President Barack Obama announced on Monday that he plans to appoint Harold Varmus, a former director of the National Institutes of Health (NIH) and co-recipient of the 1989 Nobel Prize in Physiology or Medicine, as director of the National Cancer Institute (NCI). Varmus would replace current director John E. Niederhuber, who became NCI head in 2006. The move follows recent criticism of NCI’s clinical-trial program by the Institute of Medicine (IOM), the health arm of the National Academy of Sciences.

Varmus brings a strong cancer background to the NCI. Since 2000, he has served as president of the Memorial Sloan-Kettering Cancer Center in New York City and was recently cochair of President Obama’s Council of Advisors on Science and Technology. He was appointed director of NIH in 1993. While at NIH, he was credited with guiding a new clinical center, strengthening the intramural research program, and initiating a doubling of the NIH budget. His research career began as a member of the US Public Health Service at the NIH and as a post-doctoral fellow at the University of California, San Francisco, where he served as a member of the medical faculty for more than 20 years. His research focused on cancer genes and retroviruses.

So what challenges will Varmus face in his new role as head of the NCI? The IOM issued a report in April 2010 to recommend changes to transform the NCI’s Clinical Trials Cooperative Group, the program responsible for conducting collaborative large-scale cancer clinical trials at institutions and community-based practices in the US and aboard. The review of the NCI’s clinical-trial program was initiated because of concerns by NCI stakeholders, which included clinical investigators, patient advocates, leadership within NCI’s Clinical Trials Cooperative Group, industry participants, and the NCI that “the program is falling short of its potential to conduct the timely, large-scale, innovative clinical trials needed to improve patient care,” said the report.

NCI asked IOM to assess the state of cancer clinical trials, review its Clinical Trials Cooperative Group program, and provide advice on improvements. IOM made four major recommendations: improve the speed and efficiency of the design, launch, and conduct of clinical trials; make optimal use of scientific innovations; improve selection, prioritization, support, and completion of clinical trials; and foster expanded participation of patients and physicians.

In taking over as head of NCI, Varmus will have to address not only the organizational and operational concerns raised by the IOM report but will also face the additional challenge of resource allocation for NCI clinical trials. In April 2010, the American Society of Clinical Oncology (ASCO), released the results of a survey that found that one-third of NCI Cooperative Group sites plan to limit participation in federally funded clinical trials because of inadequate per-case reimbursement. Additionally, nearly 40% of sites planning to limit NCI Cooperative Group trials reported plans to increase industry trial participation despite expressing a preference for conducting Cooperative Group trials, a concern for ASCO. “Federally funded Cooperative Group clinical trials often examine questions that the private sector has little incentive to investigate, such as the comparative effectiveness of treatments made by different companies, therapies for rare diseases, and quality of life after treatment,” said ASCO in an April 15, 2010 release.

ASCO said that NCI funding for the Cooperative Group Program has been virtually flat since federal fiscal year (FY) 2003. NCI devotes approximately $145 million annually to the program, including $60 million to the $2000 per-case reimbursement, representing only 1.2% of NCI’s FY 2009 budget of approximately $5 billion, according to the ASCO press release. An ASCO study in 2003 and a C-Change study in 2005 determined that the actual cost of conducting NCI trials was $5000–6000 per case. If NCI reimburses at a more realistic rate of $6000 per patient, tripling the current amount, this would require an additional $120 million, yet total funding would still only account for 3.6% of the NCI budget, according to an analysis by ASCO.

“While funding is not the only factor impacting participation, the significant and growing disparity between actual research costs and the amount provided by NCI to cover those costs serves as a significant deterrent,” said ASCO in its press release. “Without increased funding from the NCI, Cooperative Groups and research sites cannot increase the number of trials, the number of participants enrolled, or maintain current infrastructure to support and conduct clinical trials.”

If the appointment of Varmus proceeds as planned, it will be important to see how he will be able to effectuate improvement in the NCI clinical-trial system in an era of budget constraints and tighter resource allocation. Given the importance of cancer research and NCI’s role in cancer research, let’s hope that he will be up to the challenge.