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Tuberous Sclerosis Alliance Submits "Voice of the Patient" Report To FDA

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2.7 from 3 votes
Tuesday, November 14, 2017

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On October 26, the Tuberous Sclerosis Alliance (TS Alliance) formally submitted its comprehensive "Voice of the Patient" report to the U.S. Food and Drug Administration (FDA). The report summarizes the input provided by participants and panelists during the TS Alliance-sponsored Externally-Led Patient-Focused Drug Development (PFDD) meeting on tuberous sclerosis complex (TSC) and lymphangioleiomyomatosis (LAM), held June 21, 2017 in Washington, DC. The meeting's purpose was to allow individuals and caregivers affected by TSC and LAM to present their perspectives on living with these diseases to the FDA.

Affecting approximately 50,000 in the U.S., TSC is a genetic disorder that causes tumors to form in vital organs, such as the brain, heart, kidneys, skin, liver and lungs. TSC is also the leading genetic cause of both epilepsy and autism. LAM is a rare lung disease that affects many women with TSC and can occur in other women who have none of the neurologic or skin aspects of TSC.

"The TS Alliance is grateful to the parents and adults affected by TSC and/or LAM who bravely shared their stories during the PFDD meeting this past June," said Kari Luther Rosbeck, TS Alliance President & CEO. "We believe this report effectively highlights their daily challenges living with rare disorders, how they evaluate risk and benefit in selecting treatments, and their hopes for the future. We also want to acknowledge The LAM Foundation for partnering with us to ensure the LAM community was fully represented at the meeting."

As the report demonstrates, participants highlighted the need to treat TSC as a whole rather than as a collection of individual manifestations. TSC affects many parts of the body, and the most medically pressing TSC manifestation may change throughout a person's lifetime.

In infancy and early childhood, epilepsy is quite common in TSC, and evidence shows uncontrolled seizures are associated with poor developmental outcomes. For this reason, caregivers of young children with TSC have a relatively high tolerance for risk when it comes to considering new therapies. In adults, existing treatments used to treat TSC or LAM are not curative and may delay but not prevent the need for highly invasive procedures such as lung transplants, for example.

The TS Alliance has requested the FDA use the outcomes of the "Voice of the Patient" report as part of its benefit-risk analysis when evaluating new treatments for TSC.

"Through in-person participation of senior leadership and staff at our externally-led PFDD meeting, the FDA demonstrated its sincere desire to listen and learn more about the TSC and LAM community," said Steven L. Roberds, PhD, TS Alliance Chief Scientific Officer. "We thank the FDA for its commitment to utilizing this report when evaluating potential new therapies."

The TS Alliance's mission is to find a cure for TSC while improving the lives of those affected. To read the "Voice of the Patient" report and view videos recorded during the PFDD meeting, visit www.tsalliance.org/pfdd.

Contact:

Jaye Isham,
301-562-9890,
jisham@tsalliance.org
Source: Tuberous Sclerosis Alliance
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