Abstract 4194: Rare cancer’s ‘valley of death’: Critical gaps in data, research, and funding

Abstract

Abstract Over 370 forms of rare cancer compose at least 29.7% of all U.S. cancer diagnoses, or over 500,000 Americans per year. And yet, available data, research, and public funding related to these indications is insufficient in comparison with the 20 forms of non-rare cancers. Rare cancer is defined by the American Cancer Society as a cancer having an incidence of <6 per 100,000 per year. A review of the AACR Annual Proceedings 2018 showed that at least 150 rare cancers were not the focus of any abstract. Non-rare forms of breast cancer, expected to represent 11.9% of new cancer cases in 2018, had almost the same coverage (1081 abstracts, 25.6%) as all rare cancers combined (1155 abstracts, 27.3%). Rare cancers which were included in AACR 2018 were the focus of an average of 5 abstracts each, while the 20 non-rare cancers were the focus of an average of 154 each. That gap in basic science is also visible in broader data sources. A computational review of the 10,000 most recent cancer-related publications in PubMed showed that over 100 rare cancers had no publications, while that was true of only 1 non-rare cancer. Included rare cancers had an average of 3.2 mentions in PubMed abstracts, while non-rare cancers had an average of 115. Adequate patient-derived tumor gene expression data is critical for targeted drug development. Analysis of the Gene Expression Omnibus (GEO) repository demonstrated that, while all 20 non-rare cancers had DataSets, over 100 rare cancers had none. On average, each rare cancer included in GEO had 1.04 DataSets, while each non-rare cancer had 22.8. Government grants to companies are an important tool for bridging the drug development “valley of death” between basic science and the clinic. Between 2013 and 2017, 781 SBIRs and STTRs related to cancer research, development, or treatment were identified. 489 of them related to a specific cancer or cancers. Of those grants, 41.5% were related to breast, lung, or prostate cancers. 22.3% were related to rare cancers. Among rare cancers, glioblastoma was the specific focus of the most grants (18). 356 forms of rare cancer were not the specific focus of any grant. Pediatric cancers, which are all rare, were the focus of 8 grants (1%). These data highlight a significant gap in rare cancer-focused data, research, and public funding which contributes to lower availability of clinical trials and therapeutics for over half a million U.S. patients per year. Citation Format: Robert L. Treuting, Sean Murphy, Niall Murphy, Bovey Rao, Anekha Goyal, Nico Tuccillo, Ryan Ki-Hoon Kim, Dylan Parker, Katherine Arline. Rare cancer’s ‘valley of death’: Critical gaps in data, research, and funding [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2019; 2019 Mar 29-Apr 3; Atlanta, GA. Philadelphia (PA): AACR; Cancer Res 2019;79(13 Suppl):Abstract nr 4194.