Trusted Resources: Evidence & Education
Scientific literature and patient education texts
The FDA Encourages New Treatments for Sickle Cell Disease
source: U.S. Food and Drug Administration
year: 2018
summary/abstract:The FDA continues to work with stakeholders including patients, academics and companies developing treatments to improve therapeutic options for people living with SCD.
A growing number of new products are in the pipeline, and researchers are exploring new treatment approaches. “These potential treatments are in different stages of development, including early and late clinical trials,” Farrell says. (Clinical trials are voluntary human research studies designed to answer specific questions about the safety and effectiveness of potential new treatments or to study new ways of using existing treatments.)
Companies developing sickle cell products can ask the FDA to grant a “fast track” designation, a type of regulatory status that seeks to bring new products to market faster. “This allows for early and more frequent interactions with the FDA to discuss any issues that come up during development, such as manufacturing and trial design,” Farrell explains.
If preliminary data show potential promise over existing therapies, companies also can request “breakthrough therapy designation” to speed the development and review of products.
+myBinderRelated Content
-
Explaining Blood Transfusions to a Child With Sickle Cell DiseaseYou are receiving this brochure because ...
-
Treating Sickle Cell Anemia in ChildrenYour child has sickle cell anemia. This ...
-
Study Confirms Safe Use of Opioids for Pain Control in Sickle Cell DiseaseThe most common cause of hospital admiss...
-
Bluebird bio acquires Durham, NC, manufacturing site for lentiviral vector productionbluebird bio said today it has acquired ...
-
Greenville program tries team approach to improve lifespan of sickle cell disease patientsDr. Alan Anderson who was involved with ...
-
These patients had sickle-cell disease. Experimental therapies might have cured themScientists have long known what causes s...
-
Gene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.