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Acromegaly Community, Virtual EL-PFDD Meeting

January 21 @ 5:00 pm - 10:00 pm

Cambridge Rare Disease Network - Acromegaly Community, Virtual EL-PFDD Meeting 1
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This meeting is a groundbreaking initiative that will give patients the opportunity to voice the perspective of living with acromegaly today

About this Event

The upcoming acromegaly externally led Patient-Focused Drug Development (PFDD) meeting is a groundbreaking initiative that will give acromegaly patients and their families the opportunity to provide the Food and Drug Administration (FDA), drug developers, and NPC stakeholders with perspectives from our community on a number of important issues.


The meeting will follow the successful model developed by the FDA for similar Agency-convened meetings and provide persons with acromegaly the unique opportunity to share their story and experiences—including the challenges they face–with senior FDA officials and industry to better inform how therapies are developed and how the Agency evaluates and considers potential therapies for the disease.


It’s mission is critical that all stakeholders (patients, physicians, pharmaceutical companies and the FDA) participate, which is why I’m asking for your support for this one-time momentous event.


DATE: January 21, 2021 10:00 am – 3:00 pm EST



BACKGROUND: We have requested this meeting to help improve the understanding of this disease from the patient perspective.  We recently asked our members to participate in the ACROQOL (a tool commonly used in clinical trials to determine quality of life of the acromegaly patient) and what we saw was shocking and disturbing.  Of the participants, we found that the quality of life of the typical patient is very poor and we are looking for ways to improve this.


ATTENDEES: We are requesting all stakeholders to attend, by this we mean not only patients, but physicians that treat this condition, pharmaceutical companies that have both approved medicines and those that have medicines in the pipeline for acromegaly treatment and the FDA.  The following FDA divisions have stated they will be in attendance for this meeting:  Endocrine, Tumor and Rare disease.  I have been told others within the Agency may also attend. We welcome patients from around the world to participate.


FUNDING: All expenses for this salient event fall to the Acromegaly Community. This includes development of content, securing clinical experts and patients to share the array of perspectives across the acromegaly community, and real-time polling questions about experiences with the disease. The event will be live streamed to enable remote participation.


OUTPUT: Following the meeting, a “Voice of the Patient” report will be developed by a medical writer.