Breaking down Operation Trailblazer: Rolling INDs, outside expert submission help, and one-trial requirement

Breaking down Operation Trailblazer: Rolling INDs, outside expert submission help, and one-trial requirement

In this week’s episode of The Cancer Letter Podcast, Paul Goldberg, editor and publisher of The Cancer Letter, and Sara Willa Ernst, reporter, break down HHS’ latest vision for the future: Operation Trailblazer.  The report [https://cancerletter.com/regulatory-news/20260626_2/] is titled: “HHS Roadmap to Maintaining U.S. Leadership in Early Clinical Research and Development.” “The U.S. is apparently losing its leadership in INDs and early phase research,” Paul said. “Some of it is true and also it does not help that the whole enterprise of cancer research right now is in, if not disarray, then at least a kind of mental and spiritual discombobulation.” Most notable were proposed changes to FDA, including an expedited Investigational New Drug program, which would use expertise outside FDA to help prepare INDs before submission, and a lower threshold—just one high-quality late-stage—for confirmatory evidence, which is already common practice in oncology.  Paul checked in with sources about the report after it was released. What he heard with his ear to the ground: It’s unclear whether the plans in the report will amount to policy.  “We don't know whether it's going to happen and there is a problem right now in that FDA really took a hit in terms of staffing,” Paul said. “At the moment, there is no permanent FDA commissioner. Having leadership that's deeply destabilizing was really a problem.” “FDA really right now needs to get its house in order or as the industry says, ‘right the ship,’” he continued. “A rudderless FDA for a while there was a step in the wrong direction. So, they're going to have to clean this up and maybe they will. Let's hope they do. “That's what I'm hearing from folks as I call them. But it was important to get [the news] out on paper even though the story certainly has a large number of caveats.” Stories mentioned in this podcast include: * The Directors: Directors of two Midwestern cancer centers tell us about the challenging healthcare economics in rural areas [https://cancerletter.com/podcastc/20260626_1/] * To keep the early clinical trial enterprise in the U.S., HHS pledges to simplify regulatory requirements [https://cancerletter.com/regulatory-news/20260626_2/] * Joe Fraumeni, founder of molecular epidemiology, dies at 93 [https://cancerletter.com/obituary/20260626_3/] * Karen Huntsman’s vision gave shape to the Huntsman Cancer Institute [https://cancerletter.com/obituary/20260626_4/] * Robert Mayer tells us about pancreatic cancer’s hopeless past as new data shows promise [https://cancerletter.com/in-the-archives/20260626_5/] A transcript of this podcast is available: https://cancerletter.com/podcastc/20260701-operation-trailblazer/

The Directors: Two Midwest directors tell us about the challenging healthcare economics in rural areas

“Institutions are feeling crunched at so many different directions” How’s this for a paradox: The better cancer centers become at keeping patients alive, the more expensive cancer care becomes. This brutal tradeoff hits harder in rural areas, where the cancer burden is higher and the investigator and clinical trial representation is lower. The federal budget introduced last summer dealt Medicaid a mighty blow, which is expected to cause a spike in the number of uninsured Americans over the next decade (The Cancer Letter, July 3, [https://cancerletter.com/cancer-policy/20250703_6a/] 2025).  In the June episode of The Directors, two Midwestern cancer center directors, University of Iowa Holden Comprehensive Cancer Center Director Mark Burkard, and University of Minnesota Masonic Cancer Center Director Jeffrey Miller, discussed the financial and logistical challenges confronting cancer care outside major metropolitan hubs.  “We had a Medicaid expansion in Iowa, and that supported what's called the directed payment program (Medicaid State Directed Payments) and that's being phased out over 10 years,” Burkard said. “I think it's going to affect rural hospitals and hospitals in underserved areas even more. And some money was put back into the rural transformation funds of which our state got $209 million. But we're looking at, basically, over the next 10 years, it's a billion-dollar cut from our health system. So, we're trying to figure out how to tighten our belts to make up for that difference as a health system.” Driven by the One Big Beautiful Bill Act and subsequent CMS regulations, these changes cap supplemental payments and are expected to significantly reduce federal Medicaid spending over the next decade. The effect of the financial tightening is systemic. Even the paths taken by junior faculty members are affected. “It used to be that you could creatively dabble as a junior faculty, and then figure out how to develop your career,” Miller said. “But now, they want you to do it in pretty much three years. You get three years of unrestricted time, and then you’ve got to go out into the world and say, ‘How am I going to support my salary?’"

