The ongoing debate surrounding terminally ill patients' access to experimental treatments is reaching a critical juncture in the United States, as discussions intensify about federalizing Right-to-Try (RTT) laws. Industry experts are now calling for pragmatic solutions that focus on patient needs while maintaining scientific rigor and regulatory oversight.
Reimagining Pre-Approval Access
Leading healthcare experts emphasize that the key to expanding treatment access lies in developing an impartial and independent system that satisfies all stakeholder demands within existing regulatory frameworks. The proposed solution centers on creating a pre-approval access platform that would serve physicians and patients with unmet medical needs, while ensuring proper safety oversight and adherence to clinical research principles.
"Rather than debating whether the FDA is the problem or the solution, it would be of greater benefit to focus on the patients, and address the issues with more pragmatism," argues Dr. James Shannon, former Chief Medical Officer of GlaxoSmithKline.
The Reimbursement Challenge
A significant barrier to pre-approval access programs in the United States has been identified as the lack of reimbursement from commercial and governmental payers. Drawing from experience across 17 countries in Europe and South America, experts note that reimbursement for both drug manufacturers and healthcare providers is crucial for program sustainability.
Dr. Ronald Brus, Founder and CEO of myTomorrows, points out that successful global examples demonstrate how incentives for all stakeholders can enhance access: "To keep pre-approval access from being only for a few, reimbursement needs to be built in to the system from the start."
Evolving Drug Development Paradigm
The current drug development ecosystem faces increasing challenges, with even safe and effective drugs struggling to gain regulatory approval and market access. Industry experts propose elevating the significance of Phase 2 drug development, supplemented by real-world data collection. This approach could address current system shortcomings while offering treatment options to more diverse patient groups.
Balancing Stakeholder Interests
FDA Commissioner Scott Gottlieb has emphasized that the critical issue lies in encouraging companies to make developmental drugs available through pre-approval access programs. This aligns with the experts' view that market-based solutions are essential for creating a sustainable system that benefits all stakeholders.
The proposed reforms aim to:
- Facilitate freedom of choice for individuals
- Accelerate access for broader populations
- Generate valuable real-world data
- Improve transparency
- Maintain adequate safety oversight
- Preserve the FDA's essential role
As the healthcare industry grapples with these challenges, experts emphasize that successful reform will require acknowledging current system flaws while creating clear incentives for company participation. The goal is to regulate compassionate use more effectively while increasing the number of patients who receive potentially beneficial treatments.
