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
-
These patients had sickle-cell disease. Experimental therapies might have cured themScientists have long known what causes s...
-
The Experience and Health-Related Quality of Life After Haploidentical Stem Cell Transplantation for Adults With Sic...Haploidentical hematopoietic stem cell t...
-
The Upside of Bad GenesFor the last two decades, they’ve had ...
-
American Society of Hematology 2020 Guidelines for Sickle Cell Disease: Management of Acute and Chronic PainObjective: These evidence-based guideli...
-
Emmaus, a leader in sickle cell disease treatment, signs agreement with McKesson to expand distribution network for ...Emmaus Life Sciences, Inc., anno...
-
CTX001 Continues to Show Promise in Severe SCDA single dose of CTX001, an experimental...
-
MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative r...MaxCyte, Inc. today announces it has e...
Don’t forget to join the oneSCDvoice community!
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
Don’t forget to join the oneSCDvoice community!
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.
