NCHR Author

Decision on Rare-Disease Drug Sparked Major Controversy, Seen as Marking New Direction at FDA

MedPage Today, December 28, 2016. On April 26, an FDA advisory committee voted 7-6 that the exon-skipping drug eteplirsen for Duchenne muscular dystrophy (DMD) failed to meet the standards needed for accelerated approval. It was widely assumed that the FDA would tell the drug’s developer, Sarepta Therapeutics, to try again with better data. That, of course, did not happen. In this follow-up, we report on how it eventually did turn out for the drug and for the DMD community. The ripple effect of the eteplirsen decision could prove damaging to the healthcare system, Diana Zuckerman, PhD, president of the National Center for Health Research, told MedPage Today.”Many drug companies will be submitting applications that they wouldn’t have dreamed of submitting [before].”

Women’s Low Sexual Interest: NCHR Comment on FDA Guidance

December 23, 2016. There is a need to find treatments for low sexual interest, desire, and/or arousal in women, as those conditions affect women’s quality of life. However, the scientific standards should be solid, and consistent with long-term use, if that is the intent. If a sponsor does not want to conduct a large enough study to carefully evaluate safety and efficacy for all the known subgroups, the FDA should approve the drug only for those subpopulations that have been found to have benefits that outweigh the risks.

Target Ends Sale of Dangerous Baby Mattresses, as NJ Considers Statewide Ban

Tap into Morristown, December 19, 2016. Supplemental mattresses are mattresses sold individually, can be bought for use with play yards, and are advertised as safe. According to U.S. Consumer Product Safety Commission data from 2000 through 2013, at least 15 children died while sleeping on supplemental mattresses. These deaths involved a child being wedged between gaps created when the supplemental mattress was added to the play yard or portable crib. “The evidence is clear: These supplemental mattresses can kill children,” says Dr. Diana Zuckerman, Ph.D., President of the National Center for Health Research. “They should be banned. As someone who was born and raised in New Jersey, I’m proud to see my home state on the forefront of this important issue.”

Doctors Argue Leadless Pacemakers Deserve Broader Medicare Coverage

Modern Healthcare, December 20, 2016. Physicians want wider Medicare coverage for leadless pacemakers, arguing the current reimbursement proposal puts the device out of reach for too many patients. Experts agreed with the CMS that more research is needed. Evidence shows that while the leadless pacemaker eliminates some of the key drawbacks of existing pacemakers, they also increase the likelihood of some complications and may introduce new ones, according to Dr. Jay Ronquillo, director of biomedical informatics at the National Center for Health Research, a no-for-profit think tank focused on health research. “At this point, there is no reason for patients to feel cheated if Medicare doesn’t cover the cost of leadless pacemakers, because leadless pacemakers may be no better, and possibly worse, than pacemakers with leads,” Ronquillo said.

Potential FDA Head Could Change Drug Approval Process Dramatically

Big Think, December 17, 2016. President elect Donald Trump has had several controversial cabinet picks so far for his administration. But in the medical community many are especially concerned over the possible tapping of Jim O’Neill for head of the FDA. While one of the FDA’s main jobs is approving medications, O’Neill said in a speech in 2014 that medications should be evaluated for safety only, what he called “progressive approval.” President of the National Center for Health Research, Diana Zuckerman, told The Hill that such a move would toss insurance companies into limbo. Being unable to rest on FDA approval when deciding coverage, such a move would, throw the entire U.S. healthcare system into turmoil.”