Dying Patients Face Reality Check on Right to Try

(Bloomberg) -- Cancer patients who have run out of options are the largest group that could benefit from the new Right to Try law intended to ease access to experimental drugs, thanks to the more than 1,000 oncology medicines in development in the U.S.

But don’t expect the floodgates to open.

That’s the consensus that emerged from more than two dozen interviews at the recent American Society of Clinical Oncology meeting in Chicago, the biggest cancer gathering in the world. Bloomberg talked to executives at pharmaceutical giants from Merck & Co. to Johnson & Johnson; small biotechnology companies developing what could become the hottest new treatments; and doctors at the forefront of oncology research.

Merck, which has the blockbuster cancer drug Keytruda and a pipeline of experimental products, typified the industry’s viewpoint -- that an existing system to provide desperate patients access to experimental drugs works well enough. The drugmaker was one of the big winners at the Chicago meeting, presenting data showing Keytruda prolonged the lives of patients with advanced lung cancer longer than chemotherapy.

“The best form of access is an approved drug,” said Roy Baynes, senior vice president of global clinical development at Merck Research Laboratories. But before a treatment is on the market, “there are ways, particularly if there is unmet medical need and there is nothing in the space, there are already mechanisms that can be activated.”

A key component of Right to Try is to allow dying patients who ran out of options, through their doctors, to directly ask drugmakers for access to medicines under development, eliminating the Food and Drug Administration from the process. The goal was to remove a hurdle, although the FDA said that it has approved more than 99 percent of applications under its expanded access program.

“I’m not sure it really changes things for patients,” said Joanne Lager, head of oncology development at French drugmaker Sanofi. “I haven’t heard of any cases where a patient wants a drug, the company wants to give it and the FDA has said no.”

At the Chicago conference, some doctors expressed concern that desperate patients may be taken advantage of by unscrupulous groups or clinics that could experiment without oversight from the FDA.

Giuseppe Del Priore, chief medical officer at Tyme Technologies Inc., agreed that removing the regulators from the equation could be risky.

“People might be tempted to circumvent them, it could be dangerous,” said Del Priore, whose small biotech firm is working on its first cancer drug and has an existing process in place to provide the treatment for compassionate use.

No Change

None of the companies interviewed had plans to change their current approach. Among those that already have a compassionate-use program include large corporations such as Roche Holding AG, Novartis AG, Amgen Inc., AstraZeneca Plc, Celgene Corp., as well as smaller biotechs like Nektar Therapeutics.

Most of the drugmakers intend to keep the FDA in the loop anyway, because regulators may have information about a class of drugs that’s not available to a single company. The strongest advocate of maintaining close ties with the agency was New Brunswick, New Jersey-based Johnson & Johnson, which was the first company to put in place a novel pre-approval access program run by outside experts in ethics and medicine.

“We have to understand if the drug is harming patients,” said Craig Tendler, vice president of late-stage development, global medical affairs for oncology and hematology at J&J. “That’s the greatest concern here.”

Most Sanguine

Bayer AG, which sells the kidney cancer drug Nexavar and recently signed a partnership with Loxo Oncology for one of the most-promising new medicines in development, was the most sanguine about Right to Try, and the ability to make decisions on who may get their compounds without the FDA’s input.

“We welcome the approach,” said Dieter Weinand, head of the German company’s pharmaceutical unit. “To us, we just need to look to see if there is anything we need to do in our current program to further enhance or expand it.”

While Right to Try covers medicines for any condition, oncology treatments are likely to see the greatest demand, because a majority of terminally ill patients have cancer.

Health advocacy groups, like the American Cancer Society and the American Society of Clinical Oncology, are concerned about the implications for doctors and patients. Fewer than 4 percent of drugs that emerge from the first stages of clinical development are successful. Some fail because they aren’t effective, but others hurt the patients they are trying to help.

“I’m worried the patient will die sooner and die in greater agony,” said Otis Brawley, chief medical officer of the American Cancer Society.

©2018 Bloomberg L.P.