Over the past few weeks, through a series of eBooks, we have been highlighting some of the most researched rare diseases. Why? Well, even with fewer than 300 approved orphan drugs for nearly 7000 rare diseases, certain rare diseases receive greater attention than others in terms of research and the clinical development of therapies.
This is the third eBook which places the spotlight on rare disease research into Cystic Fibrosis, a hereditary disease that affects the production of secretions (such as mucus) from the glands in the body, particularly affecting the lungs and the digestive system (gut) in particular.
While we’ve been looking into current rare disease research, we’ve also been asking ourselves some questions about the future of the industry. Like which rare diseases will the next wave of orphan drugs treat? By the time the next wave of orphan drugs are granted safety and efficacy approval, will the patient market be as we know it today? Especially given that the rare disease industry, whilst growing at an enviable rate, has only just found its feet as a viable business model with a long road ahead to treat 7000 diseases.