MODIFICATION
A -- In vivo Pharmacology and Animal Model Support for the Therapeutics for Rare and Neglected Diseases (TRND) Program
- Notice Date
- 12/16/2016
- Notice Type
- Modification/Amendment
- NAICS
- 541712
— Research and Development in the Physical, Engineering, and Life Sciences (except Biotechnology)
- Contracting Office
- Department of Health and Human Services, National Institutes of Health, National Institute on Drug Abuse, 6001 Executive Boulevard, Room 3155, MSC 9593, Bethesda, Maryland, 20892, United States
- ZIP Code
- 20892
- Solicitation Number
- HHS-NIH-NCATS-RFI-17-001
- Archive Date
- 2/10/2017
- Point of Contact
- Jeffrey Schmidt, Phone: (301) 402-1488
- E-Mail Address
-
schmidtjr@mail.nih.gov
(schmidtjr@mail.nih.gov)
- Small Business Set-Aside
- N/A
- Description
- Purpose The purpose of this RFI is to obtain knowledge and information to assess the current state of the science and to further assist NCATS in determining the contract type best suited to meeting its needs. Specifically, the NCATS is seeking information regarding the capabilities of existing organizations that execute in vivo pharmacology studies that may require gaining access to existing non-commercially available animal models or to potentially execute needed animal model development/optimization or refinement. The objective is to determine the existing state in capable organizations of pre-emptive readiness: proactive planning and flexibility already in place or have the potential to be put in place to execute simple or highly complex tasks, specifically, but not limited to, the rare and neglected diseases space. This is a Request for Information (RFI) only and does not constitute a commitment, implied or otherwise, that the Department of Health and Human Services (DHHS), National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS), will take procurement action in this matter. This is NOT a solicitation for proposals, applications, proposal abstracts, or quotations. Further, neither DHHS/NIH/NCATS nor the Government will be responsible for any cost incurred in furnishing this information. Background The Therapeutics for Rare and Neglected Diseases (TRND) program at the NCATS has a diverse portfolio of projects that enter our program yearly. NCATS is disease agnostic in its effort to address the challenges of scientific translation and specifically, for the TRND program, drug development in the rare and neglected diseases space. Noting that there are approximately 7,000 rare diseases identified to date, this RFI seeks responses regarding how to efficiently and effectively structure and manage in vivo pharmacology tasks with a wide range of capable organizations (CRO, academic, others with capabilities to subcontract if needed) that will serve the needs generally outlined below, but that are also specifically undefined and diversified with respect to having or gaining access to specific rare or neglected disease animal models at any given time to conduct needed studies. General needs Currently, most of TRND's in vivo pharmacology studies are conducted using a one-off approach with customized arrangements being made for each study, more likely than not, dictated by where the animal model resides. This is often met by lack of available human and other resources to conduct a well powered study to support drug development efforts in a reasonable time frame. Therefore, the purpose of this RFI is to obtain extensive feedback on how a capable organization can effectively and efficiently coordinate resources needed to execute any given study or set of studies (potentially including animal model refinement/optimization or validation) such as those described below that may become the subject of a RFP. • Develop, optimize or use existing validated animal models to conduct in vivo pharmacology studies for rare disorders and neglected diseases for projects accepted into TRND's portfolio. These models are not able to be pre-determined as needs change depending on the disease, hence, access to both small (e.g., mice, rat) and large animal models (e.g., dog, cat monkey, pig) will be needed. • Characterize and validate animal models that require development or further optimization/ refining prior to use for studies involving a therapeutic under development. Therapies can cover a wide range of modalities; small molecules, and biologics (ex. antibody, proteins, cell and gene therapy). • Conduct necessary in vivo studies in validated models to support initial programmatic requirement of efficacy verification and further product development to involve studies such as: o Defining PK-PD relationships o Evaluating a range of endpoints and biomarkers in response to therapeutic candidate o Determining correct route of administration (SC, IV, PO, IT, or ICV) o Determining appropriate dosing parameters o Determining appropriate formulation(s) for administration o Examining toxicity/safety • Conduct, if necessary, in vitro pharmacology studies to test therapeutics in existing, refined or newly developed ex-vivo or cell-based models of the disease. Information Requested Below are several representative scenarios that may arise. Organizations are invited to submit a written response describing how they would specifically approach each of the scenarios outlined below as well as a comprehensive approach of how they would be prepared to overcome anticipated roadblocks/ potential barriers in tackling the general needs outlined above and a response-ready plan for likely scenarios such as those described below. Responses should be tailored to the information requested, taking into consideration the "Purpose" and "Background" described above. Please note that NCATS