FAQs
These questions and answers are more specifically related to clinical trials overall.
Why should biopharma engage with patient populations who cannot participate in a trial?
To ensure they are informed about potential future trials, expanded access programs, or other treatment options.
What is an example of a patient group that might be excluded from a clinical trial?
Non-ambulatory patients in a condition where both ambulatory and non-ambulatory populations exist.
How can biopharma address the needs of excluded patient populations?
By setting up expanded access programs or planning future trials that include them.
​What role does the FDA play in reviewing clinical trial data?
The FDA assesses whether the product is safe and effective and determines the approved label based on trial data.
How does the FDA decide on the scope of an approved label?
It can either limit the label to the studied population or broaden it if the data supports wider use. Although, a wider label harder to justify if no data exists on those populations
Can the FDA’s labeling decision affect patient access?
Yes, a narrow label can limit access, while a broader label can make the drug available to more patients.
How do insurance companies (payers) interpret FDA-approved labels?
They may restrict coverage to only the trial population, even if the FDA grants a broader label.
What are the key stakeholders in clinical trial design and drug access?
Biopharma, the FDA, insurance payers, and patient communities.
How does trial design influence patient access?
It directly affects who can get the drug, even after FDA approval, based on inclusion criteria and insurance coverage decisions.
Why is it important for biopharma to consider access beyond the clinical trial?
Because even after approval, labeling decisions and insurance policies determine which patients can actually receive treatment