The FDA Is Supporting These Rare Diseases, Did Yours Make the Cut?

The people have spoken.

The Patient-Focused Drug Development Initiative of the Food and Drug Administration (FDA) has announced the selection of diseases it will address during 2016 and 2017.

Which begs the question, why should you care?

Based on the comments and input from patients with rare diseases, the FDA has decided to pay special attention to nine rare conditions, including hereditary angioedema, autism, organ transplant patients, and more.

Every hereditary angioedema patient, family member, physician, and support group needs a round of applause for all their efforts!

After receiving comments addressing more than 50 disease states, the FDA carefully selected the diseases it would address based on categories with low clinical trial data or diseases with few therapies.

In a video promoted by the HAE Association, Marc Riedl M.D. of UCLA explores the various treatment options that have been provided for hereditary angioedema since 2008. Source: Youtube

The purpose of the decision is to improve how regulatory decisions are made based on a balance of risks and benefits. The FDA, and us at Patient Worthy, know that patients who live with the diseases are in a unique position to contribute to their understanding of the disease. These patients give the FDA more of a voice and perspective on disease severity and the treatments (if any) that are available to them.

For each disease selected to be addressed, the FDA will conduct a public meeting (including the participation of review divisions, relevant patient community members, and other interested stakeholders) to discuss the disease, its impact on patients’ daily lives, the types of treatment benefits that matter most to patients, and patients’ perspectives on the effectiveness of available therapies.

The FDA, however, realizes there are several other diseases worthy of being studied, and say they will seek opportunities to gather public input on disease areas that haven’t been selected for next year.

The FDA isn’t the only organization that realizes many other patients of rare disease need their voices and concerns heard. Take our poll to tell us which disease the FDA should focus on next. Who knows, maybe we can get your condition on the map!

Choose a social network button and share this poll to help the voices of others dealing with rare diseases be heard!

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