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Critical Information and Application Guidance

Q: What is SMARTT's mission?
A: The mission of the SMARTT program is to accelerate translation of novel therapeutic or diagnostic imaging agents from preclinical proof of concept (i.e., demonstration of efficacy in a relevant animal model) to submission of an investigational new drug (IND) application to the FDA. To accomplish this mission, the SMARTT program provides services in the form of regulatory affairs support, drug product manufacturing, and pharmacology/toxicology studies to qualified investigators.

Therapeutic candidates or diagnostic imaging agents that are not intended for treatment of heart, lung, or blood diseases, or for which efficacy in an animal model has not been demonstrated, are not eligible for SMARTT services. Also, medical devices, vectors for gene therapy, and exploratory research aimed at identifying lead therapeutic candidates are not eligible for SMARTT services.
Q: What is SMARTT's business model?
A: SMARTT accepts requests from academic institutions, non-profit organizations, and small businesses. The program accepts Request for Service Application (RSAs) during a limited period (30-45 days) beginning in January of each year. These requests undergo:
  1. NHLBI review for eligibility and programmatic balance
  2. Evaluation by a panel of expert reviewers familiar with the field of study. The reviewers will not be publicly identified.
  3. Successful applicants work with the relevant SMARTT facility to develop a project schedule and budget, which undergoes a final approval step by NHLBI prior to initiation of the work. The application and review process is designed to be completed within six months, assuming all eligibility and scientific merit criteria are met.
Q: Is SMARTT a grant program?
A: No. SMARTT provides IND-enabling services to investigators engaged in translational research.
Q: Is there a deadline to apply for SMARTT services?
A: Yes. Deadlines will be announced via e-mail through the NHLBI listserv and posted on the SMARTT program website. The next application deadline is anticipated to be early 2017.
Q: Does SMARTT provide services to all organizations?
A: No. SMARTT services are limited to academic institutions, non-profit organizations, and small businesses.
Q: Does SMARTT support basic research on heart, lung, and blood diseases?
A: No. SMARTT services are intended primarily for studies that support submission of an Investigational New Drug (IND) application to the FDA. Studies involving identification or proof of concept for a drug substance/product would be considered premature for the SMARTT program.
Q: Does SMARTT fund preclinical research for diagnostic tests?
A: No. SMARTT does not fund preclinical research for diagnostic tests, except for imaging agents or other substances requiring an IND application to the FDA.
Q: Does SMARTT fund preclinical research for devices, including delivery devices?
A: No. SMARTT does not fund preclinical research designed to obtain FDA approval for devices.
Q: Does SMARTT support translational research for gene therapy projects?
A: No. Investigators may refer to the NHLBI Gene Therapy Resource Program for support of gene therapy studies.
Q: Does SMARTT support translational research for regenerative cell therapy projects?
A: No. Investigators may refer to the NHLBI Production Assistance for Cellular Therapies program for support of cell therapy research.
Q: Does SMARTT provide regulatory affairs support services?
A: Yes. Assuming the scope of work is within the mission of NHLBI and capabilities of SMARTT's contractors, the SMARTT program provides regulatory affairs support to investigators with promising drug substances/products.
Q: Does SMARTT provide manufacturing and pharmacology-toxicology services?
A: Yes. Assuming the scope of work is within the mission of NHLBI, the SMARTT program provides GMP/non-GMP manufacturing of biologic, non-biologic and small molecule drug substances and GLP/non-GLP pharmacology-toxicology services.
Q: What are the minimum eligibility requirements for SMARTT services?
A: The minimum eligibility requirements for SMARTT services are twofold: (1) the subject of the research must be within the mission of NHLBI; and (2) the proposed project must support preclinical studies leading to submission of an IND application, including amendments or extensions of existing INDs. In this context, "Translational Research" is narrowly defined as activities occurring between discovery of a biologic or non-biologic/small molecule drug substance/product effective for the treatment of a specific disease, and initiation of an early phase clinical trial.
Q: What are the minimum requirements for GMP manufacturing or GLP pharmacology-toxicology services?
