FDA To Fast Track Certain Gene Therapies In Effort To Keep Pace With Fast-Growing Field
“For some of these products, there’s going to be some uncertainty, even at the time of approval,” FDA Commissioner Scott Gottlieb said. “But these products are initially being aimed at devastating diseases, many of which are fatal and lack available therapy. In these settings, we’ve traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies.”
Stat:
FDA Plans To Speed Path To Approval For Some Gene Therapies
The Food and Drug Administration will soon be alerting companies that certain gene therapies in development can qualify for less arduous review at the agency, Commissioner Scott Gottlieb said Tuesday. Specifically, gene therapies for hemophilia, a rare disease in which blood doesn’t clot properly because it lacks certain proteins, could be evaluated based on whether therapy increases those proteins in the blood, regardless of whether the therapy actually causes the patient to bleed less. (Swetlitz, 5/22)
In other public health news —
Stat:
The Drug Industry's Next Big Idea To Treat Alzheimer's Faces Fresh Doubts
After a generation of failures, the drug industry has pivoted to its next-best idea for treating Alzheimer’s disease. But a troubling string of clinical trial results suggests that the latest approach to the devastating disease might go the way of its forebears, continuing decades of frustration for patients and scientists. Over the past year, three clinical trials involving two similar medicines have been halted early, two because a drug was clearly not working and a third because the treatment came with worrying safety concerns. (Garde, 5/23)
The New York Times:
Ex-Valeant Executive Is Convicted Of Bilking Drugmaker In Kickback Scheme
A former executive at Valeant Pharmaceuticals International and the onetime head of a small mail-order pharmacy were convicted on Tuesday of using a secret kickback arrangement to defraud the drugmaker. A federal jury in Manhattan found Gary Tanner, the former Valeant executive, and Andrew Davenport, at one time the chief executive of Philidor Rx Services, guilty on all charges, including wire fraud and conspiracy to commit money laundering. (Thomas, 5/22)
CQ:
Senate Expected To Greenlight Veterans Health Care Overhaul
The Senate this week is expected to clear a bill revamping the nation’s health care system for veterans as the clock runs out on a crucial program allowing them to access private doctors. The bill (S 2372) would infuse the Department of Veterans Affairs Choice Program with an additional $5.2 billion ahead of an expected funding shortfall on May 31. The program, created in the wake of the VA wait list scandal in 2014, allows veterans to see private providers if they have to wait more than 30 days for an appointment or live more than 40 miles from a VA facility. (Clason, 5/23)
The Washington Post:
New Ebola Vaccine Faces Major Test In Congo Outbreak
Authorities in Congo began an ambitious campaign this week to use a pioneering Ebola vaccine to help stem a growing outbreak of the deadly virus. It’s the first widespread use of the therapeutic since a devastating 2014 epidemic in West Africa and represents a major strategic shift for public health. World Health Organization officials, criticized for their slow response four years ago, began vaccinating health workers in affected areas Monday and plan to vaccinate about 1,000 people in the next week. More than 7,500 doses have been sent to the Democratic Republic of Congo, and an additional 8,000 doses will be available in the coming days, according to WHO. (Sun, 5/22)
The Associated Press:
In The Addiction Battle, Is Forced Rehab The Solution?
The last thing Lizabeth Loud, a month from giving birth, wanted was to be forced into treatment for her heroin and prescription painkiller addiction. But her mother saw no other choice, and sought a judge's order to have her committed against her will. Three years later, Loud said her month in state prison, where Massachusetts sent civilly committed women until recent reforms, was the wake-up call she needed. (Marcelo, 5/23)
The Associated Press:
Many Cancer Patients Juggle Care Along With Financial Pain
Josephine Rizo survived chemotherapy, surgery and radiation, but breast cancer treatment wrecked her finances. Money was already tight when doctors told the Phoenix resident she had an aggressive form of the disease. Then she took a pay cut after going on disability leave, and eventually lost her job and insurance coverage. During treatment, Rizo got swamped with more than $50,000 in medical bills. "My concern was, 'Am I going to die?'" she said. "I had to kind of focus 100 percent on my health to make sure I was around for my kids." (5/22)