National Cancer Institute (NCI) Small Business Innovation Research (SBIR) Concept Award Program – Fiscal Year 2022

Background About 11,050 children in the United States under the age of 15 will be diagnosed with cancer in 2020 (https://www.cancer.org/cancer/cancer-in-children/key-statistics.html). Cancer is the second leading cause of death in children ages 1 to 14. Similarly, as a group, rare cancers are the leading cause of cancer deaths in the United States (https://rarecancer.org/). For the purpose of this solicitation, rare cancers include all the cancers listed by the NIH Genetics and Rare Diseases Information Center. The list can be found here: https://rarediseases.info.nih.gov/diseases/diseases-by-category/1/rare-cancers. However, because of smaller patient population and challenging development pathways, development of technologies focused on pediatric and rare cancers lag significantly compared to other major cancers. Innovative and transformative solutions focused on prevention, detection, treatment, and research in both pediatric cancers and rare cancers are urgently needed. Rather than just the tried and tested approaches that have not led to much success and progress, bold and “out of the box” ideas that are still based on sound scientific premise are needed to make a significant impact in the prevention, diagnosis, treatment, and care of these cancers. Thus, the NCI SBIR Development Center is launching the NCI SBIR Innovative Concept Award program to encourage the development of high-risk innovative and disruptive technologies. The concept award program will provide funding to small businesses to explore the technical feasibility and demonstrate proof of concept for the development of highly innovative therapies, diagnostic tools, or preventive strategies focused on pediatric and rare cancers. The focus is on innovation and “out of the box” ideas that have not been tried and tested before. So, preliminary data is not expected from the offerors and will not be a factor in evaluating the overall impact of the proposal. Offerors are eligible to apply if they have disruptive ideas based on sound scientific premise with a potential to make an impact in these cancers. The goal of the funding is to de-risk the technical aspect of the idea by helping to generate key proof of concept validation data. But we also want the offerors to explore the commercial potential and development path of the technology during the award period. So, in addition to funding the program will also provide additional business and commercialization resources including entrepreneurship training and mentorship to explore and refine the business model and commercialization plan. We expect that since the technology is further de-risked and the offerors have gained some preliminary data, they will be more competitive for Phase I and/or Phase II SBIR awards through the standard funding opportunity announcements. Offerors must be Small Business Concerns, as defined in Section 3.2 Definitions, within this solicitation. However, National Cancer Institute recognizes that many innovations originate in universities and research institutions, and therefore we invite academic researchers working on translational technologies to consider applying either via setting up a small business or by partnering with an existing small business. Please refer to Sections 4.2 and 4.10 of this solicitation for requirements for collaborations and requirements for registrations and certifications. Since outstanding research is conducted at a broad spectrum of institutions, this Funding Opportunity Announcement encourages applications from researchers from all institutions, including those serving primarily underrepresented groups, those that may be less research-intensive, and from all domestic geographic locations. The NCI also encourage entrepreneurs from diverse backgrounds, including those from underrepresented racial and ethnic groups, persons with disabilities, and women to work with small businesses and apply. Project Goals The goal of this solicitation is to encourage small businesses to propose “out of the box” ideas to demonstrate “proof of concept” for the development of innovative technologies to make a transformative impact in prevention, diagnosis, treatment, and care of pediatric or rare cancers. To be considered innovative, projects must have the potential to transform the way research is conducted through the development of novel tools or technologies or lead to major improvements in pediatric and rare cancer care through the development of highly innovative therapies, diagnostic tools, or preventive strategies. Projects that primarily focus on optimization, hardening, or obvious extrapolations of established technology might be less competitive. For example, the following types of projects would not be considered innovative and would not be responsive: • Therapeutics targeting genes and pathways with FDA-approved agents or agents in late clinical stage unless the novel approach mitigates known issues with approved agents • Screening and diagnostic approaches utilizing methods that could increase chance of secondary tumors • Screening, diagnostic, and monitoring approaches already in clinical use • Continuation of already funded SBIR/STTR projects Proposals are encouraged for the development of innovative approaches focused on detection, treatment and research of pediatric or rare cancers. The solicitation is agnostic to the type of technologies and modalities as long as they are highly innovative and reflect ideas substantially improved from current state of the art. Projects supported by this program should not be low risk, or incremental improvements to established technologies. These could include but not limited to novel therapeutics and prevention approaches, therapeutic devices, drug delivery approaches, and tools/devices focused on early diagnosis, prognosis and treatment response. Proposals are solicited for all innovative ideas - not limited to the ones above - as long as they are focused on detection, treatment and research of pediatric cancers or rare cancers. Projects are anticipated to have a high risk of failure with concomitant high reward. Commercial potential is evaluated by assuming the continued development of technology is successful, regardless of the inherent risk of the project. The awards will support initial exploration of untested but potentially transformative ideas that may radically change the way we understand, prevent, diagnose, treat, and manage rare or pediatric cancers. These concept awards are intended to provide funds to perform few key activities to demonstrate proof-of-concept and feasibility and lay the groundwork for future research and development in SBIR/STTR Phase I or Phase II awards. Concept awards are not intended to support continuation of already established and advanced research programs. Thus, preliminary data is not required; however, the ideas should have sound scientific premise either based on the offeror’s own research or literature evidence. These awards are focused on development of innovative products and technologies; thus, the anticipated product that will be developed should be identified and development path should be clearly laid out. In addition to funds provided to demonstrate technical proof of concept, the program will integrate customer discovery and entrepreneurship training program and business and commercialization mentorship to ensure that the awardees understand the business model, market fit, and to help them optimize their commercialization plan. As such, the awardees are required to go through the NIH I-CorpsTM (see section 2.3) program. The awardees will also receive mentorship from industry and business mentors to help them refine their business model and commercialization plan. Activities and deliverable proposed by the applicants would differ based on their technology types and stage of development. However, the offerors should ensure that they clearly identify the clinical problem and cancer type(s) that the proposal will focus on with adequate justification. In addition, the offerors should propose experiments to obtain initial de-risking and proof-of-concept data and at the completion of the project present a report with the results of the experiments to the NCI. Examples of activities and deliverables that could be could proposed include (but are not limited to) the following: Page 49 Therapeutic Projects: The offeror could propose most or all of these activities for pediatric or rare cancers. • Target Identification and Validation • Screening and Identification of drug candidates • Identification of lead and Lead candidate optimization • In vivo efficacy studies • Preliminary PK/PD studies Device Development Projects: The applicant could propose most or all of these activities for pediatric or rare cancers. • Evaluation and justification of clinical need • Product concept and protype development • Early feasibility studies • Phantom validation • In vivo validation studies • Software development (if needed) • Biocompatibility, Sterility, and Safety studies Diagnostics Development: The applicant could propose most or all of these activities for pediatric or rare cancers. • Biomarker discovery and biomarker optimization • Assay development and optimization • Define assay performance and analytic validation • Sensitivity, specificity and reproducibility • Validation studies in PDX samples or clinical samples