During the last 10 years, the pharmaceutical industry has ramped up its interest in rare disease. The number of new rare disease therapy approvals globally doubled from 29% in 2010 to 58% in 2018, according to research by Tufts CSDD.
A rare disease is defined as a medical condition that affects fewer than 200,000 people in the United States or fewer than 1 in 2,000 people in Europe.
These diseases are defined by small patient populations and this leads to several challenges for drug developers. The market opportunity is far smaller than pharmaceutical companies have traditionally experienced when launching blockbuster drugs.
Patient identification is also difficult, as is preparing healthcare professionals to diagnose patients with a disease they may not have heard of and may never come across during their careers. Additionally, as more companies target the same diseases, competition for these small groups of patients increases.
To successfully navigate this environment, the industry will need to alter its approach. We interviewed Daniel Zaksas, PhD, Senior Vice President, Director of Scientific and Medical Affairs at Dudnyk to learn his advice for launching a therapy in a small patient population.