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Pink Sheet – FDA’s Pazdur Believes ‘Advanced Research’ Project Could Address Trial ‘Wastefulness’

Pink Sheet – FDA’s Pazdur Believes ‘Advanced Research’ Project Could Address Trial ‘Wastefulness’

President Biden’s proposed Advanced Research Projects Agency for Health (ARPA-H) might be able to provide the leadership necessary to cut across barriers between industry and government and within the private sector to make drug development more efficient, Rick Pazdur, US Food and Drug Administration Oncology Center of Excellence Director said.

“One of the things that keeps me up at night is really the wastefulness that we see at the FDA and the clinical trial setting. For example, each company doing their own clinical trials, not working together or not working with each other. How many PD-1 drugs do we need? How many duplicate clinical trials do we need driven primarily by competitive and economic concerns for the individual drug company?” said Pazdur, speaking on 29 July at a Friends of Cancer Research event on the 21st Century Cures Act follow-up legislation Cures 2.0.

Pazdur said he hopes ARPA-H can address these and other barriers to spur more collaborative research.

FDA did not respond to follow up questions from Pink Sheet on how ARPA-H could address Pazdur’s concerns and how the research agency would need to be structured to best do so.

The agency, in particular Acting Commissioner Janet Woodcock, have long pushed sponsors to be more collaborative in clinical trial efforts to increase efficiencies and generate more usable data, including by repeatedly promoting master protocol designs. But FDA has had few successes in this area and COVID-19 therapeutic research only highlighted the problems with the status-quo.  (Also see “COVID-19 Should Force ‘Soul Searching’ Over Fragmented US Clinical Trials System, Woodcock Says” – Pink Sheet, 8 Oct, 2020.)

A May Congressional Research Service report on ARPA-H had a mixed opinion on whether ARPA-H would be able to facilitate better clinical trial coordination and infrastructure or whether existing government programs may be more suited to these tasks.  (Also see “Biden’s ‘Advanced Research’ Agency May Have Pros And Cons, Congressional Research Service Concludes” – Pink Sheet, 22 May, 2021.)

The Cures 2.0 sponsors, Reps. Diana DeGette, D-Colo., and Fred-Upton, R-Mich., aim to use the White House’s plan for the new health research agency, ARPA-H as a cornerstone of their new legislation. (Also see “Cures 2.0 Leaders Sees Biden ‘Advanced Research’ Project As Ticket To Bill’s Success” – Pink Sheet, 18 May, 2021.)

At the 29 July Friends of Cancer Research event, DeGette and Upton interviewed Pazdur and US National Cancer Institute Director Ned Sharpless on topics related to their draft legislation – a sign they may take Pazdur’s comments to heart as they continue to work on the bill.

Sharpless said he envisions ARPA-H as an organization that will be able to be more nimble than the traditional National Institutes of Health structure of which NCI is one part. ARPA-H is envisioned to be under the wider NIH banner as well. 

ARPA-H should be designed to get around “weedy government stuff” that can slow current NIH processes down, he said. It should have more flexibility to work with industry and novel contracting authorities. 

The NCI Director envisions ARPA-H as a center for research that neither the government nor industry would likely do on their own.

For example, he described a potential ARPA-H project often touted by NIH Director Francis Collins to design a blood test that could detect cancer.

“That’s a technology that’s potentially transformative if it works, but it needs to be evaluated in a proper, well designed trial with a mortality endpoint. That would be very hard for industry to do on their own for reasons I won’t go into. But it’s complicated. That would be very hard for the NCI to do. We could we could do it, but it would take a very long time. But I think something like ARPA-H could sort of slot in that middle and bridge that public-private divide, and really accelerate research profoundly,” Sharpless said.

Other projects floated for ARPA-H by NIH Director Francis Collins have included figuring out how to apply mRNA vaccine technology to cancer, and work on biomarkers for Alzheimer’s.

The key to ARPA-H’s success will be the correct design and authorities from Congress, Sharpless said.

“If it just looks like the NIH and has no differences with the NIH, it’ll be like the NIH. But if it has novel authorities to work with industry or to do work with the FDA, or other parts of government in a more nimble manner, it can be highly, highly valuable and really transformative for cancer research,” Sharpless said.

NIH Director Francis Collins has tried to weave a careful narrative that it is both important for ARPA-H to be located within his agency, but also be distinct in operations from many other parts of the agency. (Also see “Would Biden’s ‘Advanced Research’ Projects Mesh Well At NIH?” – Pink Sheet, 16 Apr, 2021.)

“To have ARPHA-A somewhere else other than NIH, I think would be a big mistake, it would be duplicative in all kinds of ways, it would not take advantage of all the basic science foundation that exists through NIH out of which ideas for our ARPA-H will come bubbling up,” Collins said 14 July at a Stat News event.

“But you want to be sure that it is not the 28th institute, that it has a lot of autonomy, that it is not put into the place of going through the very somewhat slow and oftentimes conservative two levels of peer review that have characterized most of what NIH has done – wonderfully so I might say, but it doesn’t fit with certain circumstances.”

Collins said it will be important that ARPA is set up with a director who has an “entrepreneurial approach,” with the authority to pick projects and hire project managers from outside the government. Project managers should be given the authority they need to design and manage projects, he added.

The NIH Director, should be “pretty much hands off” when it comes to selection and managing of ARPA-H projects, Collins said.