When Dr. Stanley Dudrick died in 2020, his obituary noted that he had saved more than 10 million lives thanks to a groundbreaking treatment he developed in 1967. Today, countless other innovators are being prevented from delivering the next generation of life-saving medical innovations to patients due to regulatory policies that have not kept pace with modern medicine.
In the 1960s, Dudrick’s revolutionary treatment — total parenteral nutrition, or TPN — supplied vital nutrients intravenously to critically ill patients. By the time I started my surgical training in the mid-1980s, TPN had been in use for more than a decade and remains in use.
It’s an inspiring story — in less than a decade, the notion of lifesaving, intravenous nutritional support had gone from centuries-old dream to clinical reality.
It was during those same 1960s that Congress last mandated changes for the U.S. Food and Drug Administration to impose on the process required of pharmaceutical innovators who develop pioneering drugs. However, the clinical trial process mandated by Congress was designed for the development of drugs that treat relatively common diseases and conditions — those that affect a sizeable part of the population. Unfortunately, today’s most innovative treatments do not fit neatly into the clinical trials mold established in the 1960s. As a result, patients are being kept from potentially life-saving treatments.
We must modernize the drug approval process so that patients can get the care they need.
Nowhere would this be more effective than for rare disease patients. About 30 million Americans suffer rare diseases. Yet, fewer than 5 percent of rare diseases have an FDA-approved treatment. There are, however, cutting-edge, individualized treatments that could help.
These treatments are specific to an individual patient’s unique genetic profile. Because of their nature as individualized treatments, they are unable to meet stringent clinical trial requirements that medicines exhibit effectiveness across a broad pool of patients. Because of this regulatory mismatch, rare disease patients, many of whom have time-limited opportunities to reach recovery or remission, are prevented from accessing these treatments.
American patients are even being forced to travel to other countries to receive and benefit from innovative drugs and treatments unavailable here. Americans should not have to move overseas to save their lives — their care should be determined by their doctor, not outdated federal government policies.
The Right to Try Law of 2018 opened pathways for terminally ill patients to receive experimental treatments. Lawmakers should be expanding similar pathways for those suffering from rare diseases by passing Right to Try for Individualized Treatments laws, which allow patients with rare and ultra-rare diseases to access investigational individualized therapies if they have exhausted other options. Six states have already adopted such legislation, and congressional lawmakers should be working to advance similar reforms at the federal level.
Just as Dudrick’s TPN treatment saved millions of lives when it became widely available in the 1960s, making Right to Try for Individualized Treatments the law of the land can save untold Americans today. It would not only codify patients’ right to improve and preserve their own lives but allow other developers of medical innovation the opportunity to deliver future medical breakthroughs to a population that is too often overlooked.
