SOLICITATION NOTICE
B -- Development of a Population Pharmacokinetic Model for ManNAc in Patients with GNE Myopathy
- Notice Date
- 12/12/2016
- Notice Type
- Presolicitation
- NAICS
- 541690
— Other Scientific and Technical Consulting Services
- Contracting Office
- Department of Health and Human Services, National Institutes of Health, National Heart, Lung and Blood Institute, Rockledge Dr. Bethesda, MD, Office of Acquisitions, 6701 Rockledge Dr RKL2/6100 MSC 7902, Bethesda, Maryland, 20892-7902
- ZIP Code
- 20892-7902
- Solicitation Number
- HHS-NIH-NHLBI-CSB-(HG)-2017-037-DM
- Archive Date
- 1/3/2017
- Point of Contact
- Dorothy Maxwell, Phone: 301-435-0352
- E-Mail Address
-
maxwelld@mail.nih.gov
(maxwelld@mail.nih.gov)
- Small Business Set-Aside
- N/A
- Description
- INTRODUCTION: THIS IS A PRE-SOLICITATION NON-COMPETITIVE (NOTICE OF INTENT) SYNOPSIS TO AWARD A PURCHASE ORDER WITHOUT PROVIDING FOR FULL OR OPEN COMPETITION (INCLUDING BRAND-NAME). THIS IS A NOTICE OF INTENT, NOT A REQUEST FOR A PROPOSAL. A SOLICITATION DOCUMENT WILL NOT BE ISSUED AND PROPOSALS WILL NOT BE REQUESTED. The National Heart, Lung, and Blood Institute (NHLBI) Office of Acquisition (OA) on behalf of the National Human Genome Research Institute, (NHGRI), intends to negotiate and award a purchase order on a non-competitive sole source basis to Enhanced Pharmacodynamics LLC., 701 Elliott Street, Buffalo, New York 14203-1101 to procure the following: Procurement: 1. To develop a population PK model to describe the plasma concentrations of ManNAc and its metabolite sialic acid (Neu5Ac) following single and repeated oral dose administration to GNE myopathy patients; 2. To perform Monte Carlo simulations using the population PK model to generate patient-specific plasma ManNAc and Neu5Ac concentration-time profiles for various ManNAc doses with the goal of finding the lowest possible dose of ManNAc which can produces sustained increases in Neu5Ac. Background: The National Institute of Health (NIH) is the nation's leading medical research agency and the primary Federal agency seeking fundamental knowledge about the nature and behavior of living systems and the application of that knowledge to enhance health, lengthen life, and reduce illness and disability. The National Human Genome Research Institute (NHGRI), mission has expanded to encompass a broad range of studies aimed at understanding the structure and function of the human genome and its role in health and disease. A critical part of the NHGRI mission continues to be the study of the ethical, legal and social implications (ELSI) of genome research. The NHGRI/Medical Genetics Branch (MGB) seeks to identify and understand inherited disorders of metabolism and of human development. MGB investigators focus on human genetics, vertebrate embryology, inborn errors of metabolism, and neurogenetic disorders. Projects performed at the biochemical, molecular, and cell biological levels involve the direct study of human subjects as well as the development and use of experimental model systems, such as zebrafish and mouse. Studies of these rare patients and populations have proven invaluable for advancing the mission of the Branch. NHGRI/Medical Genetics Branch (MGB) are currently performing a Phase 2 clinical trial with the drug ManNAc, a precursor of sialic acid, as a therapeutic agent for patients with GNE myopathy. NHGRI Investigators collected extensive pharmacokinetic (PK = how each patient processed ManNAc and created its product sialic acid) and patient physiologic data (gender, age, weight, body surface, etc). With these data and pathway knowledge, scientists at Enhanced Pharmacodynamics (ePD) can perform so-called "Population Pharmacokinetics" by using unique computer programs to perform modeling to determine optimal concentration of drug and frequency of dosing, to inform for our Phase 3 clinical trial that is under design. ePD can also model different study designs and perform clinical trial simulations to inform about the most optimal trial design. Purpose and Objectives: The purpose of this procurement is the development of a population pharmacokinetic model for ManNAc in patients with GNE Myopathy. NHGRI/Medical Genetics Branch (MGB) expect Phase 3 trial, currently in planning stages, to lead to FDA approval of the drug ManNAc as a therapeutic for GNE myopathy patients. Therefore, an expert rationale for NHGRI chosen drug dosing schedule (timing and concentration) is needed (FDA requirement for drug approval). Enhanced Pharmacodynamics (ePD) offers a very comprehensive statistical way of analyzing at all the data and modeling it in a way that can be related to design of a next trial and is also experienced with (FDA) reporting formats for these data. Enhanced Pharmacodynamics (ePD) is highly revered in the clinical trial modeling field and previous expertise and knowledge with NHGRI Clinical Trial. Scientists at Enhance Pharmacodynamics, LLC (ePD) have over 15 years of experience in this field of work and worked with pharmaceutical industry, government and FDA to perform this work. Scope of Work : A population PK model for ManNAc and sialic acid will be developed using the data from approximately 34 patients with GNE myopathy from Phases 1 and 2 of clinical development. The non-linear mixed effects modeling software NONMEM Version 7.3 will be used to develop the population PK model. The first-order conditional estimation method with interaction (FOCEI) will likely be utilized. Following identification of an appropriate structural population PK model, a formal covariate analysis will be performed to determine if any patient demographic (age, weight, height, body mass index, body surface area or sex), clinical laboratory measures (serum creatinine and estimated creatinine clearance), genetic factors, or other disease-related indices can explain a portion of the inter-individual variability in PK parameters for ManNAc or sialic acid. Selected population PK models during model development along with the final population PK model will be evaluated for robustness using standard population model evaluation criteria (e.g., goodness-of-fit diagnostics, examination of individual predicted overlays, visual