See the DrugPatentWatch profile for Dornase
What was the Dornase alfa expanded access program?
Dornase alfa’s expanded access program was an FDA pathway that let doctors request access to the drug for patients who could not take part in a clinical trial and who had a serious or life-threatening condition, with no comparable or satisfactory alternatives available. The program is documented in FDA records and request-specific summaries through the expanded access mechanism.[1]
Who could get Dornase alfa through expanded access?
Expanded access requests are typically approved case-by-case for individual patients or small groups, when a treating clinician believes the patient may benefit and standard therapies are not available or are not suitable. The structure and eligibility depend on the specific FDA approval and the patient’s situation described in the request.[1]
How long do expanded access programs run?
Timelines vary by request type and regulatory approval. Some expanded access approvals are granted for a defined period or number of patients, and they may end when the request’s purpose is satisfied, a clinical trial becomes available, or FDA authorizations are updated or withdrawn.[1]
Does the expanded access program relate to cystic fibrosis or other indications?
Dornase alfa is most commonly associated with cystic fibrosis in FDA and clinical contexts, and expanded access is generally tied to the condition described in the individual FDA request. The specific targeted indication(s) and patient profile depend on what the requesting physician listed in that FDA expanded access submission.[1]
Where can I find FDA documentation for this program?
FDA’s expanded access database (and related FDA posting/records) is where details are recorded for each expanded access approval, including the program’s status and request information.[1]
Sources
[1] https://www.fda.gov/about-fda/expanded-access/expanded-access-submissions