CUPA Webinar
At MedaSystems, we believe in utilizing technology to expand hope. That’s why we’re paying close attention to the evolving landscape of pre-approval access, a critical pathway for patients ineligible for clinical trials to access new treatments.
A recent webinar hosted by Arizona Law’s Health Law & Policy Program and the Working Group on Compassionate Use and Pre-Approval Access (CUPA) highlighted just how vital expanded access programs have become. As experts discussed, these pathways allow patients, particularly those with rare diseases, to access potentially life-saving treatments when traditional routes are closed.
Key takeaways:
Expanded Access vs. Right to Try: Both allow access to unapproved drugs; however, expanded access requires FDA oversight and ethical review, whereas right to try removes these protections, raising concerns about patient safety and data quality.
Data Gaps: Unlike clinical trials, real-world evidence from pre-approval access often goes underutilized. At MedaSystems, we’re helping close this gap by building software that securely and ethically tracks outcomes, even outside traditional research settings.
The Rare Disease Lens: For the 1 in 10 Americans with rare diseases, pre-approval access may be the only pathway to treatment. These patients face barriers to clinical trials, ranging from strict eligibility criteria to geographic limitations, and they require better infrastructure to access and benefit from investigational drugs.
Pre-approval access isn’t just a policy issue; it’s a matter of equity, urgency, and innovation. We’re proud to support those on the front lines of compassionate use.
