TSC and LAM Patients Make Their Voices Heard by the FDA

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The FDA makes decisions that determine the lives and treatments of patients with rare diseases. Many of these people who make these decisions aren’t impacted by the disease in their own daily life. That’s not their fault, of course, but it poses a problem. No matter how well-meaning or thoughtful they might be, they can still just miss something that feels obvious to someone with the disease.

This is one reason that it’s important for the patient voice to be heard. The patients and their caretakers can share unique insights that might not be visible from the outside. This feedback is important, so the FDA knows which treatments are working, which aren’t going so great. They can explain what the community is lacking, or hoping for, and what needs they feel are being forgotten. It’s important for patient voices to break through the wall of distance. It’s important for patient voices to emphasize the humans who will be impacted.

The Tuberous Sclerosis Alliance recently made sure their voices were formally heard by the FDA. They worked in conjunction with the LAM Foundation, a group that advocates for lymphangioleiomyomatosis (LAM) patients.

Tuberous Sclerosis Complex (TSC) is a disease that causes the formation of tumors on various organs, including the heart, lungs, and brain. While the tumors are not cancerous, they cause other symptoms, including seizures, skin abnormalities, learning disabilities, behavioral and communication problem, kidney problems, heart problems, and lung problems.

These symptoms can be treated, but there is no cure for the underlying cause. To learn more about TSC, click here. LAM is a rare condition that primarily affects women with TSC. Often LAM patients don’t show many of the classic skin and neurological symptoms, but instead will have severe lung problems.

On October 26th, the TS Alliance submitted a comprehensive report titled “Voice of the Patient” to the FDA. The report essentially gave an overview of the insight provided by panelists and speakers at the TS Alliance-sponsored Externally-Led Patient-Focused Drug Development (PFDD) last summer. The meeting was held to give patients and caregivers a space to communicate their needs and experiences with the FDA.

The report emphasized the patient community’s desire to have FDA view and treat TSC as one whole disorder, rather than a sum of separately treated symptoms. TSC is a disease that permeates throughout the body, even if one organ carries the severe manifestation at a given time. Even in one individual, the most severe symptoms often change throughout their life.

The report also discussed the risks different patients are willing to take. For patients who experience epilepsy, controlling the seizures early in life carries long-term effects. Patients who did not receive proper treatment at a crucial age struggle with more developmental issues. Because of this, caregivers of young children or infants affected by TSC are willing to take risks and try out new therapies.

When it comes to older patients, risks are considered differently. Adults often use established treatments, which delay the need for invasive procedures, such as lung transplants. These invasive measures are often ultimately inevitable.

The TS Alliance and LAM Foundation created this report to help guide the FDA gauge risks and benefits of new treatments of LAM and TSC.

TS Alliance Chief Scientific Officer, Steven L. Roberds, voiced appreciation for the FDA’s willingness to work with the group on incorporating patient voices into the process. FDA senior leadership and staff attended and participated in the PFDD meeting. This showed a genuine interest in learning more about patient needs. Roberds also thanked the FDA for its commitment to taking the “Voice of the Patient” report into consideration when evaluating future decisions.


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