The dying want easier access to trial drugs

Critics say doing so undermines efforts drug firms’ desire to protect their investments and a government’s duty to evaluate safety

ormer firefighter Mike DeBartoli is a man desperate to rescue himself. He suffers from amyotrophic lateral sclerosis, the degenerative nerve disorder better known as Lou Gehrig’s disease, which usually kills within five years. He has already spent one year in a clinical trial, taking four pills a day that may have been a placebo. It didn’t help.

DeBartoli walks with difficulty and falls frequently. He’s losing his ability to breathe on his own. Now the 55-year-old from Tracy, California, has pinned his hopes on an experimental drug made by Genentech — and a new “right-to-try” law that allows desperate patients to take medications before they’ve been fully vetted by the Food and Drug Administration (FDA).

At a time when agreement on any subject seems impossible, the movement to give sick people more access to potentially lifesaving drugs is a rarity. Right-to-try laws have been adopted in 33 states and are under consideration in the remaining 17, with support from across the political spectrum.

But for all its populist appeal, the push for rightto-try laws has raised the ire of ethicists, drug safety experts and a former FDA commissioner. They say the laws do virtually nothing to expand patients’ access to effective drugs.

DeBartoli doesn’t see it that way. Since California’s law went into effect on January 1, all he needs to get his hands on Genentech’s GDC-0134 is the company’s cooperation and the backing of two physicians.

Critics counter that the state measures undermine an FDA programme that balances patients’ need for options, drug companies’ desire to protect their investments, and the government’s duty to evaluate drug safety and effectiveness.

Under the right-to-try measures, a dying patient can seek access to an experimental drug if it is an active candidate for FDA approval and has successfully passed a Phase 1 clinical trial to explore its safety, at various doses, in healthy human volunteers. That standard falls well short of proof that a drug will work, or that it won’t have side effects that could harm a patient or increase the discomfort of his or her final days.Drugmakers are under no obligation to provide experimental medication.

Right-to-try laws offer patients an alternative to the compassionate-use, or expanded-access, programme the FDA has had in place for more than two decades. As many as 1,821 applications are filed each year, and about 99 per cent of them are approved, according to the agency. In more than 11,000 compassionate-use cases over the last decade, patient deaths have halted a drug’s progress only twice, he said. Both times the medications got back on track after a brief delay.The real reason companies aren’t eager to provide experimental drugs is that they “don’t want to get involved,” said New York University bioethicist Arthur Caplan. That problem won’t be fixed by right-to-try laws, he added.

Pharmaceutical companies are set up to get their products through the FDA’s rigorous approval process, not to get drugs to patients ahead of FDA approval, Caplan said. Many wouldn’t know what dose to recommend, let alone how they’d distribute them or how much they should charge. And for unscrupulous companies, the measure’s “completely vague” language on what drugmakers can charge provides an opening to fleece desperate patients, he added.

Moreover, most companies have limited quantities of these drugs on hand and no policies to allocate them among all who want them, Caplan said.

Finally, when a patient dies or suffers adverse effects from an experimental medication, companies are much less afraid of the FDA finding out than they are of investors getting wind of the setback.

Caplan knows this because in 2015, he and his colleagues at NYU’s Langone Medical Centre were asked by Janssen Biotech Inc to create a model of a “compassionate use advisory committee” for a multiple myeloma drug called daratumumab. It was the firstever effort of its kind, which underscores how poorly prepared drug companies are to handle right-to-try requests. (Daratumumab was approved in November and is now marketed as Darzalex.)

Caplan calls the right-to-try measures “feelgood” laws that don’t address the shortcomings of the FDA’s programme but undo its benefits.

Nor is it true that greater access to experimental drugs will do much to help patients such as DeBartoli, said Dr Adams Dudley, who directs the University of California, San Francisco’s Centre for Healthcare Value. The numbers just don’t add up. Melissa Healy is a health and science reporter with the Los Angeles Times.