F.D.A. Approves Muscular Dystrophy Drug That Patients Lobbied For

The New York Times, September 19, 2016 – The Food and Drug Administration approved the first drug to treat patients with the most common childhood form of muscular dystrophy. The agency’s approval went against the recommendation of FDA experts and the agency’s independent Advisory Committee. “The agency has set a dangerous precedent,” said Diana Zuckerman, president of the National Center for Health Research. Continue reading

NCHR Flu Vaccine Testimony

March 4, 2016 – We applaud the FDA and CDC for changing the recommendations for children, to reflect the poor efficacy of the live attenuated influenza vaccine (nasal spray). We hope the FDA will continue to look carefully at whether the agency should rescind approval of the flu nasal spray, since it continues to show significantly lower efficacy than the standard flu shot towards certain flu strains.

There is another problem however, that I want to talk about today. The CDC has strongly encouraged patients to use antiviral medications if they get the flu. However, the evidence shows how little benefit Tamiflu offers, as well as significant risks for children. Continue reading

NCHR testimony at 2016 FDA meeting on TOPAS treatment for fecal incontinence

February 25, 2016 – The lack of a comparison group or data for substantial groups of women most likely to suffer from fecal incontinence makes it impossible to determine if this device would benefit them. A control group is needed to determine if the device is effective for anyone, and if it is, it should be approved only for the types of women studied, not for the types of women who were intentionally excluded.

We encourage you to recommend that these studies be conducted before a decision is made about whether or not to approve this device. Continue reading

NCHR testimony at 2016 FDA meeting on leadless cardiac pacemakers

February 18, 2016 – Leadless pacemakers have the potential to improve the care of patients with various cardiac rhythm disorders. However, this will require an understanding of how acute adverse events compare and contrast with those of traditional pacemakers. I encourage you to urge the FDA to require comprehensive data regarding the intermediate and long-term safety and effectiveness of leadless pacemakers. Post approval studies should clearly assess these diverse factors. They should provide context regarding the entire spectrum of safety and effectiveness in a way that enables physicians to make sound decisions, and that allows patients to fully and clearly understand the risks and benefits of this new technology. Continue reading

Did FDA screw up its reporting of Essure deaths?

QMED, February 17, 2016. An independent analysis of FDA MAUDE data shows 303 fetal deaths related to the Bayer contraceptive medical device, but FDA itself has only reported five. A U.S. congressman is officially demanding answers while also alleging that some doctors implanting the device received illegal kickbacks. Continue reading

NCHR Testimony at 2016 FDA meeting on Allergy Products

January 21, 2016 – Allergies can be a life-and-death situation. Approximately 100 people die each year from anaphylaxis due to food allergies. Treatment options for food allergies could save lives, significantly improve quality of life for many families, and reduce the number of severe reactions from occurring. The increase in food allergy prevalence is an important public health problem. We must require that clinical trials study all of the necessary variables to help families make informed treatment choices. Continue reading

FDA faulted for failure to track safety issues with drugs already on market

STAT, January 14, 2016. Most Americans assume that drugs approved by the Food and Drug Administration are safe to take as directed. But safety concerns often arise only after the drugs go on the market, when companies or doctors tell the FDA about cases of patients who have fallen ill or died from their medications. Continue reading

Comments of members of the Patient, Consumer, and Public Health Coalition on “Drug Interactions with Hormonal Contraceptives: Public Health and Drug Development Implications”

As members of the Patient, Consumer, and Public Health Coalition, we support FDA’s effort to better characterize drug interactions with hormonal contraceptives since reliable and accurate information is necessary to ensure women’s health. In particular, we recommend clinical evaluation of drug interactions for all drugs that are likely to be used in women of reproductive age and that have the potential to cause birth defects, in addition to improving the quality and usefulness of information in FDA-approved labeling. Continue reading

Statement of Dr. Tracy Rupp at the December 14, 2015 FDA Advisory Committee Meeting on IMProved Reduction of Outcomes: Vytorin Efficacy International Trial (IMPROVE-IT)

December 14, 2015 – The very small chance of any potential benefit and the high cost of preventing one cardiovascular event lead us to conclude that ezetimibe is not the cure most patients desire. Continue reading

Statement of Dr. Tracy Rupp at the November 24, 2015 FDA Advisory Committee Meeting on Drisapersen for Duchenne Muscular Dystrophy

November 24, 2015 – Drisapersen is a drug being studied for Duchenne muscular dystrophy. However, a recent large, phase 3 clinical trial did not find that it was effective at helping patients walk further. Continue reading

What would impact of 21st Century Cures Act be on your healthcare costs and the lives of Alzheimer’s patients?

A controversial proposed new health law would have had a terrible impact on the lives of Alzheimer’s patients and their families, who would have spent billions on medications that don’t work and can cause cancer. Continue reading

The 21st Century Cures Act could be a harmful step backward

The Washington Post, November 19, 2015. Precision medicine is the next big thing in health care, and it’s also one of the few health goals that Congress and the White House agree on. But while we await treatments targeting the precise genetic makeups of individuals and diseases, medical researchers still are not paying enough attention to the most important kinds of differences among patients: those of sex, age and race. Continue reading

Statement of Dr. Tracy Rupp at the November 6, 2015 FDA Advisory Committee Meeting on Sugammadex

November 6, 2016 – Since sugammadex has the ability to quickly reverse the paralysis induced by neuromuscular blockers like rocuronium, it represents an innovative new option to improve surgical recovery. However, as with every new drug, we need to be certain the benefits outweigh the harms. Continue reading

Understanding the controversies over a groundbreaking new health care law

Milbank Quarterly, August 10, 2015. The House of Representatives did something surprising: They passed a groundbreaking health care law with strong bipartisan support. That sounds like good news. However, this groundbreaking health care law is almost as controversial as the previous one. Continue reading

Approved but not proven: what’s up with FDA, cancer drugs?