Policy by way of podcaster—FDA issues priority vouchers after Joe Rogan episode on psychedelics

In this week's episode of The Cancer Letter Podcast, Paul Goldberg, editor and publisher, and Sara Willa Ernst, reporter, go on a trip into the world of federal health policy surrounding psychedelic medicine.  In April, FDA issued National Priority Vouchers to three companies conducting clinical trials of psilocybin for treatment-resistant depression and major depressive disorder, and methylone for PTSD, as well as an IND to study ibogaine for treatment of alcohol use disorder. This came after the president issued an executive order vowing to accelerate drug approvals of psychedelic drugs.  “One thing that they seem to want to do is reverse Nixon's War on Drugs, which stopped all of the psychedelic drugs research in its tracks in the '70s,” Paul said.  Some oncologists are not opposed to this reversal, particularly as clinical trials evaluating psilocybin as a treatment for cancer-related anxiety, depression, demoralization, fear of recurrence, and other psychological conditions have been ongoing since 2008 [https://jamanetwork.com/journals/jamapsychiatry/fullarticle/210962]. But the story behind the policy change is cause for concern.  At a press conference in the Oval Office, President Donald Trump sat next to Joe Rogan, comedian and host of “The Joe Rogan Experience" podcast. Just two and half weeks before, Rogan published a two-hour long interview on ibogaine.  Rogan texted Trump about the psychadelic’s promise as a treatment for drug addiction, brain trauma, and cognitive decline.  “The text message came back: ‘Sounds great, do you want FDA approval?’ It was literally that quick,” Rogan said at a subsequent press conference [https://www.youtube.com/shorts/VFO6Gcrcz4c] with Trump in the Oval Office.  “This is a very unscientific way to roll back the scientific kibosh,” Paul said.  The policy change didn’t appear to come from scientific consensus by an expert panel brought together by FDA or NIH.  “When the President of the United States can text you saying, ‘Would you like an FDA approval?’ I worry about prescribing, but especially off label,” said Paul, editor and publisher of The Cancer Letter. “This thing is not approved, by the way, to give credit to FDA right now.” Stories mentioned in this podcast include: * Is psilocybin heading to a cancer clinic near you? [https://cancerletter.com/regulatory-news/20260618_1/] * What NHS-Galleri taught us—and what it didn’t [https://cancerletter.com/trials-and-tribulations/20260618_2/] * The NHS-Galleri trial has and will continue to provide critical insights to refine future MCED studies [https://cancerletter.com/trials-and-tribulations/20260618_3/] * FDA clears a way for dramatic reduction in animal toxicology studies in cancer [https://cancerletter.com/news-analysis/20260618_4/] * Proposed OMB rule aims at the heart of the U.S. research enterprise—and scientists are fighting back [https://cancerletter.com/the-cancer-letter/20260612_1/] https://cancerletter.com/podcastc/20260624-psychedelics/

Peter F. Garrett to people with cancer: Your story has power

This Pride Month, Peter F. Garrett appeared on The Cancer Letter Podcast to talk about what his experience during the HIV/Aids crisis has taught him about effective healthcare communication and advocacy. Garrett spoke with Paul Goldberg, editor and publisher of The Cancer Letter, and Jacquelyn Cobb, associate editor.  In the 1980s, Garrett was a closeted gay man in his twenties. He met his now-husband, Ken, who was open, and through him was introduced to a network of people who were openly gay. They became Garrett’s “instant-friends.” “All of a sudden I knew all these great people,” Garrett said on this week’s episode of In the Headlines. “But they then were also dying.” That time period was characterized by an “overwhelming sense of fear and constant anxiety,” Garrett said. “It was so mysterious at first. Nobody knew what was going on. They didn't know if it was an infectious thing, if it was a cancer related to poppers, or some sort of environmental thing,” Garrett said. “So, it was just this very scary feeling that you never knew what a phone call was going to be. Phone calls—I remember being kind of terrifying, (these would've been landline phones, obviously) because it just seemed like bad news was coming in, and so forget about doctor's appointments… Right now, everybody's getting hay fever and allergies. That terrified you. If you had a sore throat, colds, anything with a fever, you just assumed you had HIV/AIDS.” This experience was formative for Garrett, who later became the director of External Affairs at NCI. Garrett has since founded Patient Action for Cancer Research, an advocacy organization that aims to organize and amplify patient voices in an effort to protect life-saving health research. “It was really, really scary. I found myself at the epicenter of something that was happening very quickly to a certain population but was really being talked about broadly, and that very much framed the way I think about disease in general,” Garrett said. “This is really about a pandemic, a disease like cancer that, unlike cancer, which has been around forever, was all of a sudden there, but like cancer in many ways, it had a stigma. And I think that stigma associated with disease is something that is part of what you have to think about when you're talking with patients about what they've experienced in their cancer journey.” Garrett had a message for people with cancer: Stories have power. “It's about people coming to the table and realizing that their story, should they decide to tell it, has a lot of power,” Garrett said. “Whether they tell it to their neighbor or their church community or their local reporter or The New York Times or whomever or a member of Congress, I think it's all powerful. Because it just reminds us that this is not just about science. Science is cool, but this is about being healthier.” Stories mentioned in this podcast include: * Proposed OMB rule aims at the heart of the US research enterprise—and scientists are fighting back [https://cancerletter.com/the-cancer-letter/20260612_1/] * House bill gives NCI $7.4 billion in FY 2027—$110 million above current budget  [https://cancerletter.com/capitol-hill/20260612_2/] * As his Senate testimony alleged danger from mRNA COVID vaccine, El-Deiry had an offer to run NCI Frederick lab [https://cancerletter.com/the-cancer-letter/20260612_3/] * Lessons from the AIDS Quilt: what the cancer world can learn from AIDS advocacy  [https://cancerletter.com/trials-and-tribulations/20260612_4/] * Former NCI official's nonprofit seeks to tell the stories of cancer patients [https://cancerletter.com/the-cancer-letter/20251003_1/]  A transcript of this podcast is available: https://cancerletter.com/podcastc/20260617-petergarrett/

