Given the finite resources for cancer research, understanding the focus of current clinical research efforts and the factors influencing these efforts is important.1 Ideally, research efforts should be focused on maximizing the potential for public good.2 Understanding whether research resources are applied in relation to the quality of current clinical evidence or to societal disease burden is a key step toward their rational distribution.
One measure of the quality of current clinical evidence for a particular cancer type is the quality of the evidentiary base of clinical practice guidelines promulgated by professional societies such as NCCN. A recent analysis found substantial gaps in the quality of evidence guiding clinical decisions for numerous cancer types, because most recommendations in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for the 10 most common cancers are based on evidence rated lower than category 1 (C1).3 Furthermore, quality of evidence, as determined by the proportion of C1 recommendations, varies widely by cancer type. It is unclear whether cancer research efforts are focused in areas of lower evidence quality, in which greater opportunity may exist for discovery, or in areas of greater evidence quality, in which past successes raise additional research questions that are ripe for further study.
In addition to current evidence quality, societal disease burden should ideally guide research efforts.4 Although NIH funding seems to be allocated to diseases that pose a greater burden,5 the relationship between this burden and projected enrollment in cancer randomized clinical trials (RCTs) has never been formally studied. How RCT enrollment from all funders is distributed with respect to societal disease burden and the current quality of evidence is unknown. Therefore, to assess how current cancer research efforts are prioritized, the authors investigated whether planned enrollment in current clinical trials in oncology is distributed among cancer types proportionate to evidence quality and societal burden. Assessment of the current state of clinical trial efforts with regard to these 2 factors is the first step toward optimal allocation of the finite resources available for cancer research.
Drs. Lloyd, Buscariollo, Makarov, and Yu have disclosed that they have no financial interests, arrangements, affiliations, or commercial interests with the manufacturers of any products discussed in this article or their competitors. Dr. Gross has disclosed that he receives a research grant from Medtronic and is on the advisory board for Fair Health Inc.
BurnetNGJefferiesSJBensonRJ. Years of life lost (YLL) from cancer is an important measure of population burden—and should be considered when allocating research funds. Br J Cancer2005;92:241–245.
PoonachaTKGoRS. Level of scientific evidence underlying recommendations arising from the National Comprehensive Cancer Network Clinical Practice Guidelines. J Clin Oncol2011;29:186–191.
Committee on NIH Research Priority-Setting Process. Scientific opportunities and public needs: improving priority setting and public input at the National Institutes of Health. Washington, DC: National Academy Press; 1998.
GrossCPAndersonGFPoweNR. The relation between funding by the National Institutes of Health and the burden of disease. N Engl J Med1999;340:1881–1887.
HarrisRPHelfandMWoolfSH. Current methods of the US Preventive Services Task Force: a review of the process. Am J Prev Med2001;20(3 Suppl):21–35.
Food and Drug Administration Modernization Act of 1997. Pub L No. 105-115 §113 111 Stat 2296.
NCCN Categories of Evidence and Consensus. National Comprehensive Cancer Network Web site. Available at: http://www.nccn.org/professionals/physician_gls/categories_of_consensus.asp. Accessed January 29 2012.
Estimated New Cancer Cases and Deaths by Sex US 2012. National Cancer Institute. Available at: http://www.cancer.org/acs/groups/content/@epidemiologysurveilance/documents/document/acspc-032009.pdf. Accessed January 27 2012.
HowladerNNooneAMKrapchoM eds. SEER Cancer Statistics Review 1975-2008National Cancer Institute. Bethesda, MD based on November2011SEER data submission. Available at: http://seer.cancer.gov/csr/1975_2008/. Accessed January 27 2012.
Cancer Trends Progress Report 2009/2010 Update. National Cancer Institute. Available at: http://progressreport.cancer.gov/doc_detail.asp?pid=1&did=2009&chid=96&coid=930&mid#life. Accessed January 12 2012.
The global burden of disease: 2004 update. The World Health Organization. Available at: http://www.who.int/healthinfo/global_burden_disease/2004_report_update/en/index.html. Accessed January 29 2remo012.
ChengSKDietrichMSDiltsDM. A sense of urgency: evaluating the link between clinical trial development time and the accrual performance of cancer therapy evaluation program (NCI-CTEP) sponsored studies. Clin Cancer Res2010;16:5557–5563.
LiECDeMartinoJ. Preliminary report: the development of the NCCN Comparative Therapeutic Index as a clinical evaluative process for existing data in oncology. J Natl Compr Canc Netw2010;5(Suppl 5):S1–9.
GrossCPMalloryRHeiatAKrumholzHM. Reporting the recruitment process in clinical trials: who are these patients and how did they get there?Ann Intern Med2002;137:10–16.