To the Editor:
Re “Doctors Who Discover Miracle Cures Worry About Medicine’s Future,” by Ruxandra Teslo (Opinion guest essay, Sept. 6):
Dr. Teslo is right that America’s clinical trial system has become too costly and cumbersome, particularly for emerging biotech companies. But faster Phase 1 oncology trials should not be the sole measure of reform.
Phase 1 is where an experimental therapy first enters humans, and where safety, tolerability and dosing must take precedence over speed. Oncology has extraordinarily high attrition: One analysis found that only about 6 percent of oncology drugs entering Phase 1 ultimately received Food and Drug Administration approval. That underscores why rigorous review remains essential.
The missing piece is how smaller biotech companies access the expertise needed to navigate that rigor efficiently. Rather than weaken safeguards, we should expand partnerships with specialized contract research organizations. These smaller organizations can be leaner and more agile, using advanced technology to model trial scenarios, improve study design and identify the most effective path before execution. They can then bring together clinical operations, data management and statistical and regulatory expertise with greater flexibility and attention.
If we want more patients to benefit from innovation, the goal should be smarter, more efficient trials — not simply faster ones.
Armen Margaryan Los Angeles The writer is the chief executive of NoyMed, a medical contract research organization.
To the Editor:
There are certainly inefficiencies that hinder clinical trial sponsorship, enrollment and completion. But Ruxandra Teslo ignores the key hindrance to finding new cancer treatments: the Trump administration’s cuts in funding for cancer research.
The administration has cut billions of dollars in research funding, terminating or delaying nearly 2,500 grants at the National Institutes of Health. Academic medical centers and medical research centers have lost critical funding, leading to staff layoffs, abandoned patients and prematurely terminated studies. Organizations like the Association for Clinical Oncology and the American Cancer Society are frantically warning that these cuts are badly damaging clinical research.
The 24 percent drop in 2025 for cancer research at a time when exciting treatments are emerging for melanoma, pancreatic cancer and prostate, head and neck cancers is the single biggest roadblock to innovation.
Calls to reform the regulatory environment ignore the dire financial situation defunded researchers now face. No amount of bureaucratic reform will succeed in making up for the inexplicable fiscal starvation of cancer research.
Arthur Caplan Ridgefield, Conn. The writer is a bioethicist and a professor emeritus at New York University’s Grossman School of Medicine.
To the Editor:
It’s worth mentioning that the Food and Drug Administration has gotten faster in approving single-patient compassionate-use clinical studies under its current guidelines. Our company recently had a submission for a single-patient compassionate-use trial of a new drug for pancreatic cancer approved in two days. The critical issue is whether the sponsor can clearly demonstrate that the drug is safe.
The larger issue, as the author points out, is that most new drugs originate from small biotechnology companies, which are often spinouts from universities. As National Institutes of Health spending has gotten more difficult and venture capitalist funding has been significantly diverted to anything with “A.I.” in the title, the small biotech companies that are often the originators of these new medicines are struggling to find the funding to support these Phase 1 trials in cancer patients.
Ira Spector Jenkintown, Pa. The writer is a co-founder of SFA Therapeutics and was the global vice president for clinical operations at Allergan, Pfizer and Wyeth Pharmaceuticals. He has been involved in the development of 33 approved drugs.
To the Editor:
Ruxandra Teslo is right that the United States has made early drug development too slow and expensive. As someone who conducts Phase 1 and first-in-human cancer trials, I would add that the problem extends beyond the Food and Drug Administration.
Once the F.D.A. receives a complete investigational new drug application, a trial generally may proceed after 30 days unless the agency places it on clinical hold. Yet an Association of American Cancer Institutes survey found that industry-sponsored cancer trials took a median of 170 days from submission for institutional scientific review to opening for patients. Only 9 percent of responding cancer-center clinical trial offices reported meeting a 90-day activation timeline for industry studies, an aspirational target recommended by the National Cancer Institute.
Those months are consumed by contracts, budgets, Medicare coverage analyses, scientific and ethical review, pharmacy requirements and more. Some are essential. But necessary oversight does not require that every process occur sequentially, be repeated locally or take as long as it does.
The Operation TrialBlazer pilot program that Dr. Teslo writes about is a welcome effort to widen one bottleneck. But many of the delays occur downstream, with sponsors and inside cancer centers and research institutions.
The F.D.A. can widen one bottleneck. Cancer centers, sponsors and research institutions must clear the ones downstream.
Gilberto de Lima Lopes Jr. Miami The writer is a medical oncologist and researcher at the Sylvester Comprehensive Cancer Center at the University of Miami.
To the Editor:
Ruxandra Teslo is right that the growing cost of Phase 1 trials is slowing drug development in the United States. But her assessment of Food and Drug Administration regulation as a primary source of the problem is a little too hasty.
In her telling, the F.D.A.’s mission is to mitigate “any potential risk, however negligible,” before development can proceed. Yet the F.D.A.’s longstanding practice has been to assess the risks of new therapies against their potential benefit, accepting higher risk where there is significant benefit or unmet medical need.
The F.D.A. allows most Phase 1 studies to proceed within 30 days, unless the proposed study is put on hold because of unreasonable risk or other factors. The real delays often arise after the F.D.A. has given the green light: Many months can be lost as hospitals, researchers, manufacturers and others negotiate contracts, budgets and protocol amendments.
The current legislative cycle presents a good opportunity for Congress to consider updating the legal framework for early-stage research. But even if Congress acts, the F.D.A. is likely to retain a significant role in setting the parameters for risk in the development of U.S.-approved drugs — and adapting those standards to rapidly evolving science.
The agency needs strong, independent leaders capable of supplying the technical expertise that Congress, the courts and the politicians lack.
Grail Sipes Washington The writer worked for more than a decade for the Food and Drug Administration, where she was the deputy director for the Center for Drug Evaluation and Research and the director for the center’s Office of Regulatory Policy.
To the Editor:
Ruxandra Teslo is right that cancer patients and physicians face significant bureaucratic challenges in accessing promising but unapproved medicines in the United States.
For patients with no remaining options, the essay touches on a pathway commonly called compassionate use, known as expanded access at the Food and Drug Administration. It deserves more attention.
In the Ogdens’ case, featured in Dr. Teslo’s essay, where the family sought access to a drug approved for another cancer, the F.D.A. was not the barrier. According to F.D.A. data, when a physician requests access and the drug company agrees, the F.D.A. allows treatment to proceed about 99 percent of the time.
The Ogdens lost months finding a doctor and hospital willing to use an existing pathway and coordinate with the drug company. More education on this pathway is imperative for health care providers and patients so critical treatment can be delivered as quickly as possible.
Matthew Rosen Menlo Park, Calif. The writer is a lawyer and the chief executive of MedaSystems, which makes software that drug companies use to manage expanded access requests.
The post Why Do Clinical Trials for Cancer Drugs Take So Long? appeared first on New York Times.
