug classes. This allows them to articulate strategy and communicate the importance of innovation with the FDA and other regulatory bodies.
- A smaller firm can scale up or down on a project, to ultimately offer what large firms can. Companies working with large CROs can be disappointed to find that the A-team that lured them to the table is not the same team working on the project. Also, with a smaller CRO, senior management is more accessible.
- Small CROs are flexible. They can adapt to shortened timelines or changes to projects, while a large CRO with multiple layers of bureaucracy are naturally slower to respond. And processes in place in a large organization might not work for a specialized treatment or device.
Pearl Pathways is an established, woman-owned CRO, regulatory, and quality service provider. Our team helps clients develop and execute a sound approach to clinical development – all while balancing risk, speed to market, and cost to achieve their business goals – so that FDA reviewers will more easily comprehend critical data and information. Contact us for more information.