Right to try and saving lives

Dr. James D. Veltmeyer

Special to Village News

Although it has drawn far less public attention than the battle over illegal immigration, trade wars, North Korea or Supreme Court appointments, the Trump administration is quietly initiating a revolution in health care. The administration’s more decentralized approach to this issue will result in better care at lower costs and many more lives saved.

The reforms at Department of Veterans Affairs are one example. Veterans are now being given a choice in doctors, allowing them to escape the inefficient bureaucracy to receive care from private physicians on a timely basis.

Likewise, the Department of Health and Human Services under President Donald Trump is granting waiver after waiver to the states to give Medicaid patients more choices in health care. Association health insurance is being encouraged by executive order, and the direct primary care model is looked on more favorably.

One other development is the president’s signature on the Right to Try Act of 2018.

Formally known as the “Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act of 2018,” this legislation amended existing federal law to permit certain unapproved, experimental drugs to be administered to terminally ill patients who have exhausted all other approved treatment options and are unable to participate in clinical drug trials. These drugs are not snake oil cures; they must have completed an FDA-approved Phase I clinical trial and be in an active clinical trial as well as being in ongoing active development or production.

For years before the passage of the Right to Try Act, thousands of Americans have been forced to go abroad in search of possible life-saving treatments for terminal illnesses. The Food and Drug Administration – which was created for the purpose of ensuring that only safe drugs and medications enter the U.S. market – has often been an obstacle in the way of fast-tracking life-saving medications. Like most government bureaucracies, the FDA has constructed an archaic complex of regulations and compliance costs that result in long delays in drug approval and massively increased research costs in health care.

The foot-dragging on the part of the FDA became even more serious after 1962, as a result of the Kefauver-Harris legislation which dramatically increased the agency’s power. Enacted as a result of some unfortunate tragedies associated mainly with two drugs, including thalidomide, these amendments led to a steep drop in the number of new drugs entering the market each year. The reason for the decline was due to the hugely expensive and restrictive regulations imposed on the pharmaceutical companies, causing research and development costs to soar.

Thanks to FDA regulations over the last half-century, the United States fell behind the rest of the world in gaining access to important medications. It is estimated that if they calculated the number of premature deaths caused by drug delays, deaths due to loss of innovation and deaths due to the FDA’s suppression of life-saving information, nearly half of the people who have died since 1962 had their lives shortened by 11 years.

Just the application process alone can take up to 100 hours, according to the FDA’s own estimates. If someone is dying, 100 hours is a lifetime and they can’t wait.

As a physician who faces life and death decisions every day and whose wife is battling stage IV breast cancer, I am angered by the FDA’s policies. It is one thing to prevent dangerous, adulterated drugs from flooding the American market, it is something else to prolong the approval process to the point that terminally ill patients actually die or are forced to go overseas where these drugs have existed safely for years. And, no one can say that the FDA has had a perfect record in protecting American consumers. In 1999, the agency approved a drug called Vioxx which killed 60,000 people and triggered 140,000 heart attacks. The agency has also looked the other way as cheap generics manufactured in highly questionable drug factories in China and India were permitted to enter the U.S. market.

With the Right to Try legislation, however, Congress and the Trump administration are making an end-run around the red-tape bureaucrats at the FDA and some fellow citizens are already benefiting.

Bloomberg News said in January that a California man diagnosed with glioblastoma, a type of brain cancer that is usually fatal within two years, had been granted access to a therapy called Gliovac, produced by a Belgian pharmaceutical. This individual is one of approximately two dozen or so people currently taking Gliovac. He is paying nothing for the drug, and there have been no side effects in the trial so far.

In another case, Matt Bellina – a former Navy pilot who suffers from ALS, also known as “Lou Gehrig’s disease” and one of the individuals the law was named for – has been receiving the experimental therapy NurOwn which is in phase III clinical trials.

Before Right to Try, the process for obtaining experimental drugs was so challenging that fewer than 1,000 people sought and received federal approval to take such drugs in 2013. That’s what led to similar legislation being enacted in 40 states over the last several years, with bipartisan support. Trump’s signature to the federal law has been a long overdue gift to the terminally ill.

Immunotherapies and other promising treatments are offering a realistic vision – in the near future – of a world without cancer, Alzheimer’s disease, Parkinson’s disease and others. It’s time for big government to get out of the way and let the scientists, researchers and innovators do what they do best – find ways to make people live longer, healthier and happier lives.

Dr. James Veltmeyer is a prominent La Jolla physician voted “Top Doctor” in San Diego County in 2012, 2014, 2016 and 2017. Veltmeyer can be reached at [email protected].