Keeping the Focus on Patients


Treating illness and disease is no easy task. It takes a combination of dedicated medical and healthcare professionals, hardworking scientists and researchers and a strong support system of family and friends to make a patient's course of treatment as effective and comfortable as it can be. The best way to make that happen? Involving patients in the process.

To that end, the Food and Drug Administration (FDA) implemented the Patient Focused Drug Development (PFDD) initiative in 2012. A main component of the most recent authorization of the Prescription Drug User Fee Act (PDUFA), PFDD provides patients living with a wide variety of diseases and illnesses a way to talk directly to the FDA in public forums. The idea is that by directly engaging with patients, the FDA can get a better understanding of how they are actually managing their conditions and the impact their medications have on them. The FDA, in return, can take this input into account when it considers the benefits and risks of potential treatments, ultimately lowering the chance of negative effects and improving the chance of successful treatment.

The FDA has made great use of this program since it began. Six formal meetings were held last year alone, bringing in patients and advocacy groups representing many illnesses you have heard of, and likely a few you didn't know existed. In April, they gathered input on breast cancer and Chagas disease, an infectious disease caused by parasites. In September, Parkinson's and Huntington's Disease. In October it was mycobacterial infections. Each time, patients, families, caregivers and advocates gathered and shared their firsthand stories and opinions of how future treatments should look.

More of the same is in store for 2016, as the FDA has plans to continue arranging meetings and listening to patients to continue using this valuable program. In March, people with psoriasis discussed their experiences, and in June, those living with peripheral neuropathy (numbness and pain from nerve damage) will come together to talk with the FDA. Throughout the rest of the year, patient communities representing autism, alopecia, and several other conditions will get to do the same.

Rarely do consumers, manufacturers and regulators come together in a singular effort. Even rarer is when an idea transforms from legislation into effective, real-world action in just a few years' time. PFDD exemplifies this collaboration, and it's resulting in real impact for many patients and their families. That's exactly why Congress finds it extremely important to continuously reauthorize PDUFA when it expires every five years - to improve and add programs that benefit both patients and health care professionals.

The next Congressional review of PDUFA, slated for the fall of 2017, is just around the corner, and negotiations on the content of the new bill are already well underway. We eagerly await to see the results of these discussions and expect them to produce even more components of the PFDD initiative, including meetings with additional patient groups and a more systematic collection of feedback. Additions such as these will help ensure that new treatments are designed to treat disease and illness in ways that improve quality of life for every patient. Over the course of the reauthorization process, stay up to speed on news and updates through our blog and on social media at @ModernMeds.