U.S. aims to help more cancer patients try experimental drugs
Sally Atwater’s doctor spent two months on calls, messages, and paperwork to get her an experimental drug he thinks can fight the lung cancer that has spread to her brain and spine.
Nancy Goodman begged eight companies to let her young son try experimental medicines for a brain tumor that ultimately killed him, and “only three of the companies even gave me a reason why they declined,” she said.
Thousands of gravely ill cancer patients each year seek “compassionate use” access to treatments that are not yet on the market but have shown some promise in early testing and aren’t available to them through a study.
Now the government wants to make this easier and give more heft to the requests. On Monday at a cancer conference in Chicago, the Food and Drug Administration announced a project to have the agency become the middleman.
Instead of making doctors plead their case first to companies and then to the FDA if the company agrees to provide the drug, the FDA will become the initial step and will assign a staffer to quickly do the paperwork. That way, when a company gets a request, it knows the FDA already considers it appropriate.
“We are here to help. We are not here to make a drug company give a specific drug to a patient. We don’t have that authority,” said Dr. Richard Pazdur, the FDA official leading the effort. But the agency gets little information now on how many requests are turned down and why.
The current system also is cumbersome and sometimes unfair, he said. Patients in rural or inner city areas or at community hospitals that lack the staff to work on special requests may be disadvantaged. Social media campaigns can add to the inequity.
“We do not want to have the situation where somebody who screams loudest gets the drug” and other worthy candidates don’t, Pazdur said.
The project only involves drugs for cancer, not other diseases. It has nothing to do with the federal Right to Try law passed last year, which many have called “right to ask” because it only allows patients to request a drug from a company under certain circumstances and does not mandate that it be provided.
The new FDA project is “absolutely going to change things” and push more companies to say yes, Goodman said. She founded an advocacy group, Kids v Cancer after her son Jacob Froman died in 2009 at age 10.
The FDA has not been the problem, she said. It keeps a website with links to companies’ policies and contact information for patients and has quickly approved the vast majority of these requests whenever a company has granted access.
