New medicine for a rare disease; the continuing toll of the opioid crisis; views of a former ACIP voting member — Morning Medical Update

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New medicine for Barth syndrome

The U.S. Food and Drug Administration (FDA) has approved the first treatment for Barth syndrome, a disease that affects mitochondria, the energy producers in cells. Forzinity (elamipretide) is an injection against the disease, which is rare but potentially life-threatening. Patients with it who survive into adolescence and adulthood have significant effects on quality of life and daily functioning. “The FDA remains committed to facilitating the development of effective and safe therapies for rare diseases and will continue to work diligently to help ensure patients with rare diseases have access to innovative treatments," said George Tidmarsh, MD, PhD, director of FDA’s Center for Drug Evaluation and Research.

Long-term effects of the opioid epidemic

The opioid epidemic has taken a huge human and economic toll on the United States, and analysts still are calculating all its effects across health care. The National Bureau of Economic Research has published a trio of papers that examine:

• The effects of financial conditions on physician conduct for prescribing opioids.
• Potentially fewer drug deaths if opioid prescribing practices had not changed from 1995 to 2015.
• “Pill mill” laws and their effects on retail pharmacy sales and employment.

Ending universal recommendation for the COVID-19 vaccine

Heading into cold and flu season, vaccine policy, availability and effectiveness have become some of the hottest issues across health care. Kelly Moore, MD, MPH, is president and CEO of immunize.org, and is a former voting member of the Advisory Committee on Immunization Practices (ACIP). She discussed ACIP’s recent recommendation changes for the COVID-19 vaccine, which sparked intense debate in the immunization community.