Pancreatic cancer breakthrough came from smart scientists making smart bets—not from politicization of science

The oncology community is still riding the high of the success of daraxonrasib—the first drug to show real promise in treating metastatic pancreatic cancer. The drug doubled overall survival for patients in the phase III RASolute-302 trial. At the same time, science continues to become increasingly politicized. The White House Office of Management and Budget released a 412-page proposal May 29 that inserts political appointees into all stages of reviewing and awarding of federal research grants, centralizes OMB control over awarding federal funds, lists “specific principles” that must be applied to review of research programs of all federal entities, including NIH. In this episode of In the Headlines, Paul Goldberg, editor and publisher of The Cancer Letter, and Jacquelyn Cobb, associate editor, talk about how these two stories are inseparable. Political review could put future breakthroughs like daraxonrasib in jeopardy when politicization of science is rooted in distrust. “I was actually thinking of talking to someone who understands Project 2025 to talk about the roots of this kind of dislike for science, which is…I mean, what's not like? Is a treatment for pancreatic cancer a bad thing?” Paul said. Daraxonrasib’s success is thanks to the decades of basic, translational, and clinical research that preceded it, largely funded by federal research dollars. Indeed, the NCI RAS Initiative jumpstarted the field of RAS in 2013.  “To me, the best part of the story, your story, was going back to the link to the NCAB-BSA joint meeting where you could see these guys, with Harold Varmus presiding, deciding what they want to do with the RAS Initiative. By then, you had a pretty good idea that RAS mattered and that it was worth studying. There were already drugs and it was happening.” “It was not a waste of time, but it was also a bet. And it was a smart bet that only NCI could place, and that's why we need to have political appointees—on either party, I don't even care—to look at the entire process, lest those intellectuals get into their idiot heads to cure some other cancer. I mean, my God, that would be awful.” Stories mentioned in this podcast include: * Pancreatic cancer finally starts to budge as daraxonrasib doubles overall survival [https://cancerletter.com/clinical/20260605_1/] * The story of daraxonrasib is the ultimate hero’s journey: A narrative curve with twists, turns, intrigue, drama—and a blow to a mighty foe  [https://cancerletter.com/trials-and-tribulations/20260605_2/] * Political appointees to be inserted into all stages of making research funding awards if OMB rule becomes final  [https://cancerletter.com/the-cancer-letter/20260605_3/] * At Senate hearing, Brown’s El-Deiry and other witnesses say they were targeted for arguing that mRNA vaccine may be linked to cancer [https://cancerletter.com/the-cancer-letter/20260605_4/] * Eric Small, ASCO's immediate past president, speaks on the toll of toxicity—financial, emotional, physical—in cancer [https://cancerletter.com/the-cancer-letter/20260605_5/] * Cancer patients could be exempted from Medicaid work requirements if they prove they are “medically frail”  [https://cancerletter.com/cancer-policy/20260605_7a/] * Richard Pazdur joins Friends board [https://cancerletter.com/cancer-policy/20260605_7b/] A transcript of this podcast is available: https://cancerletter.com/podcastc/20260610-omb/