predictive checks, analysis of normalized prediction distribution errors, derivation of non-parametric bootstrap confidence intervals for each parameter, etc.). Deliverables / Reporting Requirements : • Slide Presentations summarizing the results of the population PK Analysis and simulations to support dose justification as the final deliverable. • Final data transfer will be organized by NIH/NHGRI as ASCII, Microsoft Excel®, or SAS® transport files. ePD will work closely with the Government as the PK analysis dataset is built and finalized. Data Rights: • FAR Clause 52.227-14, Rights in Data - General (May 2014) • FAR Clause 52.227-17, Rights in Data--Special Works (December 2007). The Government has unlimited rights to all documents and materials produced under this contract. All documents and materials, to include the source codes of any software, produced under this contract will be Government owned and the property of the property of the Government with all rights and privileges of ownership and copyright belonging exclusively to the Government. These documents and materials cannot be used or sold by the contractor without written permission from the Contracting Officer. All materials supplied to the Government will be the sole property of the Government and may not be used for any other purpose. This right does not abrogate any other Government rights. Confidential Treatment of Sensitive Information : • The Contractor shall guarantee strict confidentiality of the information/data that it is provided by the Government during the performance of the contract. The Government has determined that the information/data that the Contractor will be provided during the performance of the contract is of a sensitive nature. Disclosure of the information/data, in whole or in part, by the Contractor can only be made after the Contractor receives prior written approval from the Contracting Officer. Whenever the Contractor is uncertain with regard to the proper handling of information/data under the contract, the Contractor shall obtain a written determination from the Contracting Officer. Privacy Act Clauses : FAR 52.224-1 Privacy Act Notification FAR 52.224-2 Privacy Act FAR 52.239-1 Privacy or Security Safeguards HHSAR Subpart 352.224-70, Privacy Act HHSAR 352.239-72 Security Requirements for Federal Information Technology Resources Privacy Act [5 U.S.C. § 552a(m)(1)]Government Contractor Provision Other Considerations: Deliver a robust Population Pharmacokinetic model that is informative for design of a Phase 3 trial for ManNAc as a therapeutic for GNE myopathy. Anticipated Period of Performance : Upon Award within 4-8 weeks Regulatory Authority: This acquisition is conducted under the authority of the Federal Acquisition Regulations (FAR) Subpart 13.106-1(b) Soliciting from a single source and is not expected to exceed the simplified acquisition threshold, only one responsible source and no other supplies or services will satisfy agency requirements. Statutory Authority : This acquisition is conducted under the authority of the Federal Acquisition Regulation (FAR) Part 13-Simplified Acquisition Procedures, Contracts awarded using FAR Part 13-Simplified Acquisition Procedures are exempt from the requirements of FAR Part 6-Competition Requirements. This requirement will be under the Simplified Acquisition Closing Statement : Industry Classification (NAICS) Code is 541690, Other Scientific and Technical Consulting Services and small business size standard is $15.0M. The acquisition is being conducted under FAR Part 13, simplified acquisition procedures, therefore the requirements of FAR Part 6 B Competitive Requirements are not applicable (FAR Part 6.001). The resultant Contract will include all applicable provisions and clauses in effect through the Federal Acquisition Circular (FAC) 2005-92 (November 18, 2016). This synopsis is not a request for competitive proposals. However, interested parties may identify their interest and capability to respond to this notice. Responses to this notice shall contain sufficient information to establish the interested parties' bona-fide capabilities for fulfilling the requirement and include: unit price, list price, shipping and handling costs, the delivery period after contract award, the prompt payment discount terms, the F.O.B. Point (Destination or Origin), the Dun & Bradstreet Number (DUNS), the Taxpayer Identification Number (TIN), and the certification of business size. All offerors must have an active registration in the System for Award Management (SAM) www.sam.gov." A determination by the Government not to compete this proposed contract based upon responses to this notice is solely within the discretion of the Government. The information received will normally be considered solely for the purposes of determining whether to proceed on a non-competitive basis or to conduct a competitive procurement. All responses must be received by December 19, 2016 and must reference synopsis number HHS-NIH-NHLBI-CSB-(HG)-2016-037-DM by 7:30 a.m. Eastern Time, may be submitted to the National, Heart, Lung and Blood Institute, Office of Acquisition, COAC Services Branch, 6701 Rockledge Drive, Suite 6149, Bethesda, Maryland 20892-7902, Attention: Dorothy Maxwell, Contracting Officer. Response may be submitted electronically to maxwelld@mail.nih.gov. Faxes will not be accepted. Responses will only be accepted if dated and signed by an authorized company representative. "All responsible sources may submit a bid, proposal, or quotation which shall be considered by the agency and shall contain the following: 1. Name of Contracting Organization; 2. Solicitation number; 3. Contract Type; 4. Total Contract Value and Discounts; 5. Description of Requirement; 6. North American Industry Classification System (NAICS) Code; and 7. DUNS, TIN or EIN Number.
- Web Link
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FBO.gov Permalink
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- Place of Performance
- Address: Government and Contractor's Location, United States
- Record
- SN04348894-W 20161214/161212234056-7f70c19247799d5c627982ff8daa0d1e (fbodaily.com)
- Source
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