Last Tuesday, May 13, 2014, the U.S. Food and Drug Administration, together with the Pulmonary Hypertension Association, held a one-of-a-kind forum composed of FDA drug reviewers and patients diagnosed with pulmonary arterial hypertension (PAH). PAH is a condition wherein the lungs’ vasculature constricts and raises blood pressure, eventually forcing the heart to exert more effort in pumping blood into the pulmonary circulation. Because there is currently no cure and PAH has proven to be potentially fatal, it will be one of a select 16 diseases the FDA will give a priority to for the first 3 years of the Patient Focused Drug Development Initiative.
While PAH’s occurrence is relatively uncommon, there are only 12 treatment options available today to those living with this condition.
The President of the Pulmonary Hypertension Association, Rino Aldrighetti, said that the working relationships established between all patients, their governments, and the pharmaceutical and medical industry involved with PAH are ideal in their own ways. He added that while there remains much to be done to answer PAH patients’ needs, the association is certain they have enlisted the help of the right people: “There is still work to be done when it comes to treating and curing PAH, but we are confident that we have the right people at the table. We applaud FDA for taking steps to listen to and learn from PAH patients.”
The FDA’s Patient Focused Drug Development Initiative is a reflection of a growing trend in the biotech and drug development industries that seeks to better connect with the patient populations of diseases in order to devise new treatments that reflect the feedback of the people most directly affected. Now more than ever, diseases are being targeted not only by researchers, but also a conflation of patients, supportive friends and families, fundraisers, public health organizations, and companies. With PAH being included in the Patient Focused Drug Development Initiative, there is an opportunity to for those dedicated to fighting the disease to make inroads into supporting the development of new therapies that could dramatically impact the quality of life and prognosis of PAH patients worldwide.