Nonprofit Drug Development is the Answer for Many Rare Disease Communities in Need of Treatment

Written By Ashely Winslow, PhD, President & Chief Scientific Officer of Odylia Therapeutics

One of the biggest issues impacting many rare disease communities is the availability of approved treatments. While research teams make promising advances every day that could lead to development of new therapies, over 95 percent of rare diseases still have no approved treatments. One reason for this gap is that traditional for-profit drug development models do not work well for rare diseases. Commercial drug developers who might consider developing drugs for rare diseases often conclude that the limited return on investment does not justify the costs and risks associated with advancing a promising therapy through clinical research and regulatory review. As a result, many promising drugs developed in academic research centers, and even industry, are left sitting on shelves. To bring more treatments to people in need, a nonprofit drug development model is increasingly seen as the only viable option. This model prioritizes the clinical potential of a therapeutic and the benefits it can deliver to patients over commercial interest and profit potential.

All drug development begins with a research and discovery phase. During the first phase, researchers conduct studies to identify a therapeutic that has the potential to treat a disease or condition. In the traditional commercial drug development model, pharmaceutical or biotechnology companies make the decision and the investment necessary to advance these promising drugs through clinical trials and submit applications to regulatory agencies (such as the U.S. Food and Drug Administration) for review and approval. While commercial entities are motivated by the potential to bring a new treatment to patients in need, the also expect that drugs can deliver a target level of profits once they are available commercially. The profit potential of a drug is largely dependent on the size of the population that will receive the drug, and the eventual price of the drug. Drug development is a long and costly process, and commercial pharmaceutical companies are most likely to consider development programs with the potential to generate the highest levels of revenue and profit. As a result, they are more likely to move forward with treatments for more common conditions that affect many people instead of treatments for rare diseases.

The nonprofit model is designed to overcome this challenge. With funding from many stakeholders including families, foundations, industry and government, nonprofit drug developers have the freedom to focus on advancing successful new treatments that commercial entities generally won’t consider. It is drug development through the lens of science and clinical outcomes rather than profit generation. Nonprofit drug developers can advance promising research to clinical trials and regulatory review with a constant focus on the potential clinical benefits rather than the bottom line.

While nonprofits can help move forward promising research without a focus on profit, these efforts do still require funding and the active participation of a range of stakeholders. Here again, the nonprofit model offers significant advantages. Nonprofits have more opportunities to develop innovative partnerships that bring together the resources and expertise necessary to rapidly advance therapeutic development programs, without concerns related to competition. With a compelling mission, the nonprofit model can also inspire more stakeholders to take an active role in helping promising research move across the finish line.

When profitability is removed from the decision-making process, we believe better treatments are developed for more people. The nonprofit investment in early phase programs means treatments are developed based on clinical potential. This creates opportunities for unique partnerships with for-profit companies as rare disease drugs are de-risked, and the potential for failure is reduced. The profile of these de-risked drugs can become more attractive for for-profit companies to invest in because the likelihood of success is high.

As many rare disease communities remain in desperate need of new treatments, it is clear that the traditional model for developing new therapies will not meet this need. Patients, advocates, funders, and leaders from government and industry can all take an active role in supporting a nonprofit model and collaborating with organizations positioned to bring new treatments to more people living with rare diseases. Working hand-in-hand with commercial models for drug development, this expanded focus provides the best chance to bring life changing treatments to more people living with rare diseases in the years ahead.

Dr. Ashley Winslow is the President and Chief Scientific Officer of Odylia Therapeutics, a nonprofit drug development company dedicated to advancing life changing therapies for people living with rare genetic diseases. In her previous roles, Dr. Winslow worked at the Orphan Disease Center at the University of Pennsylvania where she was the Senior Director of Portfolio Development and Translational Research. Prior to the Orphan Disease Center, she worked at Pfizer as an Associate Director of Neuroscience Genetics. Dr. Winslow received her Ph.D. from the University of Cambridge in Medical Genetics and Molecular Cell Biology and completed her Bachelors degrees in Chemistry and Anthropology from the University of Florida.

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