If you’re a drug company developing novel treatment for a serious unsolved disease, you have already been asked: “What’s your strategy for pre-market access?” If you’re a health care provider, health system, or disease organization serving patients with life-threatening conditions, you’ve been asked something similar. Do you have a good answer?
The patients you serve (and their doctors) have a legitimate, near-term need for treatment options, and many -if not most- who are medically suited for exploratory use of the product will NOT meet the rigorous inclusion/exclusion criteria of a well-powered research study. So why not collaborate with stakeholders to open meaningfully sized “treatment use” trials for the patients who cannot take part in your research trials – for patients who are part of your target indication and who are medically suited for exploratory use of the investigational product -just as the research subjects are. There is no real controversy around this concept. Health authorities of every developed nation recognize some form of what we call “Expanded Access” in the U.S. The FDA has recognized and encouraged Expanded Access for over 30 years. But for too long, the pharmaceutical industry and their followers had relegated the prospect of well-designed “treatment-use” access programs to the realm of the impractical. Until recently.
Expanded Access conferences, for years, featured a worn list of the operational challenges of pre-approval access. Panel sessions tasked with discussing “how to succeed” with Expanded Access focused instead on “how to say no to patients and doctors”. Narratives about regulatory hazards and threats to trial enrollment were drawn up, exalted, and repeated for years despite their breathtaking lack of substantiation. Legions of pundits and ethicists joined the discussion decrying patients’ “therapeutic misconception” and risk of “false hope”, seemingly without talking to real patients who typically expressed a well-informed appetite for new, unproven treatment options. Taken as a whole, there was ample cover for anyone disinclined to answer the “how to succeed” question. But now, in part thanks to 21st Century Cures and Federal “Right to Try” legislation, the question has been raised again. And this time, people are listening.
Another signal of change is the growing recognition that pre-market access programs aren’t merely a matter of compassion. With streamlined business models and cost-effective data analytics, companies are recognizing the value of wide-ranging real-world clinical data that group Expanded Access can generate. Perhaps it’s worth going back to the “success” question and consider the goal of integrating meaningfully-sized Expanded Access programs into the overall drug development process in designs which strengthen -not hinder- the commercial prospects for the drug company.
Here are three facts to consider:
1) GROUP Expanded Access is still King in the United States: Despite recent actions to make single-patient “compassionate use” easier for patients and doctors to request, 90% of U.S. patients treated under Expanded Access have been part of intermediate size (up to ~150 patients) and large size (hundreds or thousands of patients) group programs. These are special clinical trials for “treatment use” in medically suited patients who cannot get into a drug’s research trial; and they can be very large in cases of life-threatening disease. In comparison to handling individual requests, protocol-driven group Expanded Access is typically more efficient for sponsor, health care provider, and FDA reviewers. And it is the intended channel for all but the most exceptional medical cases.
2) Data Capture is encouraged: Not all countries ask sponsors to learn from the patients treated in Expanded Access trials, but the U.S. does. FDA has actively promoted the value of outcomes data generated in well-designed early access programs. Case history from 30 years of US Expanded Access has revealed responder group discovery, resolution of pre-existing drug safety concerns, and real-world evidence of efficacy for use in support of regulatory and business decisions.
3) Cost recovery is permitted in most countries, including the U.S.: Sponsors are not expected to shoulder the financial burdens of Expanded Access. Through authorized cost recovery, drug companies can be compensated for the cost of supplying product and complying with regulatory requirements. In some countries, health ministries will reimburse the sponsor at a projected market price. In the U.S., where health systems are more fragmented, modern solutions allow patients and charitable support to cover some of these costs.
Thought leaders are now committed to a smarter clinical development landscape for new medicines in unsolved killer diseases – a landscape that recognizes the vast unmet need for options in the medical community, the alignment of interests between patients and industry, and how those interests may be propelled by modern Expanded Access strategies.
Since 2017, the annual Expanded Access Summit has served as the central forum for this discussion in the global pharmaceutical industry. Over 40 of the world’s top regulatory and clinical science experts have served on the Summit’s speaker faculty, and policymakers from FDA, NIH, ASCO, National Health Council, EUCOPE, and other institutes help ensure that the outcomes of the Summit have real impact in practice.
The 2020 Expanded Access Summit is scheduled for January 27-29, 2020 at the National Press Club in Washington DC. The innovators and the public are finally answering the real question of “How to Succeed” in this space.