April 28 FDA Meeting: an excellent opportunity for acromegaly patients to get involved

 

On April 28, 2020, there is an FDA Externally Led Patient-Focused Drug Development meeting organized by the Acromegaly Community, a leading patient advocacy organization. To learn more about the meeting, PLEASE CLICK ON THIS LINK OR CONTACT JILL SISCO, ACROMEGALY COMMUNITY PRESIDENT HERE. 

It’s important to note that the FDA has made a great effort to hear from patients and patient advocates to help ensure that experiences, needs, expectations, and priorities are understood and incorporated into the development of new drugs and therapies. In the world of rare diseases, there has decidedly been considerable progress. Much of the advancements were made possible by the Orphan drug act, which was passed by the US Congress in 1983 to encourage the development of drugs for rare diseases. Before this legislation, the industry had little incentive to develop treatments for small patient populations because the drugs were expected to be unprofitable. (We will have more to say about the Orphan Drug Act of 1983, drug prices, and insurance challenges in later articles and podcasts.)

But much work still needs to be done. Many rare disease patient populations have significant unmet needs. On a positive note, in addition to the existing drugs developed for pituitary disease by giants like Pfizer, Novartis, Ipsen and others, several new drugs are currently on the horizon for pituitary patients from small, creative companies such as Crinetics, Ionis, Chiasma, and Tiburio. There is information here on these efforts and links to the trials. On the not so positive side, quality of life is still a challenge, patient diagnosis times take decades from the onset of symptoms, and physicians, particularly non-specialists or primary care docs, are unaware of the issues with acromegaly even after a patient is diagnosed and treated.

This April 28, there is an opportunity for all Acromegaly patients to address their needs and challenges in an Externally led FDA meeting known as a PFDD meeting (Patient-Focused Drug Development). These meetings, which are attended by patients, advocacy groups, academics, clinicians, industry partners, are designed to communicate and provide an understanding of the disease and available treatments. This meeting offers FDA the opportunity to hear directly from individuals, caregivers, and other stakeholders about their experiences and perspectives with acromegaly

The April 28 FDA Externally Led Patient-Focused Drug Development meeting is led and organized by the Acromegaly Community, a leading patient advocacy organization. To learn more about the meeting and how to receive a stipend to cover expenses, please click on this link or contact Jill Sisco, Acromegaly Community president here.

Please note: For those unable to attend, the meeting will be streamed live online – please stay tuned for more details on this.

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