Stories about: Ethics and policy

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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Infant behavior, dyslexia and war orphans: A portrait of Peter Wolff, MD

Peter Wolff MD
Peter Wolff c. 1977 (Ed Fitzgerald/Boston Globe)

Peter Wolff, MD, recently retired from Boston Children’s Hospital after more than 60 years in service to clinical psychiatry, behavioral science research and ethical oversight of human subject research.

When he started as a psychiatrist at Boston Children’s Hospital in 1956, Peter H. Wolff, MD, was seeking a deeper understanding of infant behavior. At a time when psychoanalysis was the framework for understanding the infant psyche, Wolff applied scientific methods used to study animal behavior — carefully observing an animal in its natural environment and seeking to discern patterns. His approach would revolutionize our understanding of infancy.

“We knew a great deal about a stickleback fish, the graylag goose by just watching what they do in the field — field observations — but nobody had ever done that with humans,” Wolff shared in an interview in 2009, “and it seemed to me a logical thing to try to do that.”

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Why we’re glad the Cures Act passed: Its provisions benefit children

Cures Act children
Via @POTUS

Amy Judge DeLong is manager of Federal Government Relations at Boston Children’s Hospital.

In the midst of a seismic shift in Presidential administrations and anticipation of the incoming Congress, a landmark medical research bill with several provisions important to children cleared the lame duck session of Congress. The 21st Century Cures Act (Cures) is the end result of nearly three years of bipartisan Congressional activity. Last week, it was signed into law.

Cures includes scores of provisions aimed at strengthening National Institutes of Health funding for medical research and accelerating review efforts at the Food and Drug Administration. The law cleared Congress with overwhelming majorities, an example of bipartisanship that may be challenged in the months ahead.

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Parents are generally open to placebo use in children – with caveats

placebo use in children

Placebos are a key ingredient of any controlled clinical trial, the yardstick against which experimental drugs are measured. Placebos are also increasingly used as a treatment in their own right, as studies show that they make people feel better through a “mind-body” effect. But do parents find placebos acceptable for their children? A study published today by The Journal of Pediatricsled by Boston Children’s Hospital, found the answer is mostly yes, provided ethical guidelines are followed.

“The question of placebos is more complex when it comes to children, since parents must make medical decisions on their behalf,” says Vanda Faria, PhD, a research fellow at Boston Children’s Hospital’s Center for Pain and the Brain and first author on the study. “Large placebo responses have been seen in a variety of pediatric conditions, and parent’s perceptions can influence how well placebos work. At the same time, little is still known about the potential harms of prolonged drug therapy on children’s development. Sometimes, the best intervention might not involve pharmacotherapy.”

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If I have the mutation, will I get the disease? New research looks at genetic ‘penetrance’

genetic penetranceRecently announced preliminary results of the BabySeq study included pathogenic or “likely pathogenic” variants linked to heart conditions in three apparently healthy babies. Two are being followed at Boston Children’s Hospital and have had cardiac testing. But is this testing necessary, and are these infants truly at risk? It’s too soon to tell.

Then, last week, a report from the Mayo Clinic raised an alarm about overzealous use of genetic testing in healthy individuals. After a 13-year-old boy died from a heart syndrome, about two dozen family members had genetic testing. All tested positive for variants in a gene linked to long-QT syndrome and were diagnosed with the disease. Yet none had cardiac symptoms, and only one had a positive EKG at any point — the boy’s brother, who had a defibrillator implanted. When the Mayo team reanalyzed the test results using a more up-to-date genetic database, they concluded the variant is harmless.

And this week, in Science Translational Medicine, researchers at Brigham and Women’s Hospital, Boston Children’s and Massachusetts General Hospital address the question: If people carry a genetic mutation linked to a condition, what are the chances they will develop that condition over time? As part of the genomes2people project, the researchers tested participants in two long-term population studies — the Framingham Heart Study and the Jackson Heart Study — for 56 genes representing 24 hereditary cancer and cardiac syndromes. They did not know the participants’ actual health status. As it turned out, carrying a mutation increased risk for the related disease 4.7-fold in African Americans and 6.4-fold in European Americans, who had longer follow-up. This was true regardless of family history.

