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Expanding Horizons: How Every Cure Plans to Increase Rare Disease Drug Access While Reducing Trial Costs

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David Fajgenbaum, MD, MBA, MSc, cofounder of Every Cure, shared that the organization is able to reduce clinical trial costs to $1 to $5 million per drug trial, and that clinical trials are not always necessary to expand a drug's indication.

In this installment of the "Expanding Horizons" series, David Fajgenbaum, MD, MBA, MSc, cofounder of Every Cure and physician scientist at the University of Pennsylvania, explains the logistics of expanding rare disease drug access and financial considerations in clinical trials.

Fajgenbaum expresses excitement about significantly reducing the cost of trials for repurposed drugs to between $1 and $5 million, in contrast to the substantial amounts spent by drug companies on developing new drugs. According to research published in JAMA Health Forum, the National Institutes of Health spent an average of $33.8 million per FDA-approved drug between 2010 and 2019, including $13.9 million per drug for phase 1 trials, $22.2 million per drug for phase 2 trials, and $12.9 million per drug for phase 3 trials. The cost reductions by Every Cure are seen as a key element in making drug repurposing more feasible.

He also addresses insurance challenges, including the issue of setting different prices for the same drug with different indications, and discusses ongoing conversations with insurers. The goal is to pick trials strategically, where successful outcomes would economically make sense for insurers to cover the drug.

“It's all coming together right now,” Fajgenbaum said. “Frankly, we should have been doing this for a long time, but there's no time that's better than now.”

Check out part 1 and part 2 of the "Expanding Horizons" feature series. To learn more about Every Cure's work toward increasing rare disease treatments, visit www.everycure.org

Reference

Zhou EW, Jackson MJ, Ledley FD. Spending on phased clinical development of approved drugs by the US National Institutes of Health compared with industry. JAMA Health Forum. 2023;4(7):e231921. doi:10.1001/jamahealthforum.2023.1921

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