Closer Look
Gene therapies are dying on the vine. What can we do to save them?
Molly Ferguson for STAT
Gene therapies are the saviors of people with rare diseases, right? Not really, not by a long stretch. While the tools of molecular biology have become so refined that some rare diseases succumb to bespoke treatments, the overwhelming majority do not, and not because the science isn't there yet. As a few dozen gene therapy researchers and a handful of advocates and federal officials detailed at a recent invite-only event in Washington, D.C., powerful therapies are dying on the vine for want of investment.
As gene therapies for sickle cell disease and Duchenne muscular dystrophy reach or come close to approval, they join very few others. "Seven out of 7,000," UCLA's Donald Kohn said, citing the fraction among total rare diseases. "We have a lot of work to do." Some ideas: a massive nonprofit biotech, lower regulatory standards, hospital-based CAR-T (like bone marrow transplants), perpetual clinical trials, Chipotle medicine. STAT's Jason Mast translates.
Health inequity
Marginalized patients are 'jumped over' for ER care
STAT's Usha Lee McFarling brings this report: Nearly 1 in 4 marginalized patients has experienced being "jumped over" while waiting for emergency care, according to an analysis of more than 300,000 emergency department visits conducted by a group of researchers at Yale. The study in JAMA Network Open found patients most likely to experience such "queue violations" — in which patients who were less sick or had arrived later were treated before them — were Black, Hispanic or Latino, or insured by Medicaid.
Patients who were treated later than they should have been were also more likely to be placed in hallway beds instead of regular beds, to leave before treatment was complete, and to return to the ED within 72 hours. The causes, the authors posit, include various forms of racism, from individual bias, such as an ED staffer trivializing the concerns of Black patients, to institutional bias, such as privately insured patients getting referred to ERs with detailed care plan notes from their primary physicians.
drug pricing
Opinion: Attacks on the drug industry would have blocked sickle cell treatment
Courtesy Ted Love
You may know Ted Love (in family photo above) as head of the trade group BIO and former CEO of Global Blood Therapeutics. You may not know he was born in Alabama in the Jim Crow South, where his family doctor inspired him to help people in need, Love writes in a STAT First Opinion. As a physician, he saw how medicines failed to benefit society's most vulnerable, particularly people of color. He shifted into biotech, where his company developed a daily pill to treat sickle cell disease.
Global Blood was later sold to Pfizer in a deal Love says the FTC might now block, given its new posture against mergers. "We could not have reached patients in lower-income countries … without being acquired by Pfizer," he writes. The Inflation Reduction Act, which allows Medicare to negotiate drug prices, also worries him. The combination "will ensure that more patients with overlooked or hard-to-treat conditions unnecessarily suffer for a longer time." Read more.
In the newest episode of Color Code, host Nicholas St. Fleur talks with former NIAID chief Anthony Fauci about why Covid-19 wasn't the "great equalizer" but instead placed a disproportionate burden on Black and brown communities. Listen here. 
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