A: Assuming the scope of work is within the mission of NHLBI and capabilities of SMARTT's contractors, the minimum requirements for GMP manufacturing or GLP pharmacology-toxicology services are (i) demonstration of efficacy in at least one relevant animal model and (ii) a pre-IND meeting with the FDA, the outcome of which provides a reasonable expectation that the drug substance/product will progress towards an IND application if the project is successful. Investigators who have completed in vivo efficacy studies may apply for regulatory affairs services/consultations to support preparation for a pre-IND meeting. SMARTT does not have sufficient resources to support proof-of-concept, dose or product optimization, or discovery phase activities. Please refer to the SMARTT website for additional information on SMARTT's Role in Translational Research.
Q: What are the minimum requirements for non-GMP manufacturing or non-GLP pharmacology-toxicology services?
A: Assuming the scope of work is within the mission of NHLBI and capabilities of SMARTT's contractors, the minimum requirement for non-GMP manufacturing or non-GLP pharmacology-toxicology services is demonstration of efficacy in at least one relevant animal model. SMARTT does not have sufficient resources to support proof-of-principle or discovery phase activities. Please refer to the SMARTT website for additional information on SMARTT's Role in Translational Research.
Q: How do investigators request services from the SMARTT Program?
A: The request process begins with completion of a Request for Services Application (RSA) through the SMARTT program website.
Q: How much can I expect to receive in the form of SMARTT services?
A: Unfortunately, the SMARTT program does not have sufficient funding to provide full support for GMP manufacturing projects and GLP studies, which typically cost millions of dollars. Although exceptions are possible, in most cases the cost of services provided by SMARTT facilities will not exceed approximately $500,000 per project. When a determination is made that the requested services exceed this amount, investigators will be asked to consider a partnering arrangement wherein the costs will be shared or responsibilities for completing certain activities will be divided.
Q: What questions will I be asked on the Request for Services Application (RSA)?
A: Following is a list of questions on the RSA:
  1. Soundness of the overall approach.
    1. Describe the specific aims and milestones of the proposed study/project.
    2. Indicate how the requested services will enable completion of the aims and milestones.
    3. Discuss any potential obstacles or uncertainties related to the goals of the study/project, and how they would be addressed.
  2. Significance.
    1. Describe the background and significance of the proposed study/project. Note whether the study/project develops or employs novel concepts, approaches, or methodologies.
    2. Discuss how the study/project addresses unmet needs and compare the drug substance/product with others currently available and/or the current standard of care.
    3. Describe the public health benefit and/or impact to patients afflicted with the subject disease or condition.
    4. Describe what patient groups may benefit from successful completion of the study/project and discuss currently available treatments, related clinical trials, and potential contraindications or drug interactions, if known.
  3. Feasibility.
    1. Consider each specific aim and milestone and discuss any feasibility issues and/or potential obstacles to success that have been or will need to be addressed.
    2. Describe any preliminary studies and/or FDA meetings conducted in support of the proposed study/project.
    3. Attach references (preferably in PDF format) containing highly relevant information such as preliminary studies, methods and FDA meeting minutes.
    4. Describe how the proposed study/project addresses any FDA requirements or concerns.
  4. Investigator readiness.
    1. Assuming successful completion of the services associated with the proposed study/project, describe planned next steps for the products, results or outcomes of the study/project. Include a description of quality control and/or regulatory activities, if applicable. Note how successful completion of the study/project will support submission of an IND application, if applicable.
    2. Briefly describe your clinical development plan, including completed and planned preclinical studies. Include information related to dose range finding, safety and toxicity studies, indication(s), target patient population(s), intended route of administration, dosing frequency and duration of treatment.
  5. Intellectual property.
    1. Describe any proprietary rights, confidentiality, or intellectual property issues related to the proposed study/project.
    2. Describe whether any compositions, invention disclosures, methods or products related to the study/project are the subject of current or planned invention disclosures, patent applications or patents.
    3. Discuss whether any of the compositions, products or methods used in the study/project have been patented and/or if the intellectual property rights are not controlled by you or your current institution. Address any potential issues related to freedom to operate within the boundaries of the proposed project.
