“Safety and efficacy data is needed before generics can be used on rare disease populations,” said Lerman.
FORT LAUDERDALE, Fla. (PRWEB) May 30, 2019
Attorney and rare disease patient Candace Lerman is sounding the alarm on the potential risks associated with doctors prescribing generic drugs to patients for off-label use when the brand name product has not yet undergone a clinical trial.
Lerman has Immune Thrombocytopenia (ITP), a rare blood disorder where the body destroys platelets, leaving her at risk for potentially fatal internal bleeding. In 2014, she worked with her doctor to repurpose Rituxan, a monoclonal antibody which is FDA approved to treat Non-Hodgkin’s Lymphoma and Rheumatoid Arthritis. The off-label treatment resulted in a remission period of over four years with a normal platelet count. However, Lerman recently discovered her ITP is out of remission, and now with biosimilars flooding the market, she is concerned about the potential, unknown side effects of using these types of products.
Like Lerman, more than 30 million Americans are impacted by one of over 7,000 rare diseases, but less than 5% of those conditions have an FDA-approved treatment. Patients with rare conditions commonly repurpose existing drugs to manage the symptoms of their diseases, but little is known about potential risks from these therapies.
In light of recent news highlighting problems with the production and importing of generic drugs, Lerman is stressing the need for pharmaceutical companies to work with rare disease patients to put their products through clinical trials. “Safety and efficacy data is needed before generics can be used on rare disease populations,” she says. Contamination, poor manufacturing processes, and counterfeit drugs pose an extreme risk to rare disease patients, who have been underserved for decades.
“Recent stories highlighting the issues surrounding generic drugs has caused me to think twice about my treatment options for ITP,” said Lerman. “While affordable drugs are extremely important, I am not willing to risk my life by using a generic drug or biosimilar where the branded medication has not been vetted by the Food and Drug Administration for my condition.”
To address the need for better ITP research, Lerman is launching the ITP Patient Driven Research Initiative, with the goal of establishing clinical diversity criteria to create better targets for drug development.
“While the ITP community has celebrated the approval of three drugs for our condition, many patients are still struggling to access effective treatments,” Lerman added. “The ITP Patient Driven Research Initiative is being led by a patient, for patients, to finally address the unmet need of stopping immune-led destruction of platelets.”
Lerman continued: “I look forward to working with patients, pharmaceutical companies, researchers, and legislators to improve outcomes for the ITP community.”
The ITP Patient Driven Research Initiative can be found on Facebook at https://www.facebook.com/ITPResearch.