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Stem Cell Information

Approval Process for the Documentation of Compliance with NIH Guidelines on the Use of Human Pluripotent Stem Cells in NIH Intramural Research, January 16, 2001

Release Date: January 16, 2001

This Guideline Notice has been superceded as it pertains to human embryonic stem cell research. Please refer to the Notice of Withdrawal of NIH Guidelines for Research Using Pluripotent Stem Cells.

National Institutes of Health

Introduction

On August 25, 2000, the National Institutes of Health (NIH) published an outline of procedures for the approval of the use of human pluripotent stems cells in NIH supported intramural and extramural research. These procedures are based on the National Institutes of Health Guidelines for Research Using Human Pluripotent Stem Cells, hereafter referred to as the NIH Guidelines, published in the Federal Register and effective on August 25, 2000. Because the NIH Guidelines contained a few incorrect citations and other minor errors, a notice of correction (65 FR 69951) was published on November 21, 2000. This second notice details the specific procedures that must be followed by NIH intramural investigators proposing to use NIH funds for research using human pluripotent stem cells derived from human embryos and human fetal tissue.

All intramural research undergoes regular scientific peer review through established procedures in each Institute/Center (IC). In addition, for research that proposes the use of human pluripotent stem cells, there will be a formal review of documentation of compliance with the NIH Guidelines. This latter review will be conducted by the Human Pluripotent Stem Cell Review Group (HPSCRG) which is a working group of the NIH Center for Scientific Review Advisory Council (CSRAC). The process for documenting compliance with the NIH Guidelines is separate from the intramural scientific review process. The two processes will take place in parallel in order to ensure that all aspects of scientific review and review of compliance are considered in a timely manner. Intramural investigators proposing research using human pluripotent stem cells must be mindful of the requirements and deadlines for both processes in order to avoid delays in the initiation of proposed research. NIH will not allow funds to be used for research utilizing human pluripotent stem cells derived from human embryos or human fetal tissue until appropriate approvals have been obtained. Evidence of compliance with the NIH Guidelines does not affect the scientific merit review of an intramural research proposal nor does it ensure funding by the Institute/Center (IC).

Review of compliance with the NIH Guidelines is a two-stage process. Any request proposing to use NIH funds for research utilizing human pluripotent stem cells derived from human embryos or human fetal tissue must include the submission of compliance documentation to the HPSCRG. The HPSCRG members will review the submitted documentation and their individual recommendations will be forwarded to the CSRAC for final consideration. When the HPSCRG receives compliance documentation in support of a request for funds that proposes the use of a line of human pluripotent stem cells that has not been previously reviewed by HPSCRG and recommended to, and approved by, the CSRAC, the HPSCRG review will take place in a public meeting. Review of compliance documentation for the use of a previously NIH approved cell line will not take place in a public meeting of the HPSCRG. The final approval of documentation of compliance will always take place in a public meeting of the CSRAC.

SUBMISSION OF DOCUMENTATION

Timetable

Documentation is to be submitted by intramural investigators to their IC Scientific Director, who will subsequently forward compliance materials to the Office of Science Policy, Office of the Director, NIH. Simultaneously, a copy should also be sent to the Deputy Director for Intramural Research. An original and two copies of the documentation are to be submitted to:

NIH Office of Science Policy
Attention: HPSCRG
Building 1, Room 218
MSC 0166
9000 Rockville Pike
Bethesda, MD 20892
(301) 594-7741

The schedule for receipt and review by HPSCRG and CSRAC follows:

Materials received by Office of Science Policy Review by HPSCRG Consideration by CSRAC

March 15

April

May

July 15

August

September

November 15

December

January

Packages of documentation received by the Office of Science Policy after the established deadlines will be held for the next cycle of HPSCRG and CSRAC consideration.

Materials to be submitted to the NIH Office of Science Policy for research on human pluripotent stem cells derived from embryos and fetal tissue are set forth below at parts I and II, respectively.