Vector sat down with Nina Gold, MD, a senior resident in Pediatrics and Medical Genetics at Boston Children’s, for her perspective. Gold is a first author on this week’s report.

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News Notes: Research and innovation news

A few things that caught Vector’s eye during the week:

FDA drug evaluation staff: An unmet need

overflowing inbox at FDA

New laws or initiatives to expedite drug approvals, especially for orphan drugs, have prompted the Food and Drug Administration to create new positions to review the applications. Unfortunately, these slots are proving hard to fill. Kaiser Health News reports that the FDA has more than 700 job vacancies in its Center for Drug Evaluation and Research (CDER), which approves new drugs. The problem? Pharma pays more — “roughly twice as much as we can,” said CDER director Janet Woodcock at a recent rare-disease summit. The 21st Century Cures Act, still awaiting Senate approval, could help by allowing the FDA to offer higher salaries and relaxing postgraduate degree requirements for some positions.

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BabySeq: Early results of newborn genomic sequencing are mixed

BabySeq
While a previous study indicated parents were very interested in newborn sequencing, just 7 percent of those approached have enrolled in BabySeq so far.

It seems like a great idea. We all have our genomes sequenced at birth, and any findings that suggest a future medical problem are addressed with early interventions, optimizing our health and extending our lives. But are parents of newborns ready to embrace the vision? Yes and no, according to interim results of a first-of-its-kind randomized trial of newborn sequencing. Findings from what’s known as the BabySeq Project were presented last week at the American Society of Human Genetics (ASHG) 2016 Annual Meeting.

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The 21st Century Cures Act: Addressing unmet needs in children with rare disease

21st Century Cures Act and children
Among its other provisions, the Cures Act would advance implementation of the 2013 National Pediatric Research Network Act, boosting therapeutic development for rare childhood diseases.

Medical solutions often require countless hours of investigation, months of testing and monitoring, years of post-trial and market analysis and billions of dollars of investment — with no certainty of success.

Last year, after years of groundwork, the U.S. House of Representatives passed the 21st Century Cures Act. A companion measure is being developed in the Senate, and stakeholders are optimistic that agreement on a package — even a slimmed down bill — could happen this year.

While Congress has addressed research and medical product regulatory needs before, the Cures Act has been unique in its comprehensive approach, looking at all elements of the research spectrum — from basic discovery science to translational research to regulatory review. It would upgrade the National Institutes of Health’s research capabilities and update the Food and Drug Administration’s approval policies to get new drugs and devices to the clinic sooner.

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Expectation vs. reality: Rare disease parents’ mixed feelings about genetic research results

rare disease genetic resultsTypically, when you enroll in a study, it’s not with the expectation that you will receive results. In genomics studies, it’s becoming common to give families the option to get individual results — the newborn sequencing study, Baby Seq, is just one example — as an incentive to participate. Families of children with rare disease, especially undiagnosed illnesses, need no incentive: they’re desperate for answers.

But how do families actually feel once they get genetic results? We conducted interviews with nine rare disease parents (six mothers, three fathers) whose children were enrolled at the hospital’s Manton Center for Orphan Disease Research. What we found is more complexity than we expected.

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Science Seen: Poverty and accidental death, 1999-2012

Unintentional injuries are the fifth largest cause of death in the U.S., and these mortalities have been rising over the past decade. At the same time, more people are living in areas with high poverty levels. Are these trends connected?

An analysis published last week in PLOS One gives evidence that the answer is “yes.” Emergency medicine physician Eric Fleegler, MD, MPH, and colleagues crunched U.S. Census Bureau data against mortality data from the National Center for Health Statistics for 1999 to 2012 — providing resolution down to the county level.

Not only did fatalities increase in tandem with the level of poverty in a county, the study found, but this effect worsened over time, especially for deaths from certain causes such as poisonings, shown here. Read the details in our sister blog, Notes. (Interactive image: Erin Horan)

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