21st Century Cures for Those Who Need It Most
Advocacy Rare diseases affect 1 in 10 Americans, meaning more than 28.5 million Americans are without a solution to the often debilitating problems a rare diagnosis brings in to their lives.
Fat loss? Excessive hunger? Could it be GL?
A person with GL appears very thin and often muscular. While this may sound ideal to some, it is important to understand the role that fat plays in regulating systems throughout the body.
Generalized lipodystrophy (GL) is a rare sub-type of lipodystrophy characterized by a complete fat loss, in turn causing lack of leptin and metabolic complications. The condition is characterized by the loss of fat tissue all over the body and can either be inherited or acquired. Depending on the type of GL, fat loss can be present at birth, shortly after or occur later in life.
Fat produces an essential hormone called leptin. People living with GL have low leptin levels that lead to excessive hunger and an inability to process food properly. The serious complications that occur from the lack of fat and low leptin levels include dysfunction of organs.
Many medical advancements have been made in recent years and treatments are available. Even so, it remains imperative that vocal leaders within the health care community continue to expand knowledge on GL and advocating for those living with this disease.
By Andra Stratton, President and Co-Founder, Lipodystrophy United
The status quo for rare-disease patients is unacceptable. Ninety-five percent of rare diseases currently lack a treatment approved by the Food and Drug Administration (FDA).
The 21st Century Cures Act, which passed the House of Representatives with broad bipartisan support last July, has the potential to drastically change the current landscape for rare disease patients.
The act would provide new incentives to dramatically increase the number of treatments while also modernizing the drug development process to expedite the availability of safe and effective, new medicines.
Call to action
The Senate Health, Education, Labor and Pensions Committee recently announced plans to consider individual medical innovation bills, rather than advancing comprehensive legislation as a companion to 21st Century Cures. Included are several bills of importance to the rare community, such as the Advancing Targeted Therapies for Rare Diseases Act of 2015.
Unfortunately, two bills that were an essential part of the House package were excluded. The Orphan Product Extensions Now, Accelerating Cures & Treatments (OPEN ACT), which would grant six extra months of market exclusivity for drugs to treat common diseases that are repurposed and approved for rare diseases, has the potential to double the number of therapies available to rare disease patients. The Patient-Focused Impact Assessment Act would require the FDA to incorporate patient engagement feedback in the drug development process.
Both bills are bipartisan, supported by hundreds of patient organizations and critical in Congress’ efforts to bring lifesaving treatments to rare disease patients across America. We urge the Senate to swiftly consider them among the other medical innovation bills.