The “right to try” experimental treatments is gaining ground in the United States

News 13 March, 2018
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    Tuesday, 13 march 2018, 21:36

    Tuesday, 13 march 2018, 21:36

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    In the Face of certain death, that to lose patients to try experimental drugs not yet authorised?

    This may be a child with a rare form of muscular dystrophy. A young woman plagued by a cancer of the colon. Or still a patient condemned by the incurable lou gehrig’s disease.

    The drugs already authorised, when they exist, are to them of no effect, and the hope of these patients is to participate in a clinical trial of experimental treatments by the laboratories. But either because they are too ill, too young, too old, or too far, they have not been selected.

    It is this population that desperate for the past four years, the elected representatives of the two parties, in 38 of the 50 american States, wanted to comfort them by adopting the laws on the “right to try” experimental treatments outside clinical trials.


    The cause is so popular that president Donald Trump and vice-president Mike Pence have put their weight behind a proposal for a law submitted to a vote of the House of representatives Tuesday in Washington, d.c. the goal of establishing a “right to try” national (the Senate passed a different version).

    But the elected democrats are opposed by a majority, thus preventing some of the text to reach the bar of two-thirds of the vote which was required under the accelerated procedure chosen by the republican majority. The majority leader has, however, promised to try again, and succeed.

    Doctors and associations of patients with rare diseases have denounced a law demagogic creator of “false hope” to patients, and that will not change anything in reality.

    Today, doctors can already ask the pharmaceutical companies to give an experimental drug to their patients by way of derogation, provided that the treatment has passed the first stage of clinical testing, that is to say, the phase contents in which the drug was tested on twenty individuals in good health to verify that it is not toxic.

    If the laboratories accept (there is a lack of figures on their response rate), the u.s. health authorities (FDA) approve the application in 99% of cases, usually in a few days, and recommending that sometimes adjustments.

    Each year, thousands of patients benefit from this access route “scope”.

    “It helps some, but it is rare. Most of the drugs in the research phase fail”, however, says to AFP professor Arthur Caplan, director of medical ethics at the school of medicine of New York university.

    The labs are reluctant

    The new law, in fact, would not be “law”: the laboratories will remain the only ones to decide to share or not their molecules.

    The difference, to the chagrin of opponents, including Arthur Caplan is a part, is that the FDA will have no further right to look and will simply be informed in the event of a severe negative effect.

    “They talk about the right to try, but we already have a right to try (…) people are clamoring already all the time to the laboratories,” notes Arthur Caplan.

    The national organization for rare diseases (NORD) is also opposed, fearing that the law creates “confusion” and “false hopes”.

    Not to mention that an experimental treatment may, according to Arthur Caplan, “make you die faster”.

    All agree, however, that companies are not doing enough.

    The small laboratories, engines of innovation, often do not have the means to manage individual requests for medicines in most of their clinical trials.

    As for the large companies, they are afraid that the death of a patient – very patient – does not create bad press titles, or barriers to the approval of the drug by the health authorities.

    The new law wants to encourage them to participate: it would give them legal immunity strengthened, and states that the FDA will not be able to block the placing on the market of a medicinal product because of the death of a patient outside clinical trials.

    “We want to give businesses a degree of clarity and assurance so that they can help someone in the context of the right to try, without risking to see their clinical trials suspended by the FDA, if a person dies”, explains to the AFP Starlee Coleman, of the Institute Goldwater, a think-tank libertarian in the origin of these laws.

    For evidence of malfunctions americans, she points out that the French system is more advanced for the uses of exceptional and overriding of drugs without the authorisation of the placing on the market.

    According to the latest report of the French Agency for the safety of the drug (MSNA), nearly 20,000 patients have benefited from an individual license in 2016 in France. Twenty times more than in the United States.