  1. HUMAN PLURIPOTENT STEM CELLS DERIVED FROM EMBRYOS

       

    1. MATERIALS TO BE SUBMITTED TO THE OFFICE OF SCIENCE POLICY

       

         

      1. Cover Page with the following information (See sample below)

         

           

        1. Name of the Principal Investigator (PI).

           

           

        2. Title of the intramural research protocol.

           

           

        3. Name, title, telephone number, fax number and email address of the Laboratory Chief and the Scientific Director submitting the information.

           

           

        4. Signature of Laboratory Chief and the IC Scientific Director.
        SAMPLE COVER PAGE
        Principal Investigator: Roberta Dilan, M.D.
        Title of Project: Use of Human Pluripotent Stem Cells to Treat XYZ Disease
        Anticipated Date of Submission of the Intramural Research Protocol: February 1, 2001
        Submitted by: Name of Institute/Center
        IRP Official: Eileen Smith, Ph.D.
        Scientific Director
        Name of Institute/Center
        555-555-1212 (voice)
        555-555-2121 (fax)
        smithe@xxxxx.nih.gov

         

      2. Documentation of compliance with the NIH Guidelines specific to research using human pluripotent stem cells derived from human embryos.

         

           

        1. An assurance signed by the IC Scientific Director (i.e., Institutional official) that the pluripotent stem cells were derived from human embryos in accordance with the conditions set forth in Section II.A.2 of the NIH Guidelines and that the Institute/Center will maintain documentation in support of this assurance. The official's signature will attest that the IC has documentation (or an assurance) from the person(s) who derived the human pluripotent stem cells stating that the derivation process was conducted in accordance with the NIH Guidelines (See NIH Guidelines, Section II.A.1.a). Specifically, the IC assurance should include a statement that the embryo(s) used to derive the pluripotent stem cells were created through in vitro fertilization for the purpose of fertility treatment and that the embryos had not reached the stage at which the mesoderm is formed at the time of donation. The assurance should also include statements that the donation of the embryo was voluntary; that the researcher who derived or proposed to derive the cells was a different person than the physician who provided fertility treatment; that the individuals undergoing fertility treatment were approached about consent for the donation of embryos to derive pluripotent stem cells only at the time of deciding the disposition of frozen embryos that were in excess of clinical need; that the donor placed no restrictions regarding the individual(s) who may be the recipients of transplants derived from the human embryo; and that the required informed consents and Institutional Review Board (IRB) approval were obtained.

           

           

        2. A sample of the informed consent document for the donation of the embryo(s) for the purpose of deriving human pluripotent stem cells that was used in this research (with patient identifying information removed). A description of the required elements in the informed consent process is set forth in Section II.A.2.e of the NIH Guidelines.

           

           

        3. An abstract of the scientific protocol that was used to derive the human pluripotent stem cells, obtained from the person(s) who derived the human pluripotent stem cells, describing the methods and materials used (See NIH Guidelines, Section II.A.1.c)

           

         

      3. Other Documentation

         

           

        1. Documentation of the IRB approval of the derivation protocol (See NIH Guidelines, Section II.A.1.d)

           

           

        2. An assurance signed by the IC Scientific Director that the human pluripotent stem cells to be used in the proposed research were obtained through donation or through payment that does not exceed the reasonable costs associated with the transportation, processing, preservation, quality control, and storage of the stem cells) See NIH Guidelines, Section II.A.1.e).

           

           

        3. An assurance signed by the IC Scientific Director that the proposed use of human pluripotent stem cells is not a class of research that is ineligible for NIH funding as set forth in Section III of the NIH Guidelines (See NIH Guidelines, section II.A.1.g).

           

           

        4. The Principal Investigator's written consent to the disclosure of all material submitted as part of this documentation of compliance, as necessary to carry out the public review and other oversight procedures set forth in the NIH Guidelines (See NIH Guidelines, Section II.A.1.h).

