Closing the ‘Valley of Death’: Pioneering – and paying for – new therapies - Children's National

With the advent of life-changing cell and gene therapies (CGTs) to treat pediatric diseases, price is becoming a significant obstacle to care and cures.

Successful therapies can wind up shelved or in hard-to-reach clinical trials for a litany of reasons: high manufacturing costs, significant regulatory burdens, a lack of enthusiasm from the pharmaceutical industry in the small pediatric market and the simple fact that insurance companies resist paying the price of $1 million or more for a therapeutic. When successful treatments are set aside and become victims of this market failure, leaders in pediatric medicine say the drug has been relegated to the "Valley of Death."

Experts at Children's National Hospital and other leading U.S. research institutions are working to ferry drugs across it.

A new way forward

The Access4Kids think tank is bringing together healthcare leaders to chart another way - with its hosts Catherine Bollard, M.D., M.B.Ch.B., director of the Children's National Center for Cancer and Immunology Research, Crystal Mackall, M.D., director of the Stanford Center for Cancer Cell Therapy, Julie Park, M.D., Oncology Department chair at St. Jude Children's Research Hospital, and Alan Wayne, M.D., pediatrician-in-chief at Children's Hospital Los Angeles. Organized at the Children's National Research & Innovation Campus, the think tank will include voices from Seattle Children's Hospital in Washington state and the Moonshot Presidential Office in Washington, D.C., along with experts from academic institutions, the federal government and patient advocacy groups.

They explained their vision in their recent Nature Medicine perspective, "Enhancing pediatric access to cell and gene therapies." One idea under consideration is to create a biotech enterprise - backed by public monies, foundations and philanthropies - to support the late-stage development and commercialization of pediatric CGTs. Called a Pediatric Advanced Medicines Biotech (PAMB), this new organization would follow a novel pathway outside the traditional biopharma development model.

"To cure brain tumors, sickle cell disease and scores of other illnesses that have an outsized impact on pediatric patients, we will need an innovative mechanism to ensure that money is not a barrier to scientific advancement and world-class care," Dr. Bollard said. "My colleagues and I aren't exaggerating when we say this is a 'save the world' effort. It's an initiative to disrupt the pharmaceutical industry and impact medicine, ultimately saving lives and entire lifetimes."

The think tank is the second in a series aimed at solving this formidable challenge. Led by scientists, the work goes to the heart of healthcare economics.

"We need to find a way to support the academic ecosystem by reducing costs, creating efficiencies in manufacturing and working with regulatory bodies to bolster business models that lead to safe and effective therapeutics," Dr. Mackall said. "We went to school to study science and medicine. Now, our mission includes revamping business models, as we find ways to increase access to lifesaving treatments for children in need."

Why it matters

Creating novel pathways to pay for CGTs presents a challenge and an opportunity for researchers in pediatrics and rare diseases. Drs. Mackall, Bollard and their colleagues are exasperated when scientists discover successful treatments for life-threatening illnesses, but market forces lock the drug away in hard-to-reach clinical trials or - even worse - land it on a shelf.

Consider just one example, laid out in the Nature Medicine perspective: Children with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) lack nearly all protection from viruses, bacteria and fungi, leaving them vulnerable to a world of opportunistic organisms. Researchers at San Raffaele Telethon Institute for Gene Therapy in Italy won U.S. and European regulatory approvals for a gene replacement therapy to treat the life-threatening disorder. Yet currently the therapy is only available in clinical trials, with support from research grants, philanthropic donations and institutional resources.

"This happens to successful therapeutics, and it's heartbreaking. The current business model in medicine can lead to situations where successful therapies have extremely restricted availability, and there are no built-in guarantees of sustained access," Dr. Bollard said. "We can and must do better - and we believe we have a way forward."

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