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Sally Atwater's doctor spent two months on phone calls, messages and paperwork to provide her with an experimental drug that she thought could combat the lung cancer that spread to her brain and her body. his spine.
Nancy Goodman asked eight companies to let her son experiment with experimental drugs against a brain tumor, which eventually killed him, and only three of them even explained why they had refused. .
Every year, thousands of patients with critically ill cancer seek "compassionate use" for treatments that are not yet available on the market, but have shown promise in early testing and are not accessible. through a study.
Now the government wants to make things easier and give more weight to requests. On Monday, at a cancer conference in Chicago, the Food and Drug Administration announced a project to make the agency an intermediary.
Instead of asking the doctors to plead their case first to the companies, then to the FDA, if the FDA agrees to provide the drug, the FDA will become the initial step and appoint a staff member to quickly take care of the paperwork. So, when a company receives a request, it knows that the FDA already considers it appropriate.
"We are here to help, we are not here to force a drug company to administer a specific drug to a patient, we do not have that authority," said Dr. Richard Pazdur, FDA manager. But the agency gets little information on the number of rejected applications and the reasons for these requests.
The current system is also cumbersome and sometimes unfair, he said. Patients in rural areas or city centers or community hospitals that are understaffed to meet specific demands may be at a disadvantage. Social media campaigns can exacerbate unfairness.
"We do not want the situation to be the one where someone who screams the loudest gets the drugs" and other deserving candidates, according to Pazdur.
The project only concerns cancer drugs, not other diseases. This has nothing to do with the federal law on the right to judge passed last year, which many have called "right to ask" because it only allows patients to request a drug from a company in certain circumstances and does not require it to be provided.
The new FDA project "will absolutely change things" and push more companies to say yes, Goodman said. She founded a advocacy group, Kids v Cancer, after the death of her son Jacob Froman in 2009 at the age of 10.
The FDA has not been the problem, she said. It maintains a website containing links to company policies and patient contact information. He quickly approved the vast majority of these requests when a company has authorized access to them.
That's what happened when Dr. Chul Kim, a pulmonologist at the Georgetown Lombardi Cancer Treatment Center, first tried to compassionately use a patient, in this case to help Atwater, whose cancer was spreading despite the usual treatments.
"I thought it was urgent to change treatment," and early results suggested that the experimental drug could enter the brain, which many treatments can not do, and fight cancer in this region, Kim said.
He started the process in early February and finally got the drug at Atwater at the end of March.
"I have other patients and I needed to save time for it," and I was fortunate to have staff working with the company on a study that could to help, said Kim. Once the company agreed, it only took one day for the FDA to do the same.
"It takes a bit of work," said Dr. Ajai Chari at the Mount Sinai Tisch Cancer Institute in New York, where dozens of patients have gained compassionate access over the past decade. "Many people have to give up what they do to make it all happen."
Chari just did it for Michael Walsh, a 58-year-old New York musician who has been suffering from myeloma since 2011.
"He has exhausted all the FDA-approved therapies," including 13 types of chemotherapy, said Chari. A few weeks after the start of the experimental treatment, Walsh had a dramatic reduction in her cancer.
"I work, I do things," said Walsh. "It gives me a bit of space for the cancer to be under control."
Atwater, the 68-year-old Washington woman treated in Georgetown, hopes her experimental drug will do the same. She said that she asked her sister with breast cancer how she would know if the treatment was working.
She said: "You're still here, is not it?" Said Atwater. "I think it's worth the risk, at least I hope that's the case."
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Marilynn Marchione can be followed at @MMarchioneAP
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The Health and Science Department of the Associated Press is receiving support from the Howard Hughes Medical Institute's Department of Science Education. The AP is solely responsible for all content.
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