Draft guidelines for validating treatment processes
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A public meeting is also planned “to obtain patients’ and caregivers’ perspectives on the impacts of rare diseases on daily life” that might help in the development of new treatments and in improving clinical trials. “The FDA is committed to supporting the development of treatments for patients with rare diseases and has been focused on advancing policies that will help enable these opportunities,” Scott Gottlieb, the agency’s commissioner, said in a releasethat accompanied the draft guidance. discusses select issues commonly encountered in rare disease drug development,” Gottlieb added.
“While similar issues often also come up for common diseases, they’re frequently more difficult to address in the context of a rare disease for which there’s often limited medical and scientific knowledge.” When finalized, the guidance will present the agency’s current thinking on the topic.
Because of the deadly nature of some rare diseases, experts say, it can be difficult to get enough patients for a viable control group — patients who will not be given the investigative treatment, but a placebo, to see how their outcomes compare with those who are treated.
"I value the broad exposure ASCO affords my specialty throughout the oncology world.We welcome you to submit a topic via our online form.View our current list of guidelines and updates in development. Public comments are being accepted through mid-March regarding the draft, titled “Rare Diseases: Common Issues in Drug Development,” and subtitled “Guidance for Industry.” Released on Jan.
Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how disease biomarkers might be identified and used.Your access to the NCBI website at gov has been temporarily blocked due to a possible misuse/abuse situation involving your site.