Q: By what criteria will my Request for Services Application (RSA) be evaluated?
A: Expert reviewers will evaluate RSAs against the following criteria:
  1. Soundness of the overall approach (20%): Has the applicant clearly described the specific aims and milestones for the study/project? How well does the requested service address these aims and milestones? Are there any gaps, unanticipated problems or potential obstacles that have not been addressed in the application?
  2. Significance (20%): Does this study/project address an important disease or unmet need? Will this study/project have a high impact on public health and/or individuals afflicted with the subject disease or condition? Would this study/project benefit patients for which few or no treatment alternatives exist today? Does the study/project develop or employ novel concepts, approaches, or methodologies?
  3. Feasibility (20%): Is it likely that the RSA specific aims and milestones will be completed successfully? Are there any potential obstacles to success that have not been addressed by the applicant? Are there any RSA objectives that might be eliminated or modified without impacting the overall success of the study/project? If applicable, has the preclinical studies plan been reviewed by the FDA? If so, has the applicant adequately addressed all FDA comments or concerns? Is it likely that completion of the RSA objectives will assist with translation of basic observations into one or more therapies for the disease under study, including, when appropriate, the inclusion of proof-of-principle efficacy that would enable progression to the next step towards IND approval?
  4. Investigator readiness (20%): Are there adequate plans (including personnel and environmental support) to manage the products, results or outcomes of the study/project? Are there adequate plans for the rigorous management and quality control of research data or materials returned to the applicant from SMARTT production facilities? Has the applicant adequately described how the study products and/or results will be used? Has the applicant adequately described the next steps after the study/project is completed? Are there any concerns related to the applicant's qualifications or capacity for continued progress towards clinical studies?
  5. Intellectual property (10%): Are there any known or potential issues related to proprietary technology, compositions, methods or products related to the proposed study/project? Has the applicant addressed known or potential issues related to publication or sharing of materials and/or results generated by the study/project?
  6. Discretionary (10%): In view of the five criteria listed above, what is your overall impression of the RSA? Does the study/project have a high potential for successful translation to the clinic? Are there other factors beyond the five criteria that should be considered? Based on your knowledge of the field do you believe this study/project to be highly meritorious and worthy of SMARTT support?
Q: If my request for services is not approved, can I re-apply?
A: Yes. Investigators who have completed additional studies that address reviewers' comments may re-apply for SMARTT services. Such applications must include a statement describing in detail how the new application is materially different from the previous one.
Q: If my request for services is rejected, can I appeal?
A: No. The SMARTT program does not have an appeal process.
Q: After submitting my request for services application (RSA), can I add additional information?
A: No additional information will be accepted.
Q: Can I withdraw my request for services application (RSA) without penalty?
A: Yes. The RSA may be withdrawn at any time prior to an NHLBI decision without penalty. Re-submissions subsequent to withdrawal will be considered as new applications.
Q: Does SMARTT provide services to foreign investigators?
A: No. SMARTT does not provide services to investigators from organizations outside of the United States.
Q: Can you suggest other funding sources for translational research?
A: Yes. A list of alternative funding sources is available here on the SMARTT website.
Q: Should I acknowledge NHLBI's assistance in my publications and presentations?
A: Yes. Investigators receiving SMARTT services must agree to acknowledge SMARTT support in publications and presentations. These citations are critical to ensuring continued support for the SMARTT program. Guidance for acknowledging NHLBI SMARTT support is provided here.
Q: Can investigators approved for SMARTT services work with a different manufacturer other than the SMARTT contractors to develop methods and produce drug products?
A: No. SMARTT services are provided only through its contractors and subcontractors. Funding of services to a third party is not permitted. When an applicant requests transfer of manufacturing services from an outside source, consideration will be given to the following factors, which may affect feasibility and cost:
  • Final formulation of the drug product; are modifications needed?
  • Previous process development data; is the manufacturing process well documented?
  • Current status of toxicology, stability, quality assurance studies, etc.; will such studies need to be repeated?
  • Whether the requested use is different from the approved use, and whether bridging studies would be required.

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