Patients deserve the ‘right to try’

Following the Senate’s unanimous passage of the “Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act” on August 3, the House passed a similar bill by a comfortable margin with bipartisan support on March 21. This legislation removes legal barriers that can prevent dying patients from gaining access to promising experimental medicines.

Although the vote on the Senate bill was unanimous — and despite narrower patient eligibility criteria — increased reporting requirements for pharmaceutical companies, and a greater role for the Food and Drug Administration (FDA) in the House version, Senate Minority Leader Chuck Schumer (D-N.Y.) inexplicably blocked the Senate from voting to enact the House bill.

{mosads}Next week representatives will have an opportunity to pass the Senate rendition. They should allow President Trump to sign it into law.

Thirty-eight states have passed “right to try” laws. The measures have generally achieved bipartisan support. Because FDA law trumps state laws, these legislative victories on behalf of terminally ill patients were largely symbolic. However, the right to try movement and its successes at the state level have almost certainly forced FDA to make its own “expanded access” program more responsive to patients who are at death’s door.

FDA’s expanded access regulations took effect in 2009. As recently as 2016, FDA required a physician to spend 100 hours filling out a form before she or he could try to help patients wanting to make a last-ditch effort to extend their lives.

However, FDA still considers such doctors researchers. The agency demands that they complete an hour or more of paperwork, and mandates that they obtain time consuming institutional review board approval before prescribing potentially life-extending drugs.

Recent former FDA commissioners appointed by both Democrat and Republican administrations have come out against right to try legislation arguing that it “would erode protections for vulnerable patients.”

Some critics contend the proposed laws are unnecessary because FDA approves most expanded access requests in a timely manner. Others point to the dietary supplement industry, Laetrile, Compound Q, and even “off-label” promotion as examples of exploitation of desperate patients who must be protected.

However, these individuals ignore the potential benefits and overstate the possible harms of right to try laws. Although FDA authorizes over 99 percent of expanded access requests, that figure is probably misleading because preliminary conversations with agency officials and associated administrative burdens discourage applications. The paucity of petitioners for FDA’s permission to try experimental drugs and the narratives described by patients or family members support this belief.

Further, the proposed legislation includes important patient protections. First, the drug must have completed initial safety testing. Second, physician participation and informed consent are mandatory. Third, opportunities for profit have been removed.

True, the number of patients likely to benefit from a federal right to try law is small. But this is of little matter to a patient who experiences a dramatic remission from an unapproved drug.

Successes with early access to experimental drugs have occurred. For example, as far back as the 1980s, patients with AIDS achieved substantial increases in longevity through the use of unapproved antiretroviral drugs. More recently, the lives of several patients infected with the Ebola virus may have been saved by administration of an investigational monoclonal antibody called ZMapp.

Off-label drug use is an essential and routine part of medical care. More relevant to the issue at hand, case reports of apparently effective, scientifically-reasoned uses of approved drugs abound.

My mother lived three years longer than predicted after treatment with an off-label drug before she succumbed to ovarian cancer. Importantly, she suffered far fewer side-effects than she would have experienced with standard, approved therapies.

All patients who improve while taking investigational drugs during clinical trials are benefitting from the use of unapproved drugs. But clinical trials are organized for research purposes, not to help individual patients. Is coercing anguished patients into becoming subjects of studies from which others will profit solely to obtain possible cures morally justified?

Newer precision therapies that harness a patient’s own immune system to kill cancer cells or are directed toward discrete genetic changes in patients’ tumors, increase the odds the drugs will work, and should present smaller risks of serious side effects.

Like other government bureaucracies, FDA is staffed with well-meaning but fallible human beings. The optimal way to ensure the protection we all desire is vigilance on the part of U.S. citizens and taxpayers and at times, competitive pressures such as those presented by right to try laws.

Dr. Roger D. Klein is a member of the Regulatory Transparency Project on health care. He is a former adviser to the FDA and HHS. Roger graduated from Yale Law School and completed his post-graduate medical training at Yale Medical School.Dr. Roger D. Klein is a member of the Regulatory Transparency Project on health care. He is a former adviser to the FDA and HHS. Roger graduated from Yale Law School and completed his post-graduate medical training at Yale Medical School.