Stories about: FDA

Lack of drug testing in kids exposes them to off-label harm

drug testing in children
Loopholes in current legislation let drug companies defer testing their products in children, so the majority of prescriptions are off-label.

Florence T. Bourgeois MD, MPH, is assistant professor of Pediatrics and Emergency Medicine at Harvard Medical School, faculty in the Computational Health Informatics Program, and Scientific Director of the Biobank for Health Discovery at Boston Children’s Hospital

Every day, more than half of children seen in outpatient clinics are prescribed a medication that is not FDA approved for the child’s age or diagnosis. Such off-label prescribing is widespread across pediatric conditions and treatment settings and as many as 90 percent of pediatricians have knowingly prescribed off-label medications.

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A hole in the FDA’s approval process for pediatric drugs

ADHD long term drug safety rare adverse events FDA approval Kenneth Mandl Florence BourgeoisYou’d think drugs meant to be taken by children for years would be studied in children for a long time to measure their long-term safety.

You’d think drugs for a condition affecting millions of children would be tested in many, many children to catch any rare side effects.

You’d think all this would happen before the Food and Drug Administration, an agency known for its strict criteria, approved them for marketing.

But if a new PLoS ONE paper by Boston Children’s Hospital’s Florence Bourgeois, MD, MPH, and Kenneth Mandl, MD, MPH, is any indication, you’d be wrong.

In it, the pair reports that the FDA approved 20 attention deficit hyperactivity disorder (ADHD) drugs over the last 60 years without what would be considered sufficient long-term safety and rare adverse event data.

Their findings, they say, point to larger issues in how the FDA’s approval process addresses the long-term safety of drugs intended for chronic use in children.

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Direct-to-consumer genetic testing: a case of potential harm

direct-to-consumer genetic testingLast November, the U.S. Food and Drug Administration issued a “cease and desist” order to 23andMe, a major purveyor of direct-to-consumer (DTC) genetic testing. In its letter to the company—issued after three prior warnings—the FDA reiterated its view that 23andMe’s Personal Genome Service (PGS) constitutes a medical device requiring further premarket evaluation:

FDA is concerned about the public health consequences of inaccurate results from the PGS device…we still do not have any assurance that the firm has analytically or clinically validated the PGS for its intended uses.

The FDA’s order, based on potential rather than actual medical harm, has generated a great deal of controversy. In a recent critique published in Nature, Robert Green, MD, MPH, of the Partners HealthCare Center for Personalized Genetic Medicine, and Nita Farahany, PhD, JD, of the Duke Institute for Genome Sciences and Policy, argued against regulating DTC genomic interpretation services as medical devices:

… doing so could put FDA regulations in greater tension with the First Amendment of the US Constitution, which protects the rights of individuals to receive information, and of ‘commercial speech’ ….the agency should avoid restricting consumer genomic testing unless faced with empirical evidence of harm.

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FDA gets serious about improving medical device innovation for kids

FDA entrance-ShutterstockAssisted by a congressional mandate, the FDA has taken a new approach to helping clinical innovators overcome barriers to moving pediatric medical devices from the research stage to commercialization. So says Linda Ulrich, MD, director of the Pediatric Device Consortia Grant Program at the FDA’s Office of Orphan Products Development, who recently spoke at an Innovators’ Forum cosponsored by her office and Boston Children’s Hospital’s Innovation Acceleration Program.

Regulation and reimbursement are the largest barriers to medical device innovation. “The time it takes to develop a medical device and get it to the U.S. market can take a range from 18 months to 10 years,” says Ulrich.

The stages of device development are concept, prototype, preclinical and clinical testing, manufacturing and marketing—only for the device to become obsolete within as little as 18 months after commercialization. Inventors need to consider the return on investment after their products receive regulatory approval, given the time and funding it takes to get them to market, says Ulrich.

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The ever-changing, interwoven regulatory and drug-pricing landscape

Tangled roots
Drug approval is increasingly intertwined with pricing questions.

At last month’s BioPharm America conference, what I originally thought would be a run-of-the-mill panel wound up being a frank discussion about regulatory and pricing challenges that pharma and biotech companies are facing today. I hadn’t realized these two challenges are intertwined so closely.

The regulatory and pricing paths for new drugs in the United States have become increasingly difficult to navigate. Due to outside policy pressures, the FDA is scrutinizing drugs more than in the past, requiring much more data. Even when a drug is approved, there is no guarantee that payers will cover its full cost, as they are starting to consider the drug’s overall value—improving quality of life and decreasing costs—along with its effectiveness.

Meanwhile, in many European single-payer countries, pharmaceutical companies are being told how to price their drugs before they are considered for approval by the regulatory agencies. The likely effect is less return on investment on new drugs, which could in turn decrease the pace of innovation.

Vaughn Kailian, managing director of MPM Capital, a health care venture capital investment firm, led an eye-opening conversation around these topics.

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Guiding devices to market, and mending broken hearts

A biodegradable patch for repairing ventral septal defects (VSDs).

Imagine: You’re a pediatric cardiologist who for years has worked on the design of a device that could revolutionize the treatment of a severe atrial arrhythmia. But while you can find a lot of assistance and advice for bringing devices for adults to market, you find little help for devices intended for infants and children. What can you do?

The U.S. Food and Drug Administration could be your best friend. Better known for its role in establishing and enforcing regulations for drug and device safety and information, the FDA is also an advocate, helping bring innovative devices for pediatric treatment into clinical practice. Pedro del Nido, chief of cardiac surgery at Children’s Hospital Boston, outlined the FDA’s advocacy role last week at the monthly Innovators’ Forum hosted by the Children’s Innovation Acceleration Program.

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