The U.S. Food and Drug Administration is using some new tools in its drug approval process and at least one prominent industry leader is giving the government entity a thumb’s up.
For years, the FDA has relied solely on clinical trials. Now, the FDA is incorporating real world data (RWD) and real world evidence (RWE) into its drug approval process.
“Incorporating RWD and even RWE into drug approval, label expansion, and regulatory oversight is laudable and will ultimately benefit patients, both as a way to speed up the trial process and ultimately the approval process and in developing truly personalized care for each patient,” Valerie Guild, the founder and president of AIM at Melanoma Foundation, told the FDA Reporter.
Guild said these new tools should especially help patients who have rare cancers where RWD and RWE can add to traditional clinical trial data and give investigators a better understanding of safety and efficacy.
“We need to move to the next generation of clinical trials,” Guild said. “AIM is working on the first adaptive platform trial in melanoma to test a number of treatments and combinations simultaneously, using RWD/RWE as the control arms. Our current clinical trial system generally tests one or two drugs at a time against the standard of care —a slow and inefficient process that entails the need for many patients on each and every one of the various control arms, information that already exists in EHR.”
Guild said adaptive trials lead to personalized therapies and faster answers on a drug’s efficacy — and may save lives in the process by negating the need for patients in the control arms.
“We are appreciative of the initial encouragement we’ve received from the FDA — and non-profits like ours, who are willing and able to lead a global adaptive trial, need continued support so the trial can succeed, and so that we can be a model for other disease foundations willing to tackle such a project,” Guild said.
According to Forbes, the FDA has been moving toward these new and private technologies as it recently expanded its agreement with Flat Iron Health and COTA Healthcare.
Usually, the FDA just uses data from clinical trials. But last year then FDA commissioner Scott Gottlieb announced a framework to include the use of RWD and RWE in developing new drugs and biologics.
Gottlieb said in a statement to Forbes said the FDA recognized how important RWD and RWE were.
The agency was “committed to realizing the full potential of these tools in advancing the development of novel therapeutic products and strengthening our regulatory oversight of medical products across the life-cycle continuum,” he said.