Medscape Medical News, October 22, 2015. In approving new cancer drugs, the US Food and Drug Administration (FDA) is now heavily relying on surrogate markers of effectiveness, such as tumor shrinkage, instead of proof that an agent improves survival, according to a new analysis. Continue reading

Would Washington’s FDA Fix Cure the Patients or the Drug Industry?

ProPublica, October 20, 2015. In Congress, however, things are looking better for the manufacturers. Legislation is advancing that would speed up the FDA’s approval process for medications and medical devices, offering a rare example of how major initiatives can get traction even in today’s gridlocked Washington. Continue reading

Bernie Sanders Opposes Califf for FDA Post Cites Industry Ties

Mad in America, October 16, 2015. Current presidential hopeful and longtime Vermont independent senator, Bernie Sanders, said that he will vote against Robert Califf’s nomination to lead the Food and Drug Administration (FDA). Sanders explained his opposition by citing Califf’s extensive ties to the pharmaceutical companies that he would be in charge of regulating. Continue reading

Theranos Limits Use of Blood Test Technology

Medscape Medical News, October 16, 2015. Theranos, the clinical lab company revolutionizing its industry with low-cost finger-prick tests, is limiting the use of its tiny blood vials to assays approved by the US Food and Drug Administration (FDA), company CEO Elizabeth Holmes said yesterday. Continue reading

How medical device companies test products out on patients—after they’re released

Pacific Standard, October 8, 2015. Federal scrutiny has been a long time coming for Essure: Since the device was approved nearly 13 years ago, thousands of women have filed complaints reporting punctured tubes, debilitating muscle fatigue, and severe pain. Continue reading

Medical panel calls for further study of Bayer sterility device

The Wall Street Journal, September 24, 2015. Thirteen years after the Food and Drug Administration approved a women’s sterility device called Essure, a panel of outside medical advisers Thursday called on the agency and device-maker Bayer AG to further study why so many women are suffering severe pain from the implant. Continue reading

F.D.A. panel weighs complaints on Essure contraceptive implant

The New York Times, September 24, 2015. A panel of experts convened by the Food and Drug Administration excoriated the manufacturer of a contraceptive device for not collecting data that they say could have helped predict risks for women. The device has received thousands of complaints from women who say they were harmed by it. Continue reading

FDA to establish patient advisory committee on medical devices

Star Tribune, September 19, 2015. The U.S. Food and Drug Administration on Friday announced the establishment of its first-ever patient advisory committee on medical devices. The committee, according to the FDA, is designed to inject a patient perspective into the agency’s deliberations on device development, approval and monitoring. Continue reading

Califf nomination for FDA chief gets mostly high marks

Medpage Today, September 16, 2015. The nomination of Robert Califf, MD, as FDA commissioner is getting good reviews from most health policy and cardiology experts. One area that might cause controversy with Califf’s nomination is his ties to the pharmaceutical industry. Continue reading

Lack of publicly available scientific evidence on the safety and effectiveness of implanted medical devices

The US Food and Drug Administration (FDA) approves about 400 implanted medical devices each year through an abbreviated process called the 510(k) process, which only rarely requires clinical trials (studies of patients). Continue reading

Is Early Stage Medical Device Development Going Overseas?

Wolters Kluwer Law & Health Blog, October 15, 2014. Even though the FDA has recently proposed a new program to provide earlier market access to high-risk medical devices, FDA Commissioner Margaret Hamburg nevertheless came under fire for the agency being too slow to approve medical devices at the annual conference of the Advanced Medical Technology Association (AdvaMed) in Chicago. Continue reading

Diversity in Clinical Trials

The Wall Street Journal, August 28, 2014. In response to a law passed two years ago, the FDA was directed to assess the extent to which women and minorities are represented in clinical trials and also devise a plan to bolster their participation. The requirement was made in response to concerns that drugs and devices may often be used by subsets of the population on whom the products were not actually tested. Continue reading

Avandia: what’s known and not known

Since heart disease is the #1 cause of death among diabetics, it is reasonable to ask why a diabetes drug that possibly increases rather than decreases the risk of heart disease should remain on the market. Are warnings enough to keep patients safe? After looking at the study results, many experts are urging that patients and doctors consider whether older drugs for diabetes may be safer and more effective–as well as much less expensive. They are asking: should Avandia remain on the market? Continue reading

Essure Study Criticized

NBC News, November 4, 2013. Dr. Diana Zuckerman, who leads the National Research Center for Women and Families, a non-partisan FDA watchdog, said the survey questionnaires exhibit dubious research methods. “These studies are always done by the company that is selling the product so it is not exactly unbiased,” Zuckerman said. Continue reading