Grandfather Pushes for Rare Disease Trial Access Reform

When Theron Odlaug's granddaughter Anna developed Dravet syndrome, a rare catastrophic epilepsy, he discovered that hope for families like his often comes in the form of clinical trials. But those trials frequently exclude the patients who need them most.

Anna has endured hundreds of seizures despite taking multiple medications. When her family tried to wean her off one drug so she could qualify for a promising gene therapy trial, her seizures worsened. Now she's caught in a paradox: the medication that helps her also disqualifies her from the trial, and the company won't provide compassionate use access.

Odlaug, who spent over 40 years in healthcare leadership, knows the system well. He understands why small biotech companies often decline compassionate use requests, even though the FDA approves more than 99 percent of them within days.

Companies worry about risks to ongoing trials, adverse events complicating regulatory reviews, and limited manufacturing capacity. These concerns are legitimate, but they create a heartbreaking reality: children with the greatest unmet needs are least likely to access promising therapies.

For children with severe neurological disorders, waiting isn't neutral. Ongoing seizures and developmental regression are cumulative and often irreversible.

The Bright Side

Rather than pushing mandates that could slow innovation, Odlaug proposes incentives that would encourage compassionate use without coercing companies. His ideas include priority review extensions for sponsors who implement structured expanded access programs, liability safe harbors clarifying that adverse events won't count against regulatory approval, and tax credits to offset manufacturing costs.

None of these measures would lower FDA approval standards or force companies to act against their interests. They would simply create a culture where compassion and innovation coexist.

The proposals have gained attention because they acknowledge business realities while prioritizing patient needs. Public recognition programs could highlight companies that responsibly offer expanded access as ethical leaders in rare disease drug development.

Rare disease drug development depends on public trust from caregivers, clinicians, and advocates who participate in trials and champion innovation. When families see that promising therapies exist but remain categorically unavailable to those who don't fit trial protocols, that trust erodes.

Odlaug's advocacy represents a new model: solutions designed by someone who understands both the business constraints and the devastating human cost of inaction. His work could open doors for countless families navigating the impossible space between standard care and experimentation, ensuring that the children left behind by trial eligibility criteria aren't forgotten.