Ariad Pharmaceuticals Inc fka ARIA

[Paulieme]

A good read for BIO TECH investors! "Changes Brewing at the FDA for Orphan Drugs- Expected Changes
While some advocates asked for comprehensive formal guidance from FDA on orphan drug development, the National Organization for Rare Diseases requested an explicit written statement from FDA reaffirming its commitment to flexibly applying current drug development standards for orphan drug development. Clinical knowledge of these orphan diseases is constantly evolving, and the agency may approach drug development differently as more is revealed about the diseases.

The Institute of Medicine will produce a report tentatively titled “Accelerating Rare Disease Research and Orphan Product Development.” The report will include discussions and recommendations on discovery, drug development, FDA, the clinical trial process, insurance, Medicare/Medicaid, and devices. This is expected to put additional pressure on FDA to revise and revamp the orphan drug development process.

We think that FDA will make explicit statements in its new 2011 guidance re-affirming its right to flexibility and discretion in enforcing those drug development guidelines already in place. We also think the 2011 guideline may particularly focus on the acceptance and validation of disease-appropriate surrogate endpoints, biostatistical powering of clinical trials, and the validity of historical disease progression or disease registry data as comparators.
http://www.genengnews.com/analysis-and-insight/changes-brewing-at-the-fda-for-orphan-drugs/77899338/