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Pink Sheet – US FDA’s Pazdur Suggests NCI Should Help Sponsors Enroll Clinical Trials For Innovative Drugs

Pink Sheet – US FDA’s Pazdur Suggests NCI Should Help Sponsors Enroll Clinical Trials For Innovative Drugs

Oncology sponsors could overcome problems enrolling US patients in clinical trials by enlisting the National Cancer Institute and its network, a US Food and Drug Administration official said.

Richard Pazdur, director of the FDA’s Oncology Center of Excellence, said the NCI could be put in charge of US clinical trial enrollment and leave sponsors to handle accrual internationally. Ideally, the databases could easily be combined when the trial is complete, he said.

“One of the areas that I would love to see [is] the NCI work with and more with companies,” he said during the 17 November Friends of Cancer Research Annual Meeting. “I think we’ve really not had a lot of the NCI trials really take a look at innovative trials.”

FDA Commissioner Robert Califf, who was questioning Pazdur as part of the meeting session, agreed with the idea.

“I think there’s a lot at stake for you and [NCI Director Monica Bertagnolli] to work this out because oncology has a lot of special privileges because it’s considered so important by so many people,” Califf said. “If you get this right, this could have a huge impact.”

The clinical trials network administered by the NCI stretches throughout the US and Canada, with four adult cancer groups and another focused on childhood cancer. The network includes 32 academic medical centers, as well as community hospitals and has centralized institutional review board, imaging, and data management services.

Allowing sponsors to tap into the vast network could turbocharge trial enrollment, in part from the volume of participating sites, which would speed accrual and ideally make successful treatments available to patients sooner.

In addition, because of the network’s reach, diversity within clinical trials also could improve. Pazdur has wondered about mandating a diversity requirement, given that only 20% of participants in multi-regional clinical trials come from the US.  (Also see “Sponsors Want Answers On Consequences Of Missing US FDA Trial Diversity Goals” – Pink Sheet, 20 Oct, 2022.)

Pazdur also has said trial diversity encourages trust in the research system and approved drugs.  (Also see “In Trials We Trust: US FDA’s Pazdur Urges Diversity To Build Confidence In Medicine” – Pink Sheet, 22 Aug, 2022.)

Data Sharing, IP, Other Potential Roadblocks

Bringing the NCI into sponsors’ clinical trials would not necessarily be novel – it happened before with a leukemia drug, Pazdur said. However, the project could be extremely difficult to execute.

Drug price negotiation provisions of the Inflation Reduction Act may be shifting oncology and other drug development practices overseas. Eli Lilly CEO David Ricks said the IRA may force sponsors to wait for data on multiple indications and seeking authorization in other countries before the US.  (Also see “Political Trials On All Sides: Lilly CEO Laments Loss Of ‘Chamber of Commerce Republicans’” – Pink Sheet, 17 Nov, 2022.)

Issues such as data rights would have to be resolved before the relationship could be established between developers and the government. Sponsors also may not want to use the network for candidate products, preferring to use NCI to test secondary indications after initial approval.

Intellectual property questions also could become more relevant, especially when drug pricing complaints emerge. A cancer drug that advocates view as too expensive and tested through the NCI trials network could boost calls for the federal government to invoke march-in rights, which would allow other manufacturers of products that use IP developed with government-funded research.  (Also see “Year Of IP: From The TRIPS Waiver Proposal To The Moderna-NIH Patent Tussle” – Pink Sheet, 13 Jan, 2022.)

In addition, sponsors and the NCI may have different data collection methods, which may not be easily reconciled. Even information like patient sex sometimes is not entered into electronic records in the same format across trials, which can create problems comparing the results.

Comparability is a major problem in trials using real-world evidence. The FDA has raised questions with several products as sponsors have tried to use of RWE in decision-making.  (Also see “The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions” – Pink Sheet, 23 Sep, 2022.)

Sponsors implementing novel trial designs also want to keep some aspects of them secret until they are completed, which could be a problem. An FDA program to encourage development of novel trial designs includes a requirement that the agency must be able to discuss some aspects program participants’ cases in public.  (Also see “RWE Program Offers Sponsors Chance At Novel Uses, But In Exchange For Disclosing Proposals” – Pink Sheet, 19 Oct, 2022.)

Oncology, Clinical Trial System Improvement Campaign Still Growing

Several efforts intended to streamline clinical research in oncology and other areas already are underway.

Pazdur launched Project Site Selector earlier this year to understand why sponsors want to use foreign over domestic sites for oncology clinical trials. Enrollment speed and trial completion time were among the reasons some gave for the increased use of sites in China and post-Soviet states.  (Also see “US FDA’s Project Site Selector Kicks Off With A Look At Why Cancer Trials Go Overseas” – Pink Sheet, 1 Aug, 2022.)

OCE’s Project Equity was created to increase trial diversity (Also see “US FDA Calls For Clinical Trial Diversity Plan ‘As Soon As Practicable’ In Product Development” – Pink Sheet, 18 Apr, 2022.), and Project Frontrunner is intended to push sponsors to develop cancer treatments using randomized trials outside of the refractory setting.  (Also see “Cancer And Accelerated Approval: FDA To Crack Down On Single-Arm Trials, Refractory Disease Focus” – Pink Sheet, 10 Jun, 2022.)

Califf also has called for cutting clinical trial bureaucracy and increasing data sharing.  (Also see “Califf Outlines Clinical Trial Reform Message, Which Could Travel With Him To Commissioner’s Office” – Pink Sheet, 22 Nov, 2021.)