The FDA has a real role to play and should not be sidelined
On March 21, the US House of Representatives passed two Federal right-to-try bills (S.204, The Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try of 2017 and H.R. 878, the Right to Try Act of 2017) that would permit terminally ill patients to gain access to experimental medical treatments not yet granted formal approval by the health regulatory authorities.
Last year, the bills were passed by the Senate unanimously. At first sight, giving drugs to desperate terminally ill patients appears commendable and compassionate. But some bioethicists are not entirely convinced that a national right-to-try law will definitely make life better for these patients. On the contrary, this new law is arguably unnecessary and may even cause more risk than reward.
Proponents of the bill contend that the new law will eradicate obstacles hindering terminally ill patients from accessing medical treatments which are not yet approved by the Food and Drug Administration (FDA), the government regulatory body that manages the drug approval process. Before it could be used on people, a drug must undergo three phases of a clinical trial for it to be approved by the FDA. This is a complicated, stringent and time-consuming process which could take years. The proponents argue the bill will reduce drastically the time it requires for a drug to go through FDA.
The FDA’s drug approval process was introduced in 1962 after expecting mothers in Europe who were taking thalidomide gave birth to babies with birth defects. This tragedy drove the US Congress to pass legislation that would compel drug manufacturers to establish both safety and efficacy.
Compared to previous versions, the latest form of the legislation is an improved one that includes more rigorous informed consent procedures, more restricted eligibility, more frequent and comprehensive reporting to FDA and the requirement for manufacturers to provide reporting to the public.
However, a grave concern with these bills is that FDA would be taken out from the equation and this could open a Pandora’s box for possible harm to patients.
The new law would permit patients, their doctors and drug makers to leapfrog the FDA process and work directly with for-profit drug companies for access to unapproved drugs that have cleared the only phase 1 clinical trial, a preliminary safety testing. The FDA’s removal from the process will upend the established order for experimental drugs and undermine the clinical trial system.
The movement to supply investigational drugs to the terminally ill had its origins during the HIV epidemic in the 1990s when the HIV/ AIDS patients fought tooth and nail for access to experimental medications — as depicted in the movie ‘Dallas Buyers Club’. The right-to-try legislation in the brainchild of the Goldwater Institute, a libertarian think tank based in Phoenix, Arizona, which drafted a model legislation that 38 states in the USA so far have adopted to enact their right-to-try laws. The right-to-try movement gained momentum swiftly, often supported by libertarian-minded people who see it as a humanitarian effort.
Opponents, including International Society for Stem Cell Research (ISSCR), of the right-to-try legislation argue that the FDA currently already has a process in place, called expanded access programs/ ‘compassionate use’, for getting access to unapproved drugs for terminally ill patients who have exhausted every option.
Their doctors could lodge an Emergency Investigational New Drug application to the FDA. The agency approves 99.7% of the patient requests for expanded access that it receives while making essential dosing and safety improvement to proposed expanded use.
In reality, it is usually the pharmaceutical companies that deny access to investigational drugs for whatever reasons. In contrast, the new law would permit a seven-day interval between access to experimental drugs and FDA notification. It would also eliminate FDA’s consultation on various critical matters such as dosing, dosing schedule and route of administration which are offered under the agency’s expanded use program.
There was not much resistance and not many groups have taken a public stance on this matter. People will seldom articulate against a position which is framed as the terminally ill pursuing their right to try an experimental treatment, probably their very last chance, to save their lives.
Passing this legislation could also lead to a slippery slope to take in patients who are not terminally ill. Right-to-try activists in Texas are trying to extend the programme to chronically sick patients too!
The FDA is not the hindrance to patient access to investigational drugs. As an independent judge, it plays a crucial role in ensuring proper patient safeguards are in place. The committee should not pass legislation that would remove the FDA from the authorisation process for gaining access to an investigational therapy outside of a clinical trial. Without the federal oversight, there is a threat of abuse and even fraud at the expense of the patients who are already very ill.
Dr Patrick Foong is a law lecturer at Western Sydney University. His research interest lies in bioethics and health law.
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