reserves the option to conduct a follow-up webinar with interested Respondents after the response period has closed. Should NCATS elect to conduct a webinar, Respondents will be notified via email within a reasonable time following the response deadline. Respondents should address their experience and capabilities in the following areas: Scenario 1: TRND needs to test the efficacy of drug (X), a small molecule under development in a rare disease mouse model (Y) for a neurological disorder. The mice are commercially available and your organization has the capability to administer the drug as well as collect the needed biological samples for analysis but lack (1) the disease area expertise and technical capabilities to test the samples (per specific assays accepted by the research community), (2) the know how to appropriately analyze and interpret the data. What would be your approach to getting the study done? How would you approach this same task and conduct the study in a reasonable timeframe if your organization has the disease area and technical expertise but the mice, although accessible, reside in Dr. Jane D's laboratory at New University where resources are limited to ramp-up breeding? Scenario 2: TRND needs (1) to determine the best route of administration of an enzyme replacement therapy, a biologic under development for a lysosomal storage disorder (A) and (2) to determine the appropriate dosing parameters to get a minimum and maximum effective dose in an animal model of this rare disease, (B). The model is not commercially available and resides in Professor John M's laboratory in Global Institute (GI) outside the United States. A very real potential exists that the patent and commercialization office at GI wants reach through rights to the drug once it is commercialized or before the start-up company is acquired by a large biotech or pharma. How would you address this problem? How would you approach this same task if the model was accessible from a non US institute for a cost but animal model (C) is fragile and could not be transported long distance to the U.S. for proposed studies? Scenario 3: TRND needs to define the PK-PD relationship for an antibody therapy in a large animal model of a rare disease (D) that closely mimics the human condition in critically ill infants. The model is acute and has to be generated by a specific procedure that requires additional medical equipment necessary to sustain life. Your organization does not have the technical/scientific/medical/disease area expertise or resources to execute the study. How would you approach this challenge? What if a new but chronic genetic model exists but is not well established/validated in the research community or the primary endpoints/readouts are not as robust as originally believed but TRND needs to have convincing data in an accepted model to justify moving forward with IND-enabling GLP toxicology studies. How would you approach this problem? Scenario 4: TRND just accepted a neglected tropical disease (Z) into its portfolio and studies in a highly infectious mouse model (E) is required to explore different formulations and dosing regimens to achieve desired efficacy. Your staff is highly trained and capable of conducting proposed studies but your facility is only Biosafety Level (BSL) 2 or 3 certified but, BSL 3 or 4 containment is required. How would you approach this problem? What if your facility has the required BSL certification but does not have the appropriately trained staff to generate these acute infectious models and to run such studies? Responses should be limited to 10 pages, not including the cover page, cover letter, table of contents and graphic displays of scenario models for resource integration and/or process maps. RFI responses must include: 1) name and title of the primary point of contact for the response; 2) name and address of the institution or company; and 3) email address and phone number of the primary point of contact Responses are due by January 10, 2017 Disclaimer and Important Notes This Notice does not obligate the Government to award a contract or otherwise pay for the information provided in response. The Government reserves the right to use information provided by respondents for any purpose deemed necessary and legally appropriate. Any organization responding to this Notice should ensure that its response is complete and sufficiently detailed. Information provided will be used to assess tradeoffs and alternatives available for the potential requirement and may lead to the development of a solicitation. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted. Any solicitation resulting from the analysis of information obtained will be announced to the public in Federal Business Opportunities in accordance with the FAR Part 5. However, responses to this Notice will not be considered adequate responses to a solicitation. Confidentiality Any proprietary information should be clearly marked and will be kept confidential in accordance with federal law. The Government reserves the right to use any non-proprietary information in any resultant solicitation(s). Inquiries & Responses Inquiries and RFI Responses should be directed to the NIDA Office of Acquisitions, NCATS Primary Point of Contact (POC): Jeffrey Schmidt, at schmidtjr@mail.nih.gov. Primary Point of Contact: Jeffrey Schmidt Contracting Officer schmidtjr@mail.nih.gov Phone: (301) 402-1488 Contracting Office Address: National Institutes of Health National Center for Advancing Translational Sciences NINDS R&D Contracts Management Branch 6001 Executive Blvd., Suite 3287, MSC 9531 Bethesda, Maryland 20892-9531
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