Fast-Track proposals will NOT be accepted. Direct-to-Phase II proposals will NOT be accepted. Number of anticipated awards: 2-3 Budget (total costs, per award): Phase I: up to $400,000 for up to 12 months Phase II: up to $2,000,000 for up to 2 years PROPOSALS THAT EXCEED THE BUDGET OR PROJECT DURATION LISTED ABOVE MAY NOT BE FUNDED. Summary For existing and novel cancer therapies, there is an unmet need for quantitative biomarkers for selecting patients for targeted therapeutic products as well as for evaluating and predicting therapeutic outcomes faster and with greater precision. These biomarker reference tools are also needed to address challenges associated with heterogeneous or difficult to biopsy tumors. Such biomarkers tests may be considered for qualification by the FDA Medical Device Development Tools (MDDT) Program. FDA’s mission is to protect and promote public health by helping to speed innovations that make medical products safer and effective for the public. The FDA MDDT Program is a mechanism for FDA to qualify tools that companies can use in the development and evaluation of medical devices subject to regulatory decision-making by the Center of Devices and Page 89 Radiological Health (CDRH). MDDTs can have a variety of uses/roles in a device clinical study such as patient selection, study population enrichment, monitoring treatment response, predicting or identifying safety problems related to treatment with a medical device, or identifying patients who are or are not candidates for certain forms of therapy. Learn more about the FDA’s MDDT Program here. FDA’s MDDT Program collaboration with the NCI SBIR Development Center can help incentivize the small business community to develop these innovative tools in oncology-related regulatory decision-making and disseminate them by selling to industry or academia who are developing new device technologies, or users such as device developers that would benefit from using the MDDT in their regulatory submission. Given these similar areas of interest, FDA CDRH and NCI SBIR have developed this joint contract topic to stimulate and support innovation across our overlapping communities. Potential examples that could be MDDTs include new/high resolution multimodal imaging as biomarkers for detection of various melanomas or difficult to biopsy tumors, or laboratory-based biomarker tests to be used to help regulatory evaluation of diagnostic and therapeutic medical products. Project Goals The goal of this contract topic is to stimulate the participation of small businesses in the FDA's MDDT Program to develop quantitative biomarker tests. An MDDT is a method, material, or measurement used to assess the effectiveness, safety, or performance of a medical device. MDDTs can accelerate the device development process by providing developers with measurements and tools qualified by FDA that do not need to be re-evaluated within the context of use which helps streamline/speed device development and FDA regulatory decision-making. Offerors are expected to have identified biomarkers and tools with the potential to serve in the evaluation of newly developed similar reference tests for patient selection or device safety/effectiveness evaluation by CDRH. Biomarker-based assays that may serve as reference tools and qualify as an MDDT include tests or instruments used to detect or measure a biomarker. Categories of biomarkers that could be used in clinical or nonclinical trials evaluating devices include: susceptibility/risk biomarker, diagnostic biomarker, monitoring biomarker, prognostic biomarker, predictive biomarker, pharmacodynamic/response biomarker, and safety biomarker. CDRH also intends to consider characteristics derived from medical imaging to be biomarker tests. Activities that fall within the scope of this solicitation include development and optimization of a biomarker-based assay that meets the criteria defined by the FDA MDDT Program. Examples of technologies considered responsive to this solicitation include quantitative biomarker tests for checkpoint inhibitors to enhance cancer patient selection, quantitative imaging methods for assessing therapeutic outcomes, or an algorithm combining various biomarkers to make a comprehensive assessment in therapeutic or safety outcomes in cancer patients. Phase I Activities and Deliverables: • Develop a working biomarker-based assay that meets the criteria defined by the FDA MDDT program. • Prepare an MDDT proposal using the MDDT Qualification Plan Submission Template which includes specific requirements and activities with respect to the proposed MDDT. For additional details review ‘Qualification of Medical Device Development Tools - Guidance for Industry, Tool Developers, and Food and Drug Administration Staff.’ • Demonstrate the suitability of the assay for use in a regulatory setting. • Submit a complete Qualification Plan to the FDA’s MDDT Program. It should include description of the MDDT, context of use, and a detailed plan to collect evidence based on the context of use for qualification of the tool. Use the MDDT Qualification Plan Submission Template for this submission. • Specify the quantitative technical and commercially relevant milestones that will be used to evaluate the success of the biomarker-based assay. • Develop a regulatory strategy/plan and timeline to file a regulatory application for an MDDT. Phase II Activities and Deliverables: • Build the biomarker-based assay according to the specifications developed in Phase I. • Optimize and demonstrate regulatory/clinical utility and value by testing sufficient numbers of patients from multiple sites to unequivocally prove statistical significance with regards to patient selection. • Prepare a Full MDDT Qualification Package Submission Template which includes specific requirements and activities with respect to the proposed MDDT. • Demonstrate the safety and efficacy of the biomarker-based assay in relevant animal models if required by FDA. • Engage with FDA to refine and execute an appropriate regulatory strategy. If warranted, provide sufficient data to submit a regulatory application to obtain approval for clinical application. Page 90 • Submit a Full Qualification Package to the FDA’s MDDT Program including the data collected according to the FDA-accepted Qualification Plan. Use the MDDT Qualification Package Submission Template for this submission. Frequently Asked Questions 1. Who are the potential customers for an MDDT? MDDTs can be used by other developers, researchers, small businesses, and other industry and research groups who are working to develop technologies in the same space as the MDDT technology. These tools will facilitate the regulatory decision-making process and expedite the development of new technologies, benefiting both FDA and companies with technologies under FDA review. 2. Will FDA or NCI purchase the MDDT? Offerors must identify the eventual customers for their tool. NCI and the FDA are not potential customers for this product. 3. Are there examples of MDDTs? Yes, the MDDT page (https://www.fda.gov/medical-devices/science-and-research-medical-devices/medical-devicedevelopment-tools-mddt) lists some examples of MDDTs. There are no examples in the biomarker or the dataset spaces, which is one reason that the FDA and NCI are interested in supporting offerors working in these areas. 4. What happens if my tool is not qualified as an MDDT? You must submit your qualification plan to the FDA by the end of the Phase I contract. CDRH will review Full Qualification Packages submitted at the end of the Phase II contract and make a qualification decision regarding the tool’s acceptance as an FDA-qualified MDDT. This risk is mitigated by a company developing their Qualification Plan in accordance with CDRH feedback prior to submitting their final Qualification Plan to FDA. If awarded, companies are highly encouraged to engage FDA early on when developing their Qualification Plan for the MDDT Program.