           

       

    2. DECISIONS

       

      HPSCRG may ask the IC to provide additional materials to clarify the documentation of compliance with the NIH Guidelines. This could delay completion of HPSCRG review. Following the meeting of the CSRAC, the NIH Office of Science Policy will convey the results of the compliance review to the principal investigator, the Deputy Director for Intramural Research, and the potential funding IC.

     

  2. HUMAN PLURIPOTENT STEM CELLS DERIVED FROM FETAL TISSUE

     

       

    1. MATERIALS TO BE SUBMITTED TO THE OFFICE OF SCIENCE POLICY

       

         

      1. Cover Page with the following information (See sample below)

         

           

        1. Name of the Principal Investigator (PI).

           

           

        2. Title of the intramural research protocol.

           

           

        3. Name, title, telephone number, fax number, and email address of the Laboratory Chief and the Institute/Center Scientific Director submitting the information.

           

           

        4. Signature of Laboratory Chief and the Institute/Center Scientific Director.

           

          SAMPLE COVER PAGE
          Principal Investigator: Roberta Dilan, M.D.
          Title of Project: Use of Human Pluripotent Stem Cells
          to Treat XYZ Disease
          Anticipated Date of Submission of the Intramural Research Protocol: February 1, 2001
          Submitted by: Name of Institute/Center
          IRP Official: Eileen Smith, Ph.D.
          Scientific Director
          Name of Institute/Center
          555-555-1212 (voice)
          555-555-2121 (fax)
          smithe@xxxxx.nih.gov

         

      2. Documentation of compliance with the NIH Guidelines specific to research using human pluripotent stem cells derived from fetal tissue.

         

           

        1. An assurance signed by the IC Scientific Director that the pluripotent stem cells were derived from human fetal tissue in accordance with the conditions set forth in Section II.B.2 of the NIH Guidelines and that the Institute/Center will maintain documentation in support of this assurance. The official's signature will attest that the IC has documentation (or an assurance) from the person(s) who derived the human pluripotent stem cells stating that the derivation process was conducted in accordance with the NIH Guidelines (See NIH Guidelines, Section II.B.1.a). The assurance should also include statements that the research complies with all of the requirements of 42 U.S.C. 289g-1, 42 U.S.C. 289g-2(a) and (b) and 45 C.F.R. 46.210; that the required informed consents were obtained; and that IRB approval was obtained.

           

           

        2. A sample of the informed consent document that was used for the donation of the human fetal tissue for the purpose of deriving the human pluripotent stem cells (with patient identifying information removed) (See NIH Guidelines, Section II.B.1.b). A description of the required elements in the informed consent process is given at Section II.B.2.b of the NIH Guidelines.

           

           

        3. An abstract of the scientific protocol that was used to derive the human pluripotent stem cells, obtained from the person(s) who derived the human pluripotent stem cells, describing the methods and materials used (See NIH Guidelines Section II.B.1.c).

           

         

      3. Other Documentation

         

           

        1. Documentation of IRB approval of the derivation protocol (See NIH Guidelines, Section II.B.1.d).

           

           

        2. An assurance signed by the IC Scientific Director that the human pluripotent stem cells to be used were, or will be, obtained through donation or through payment that does not exceed the reasonable costs associated with the transportation, processing, preservation, quality control, and storage of the stem cells (See NIH Guidelines, Section II.B.1.e).

           

           

        3. An assurance signed by the IC Scientific Director that the proposed use of human pluripotent stem cells is not a class of research that is ineligible for NIH funding, as set forth in Section III of the NIH Guidelines (See NIH Guidelines, Section II.B.1.g).

           

           

        4. The Principal Investigator's written consent to the disclosure of all material submitted as part of this compliance documentation, as necessary to carry out the public review and other oversight procedures set forth in the NIH Guidelines (See NIH Guidelines, Section II.B.1.h).

           

       

    2. DECISIONS

       

      HPSCRG may ask the IC to provide additional materials to clarify the documentation of compliance with the NIH Guidelines. This could delay the completion of the HPSCRG review. Following the meeting of the CSRAC, the NIH Office of Science Policy will convey the results of the compliance review to the principal investigator, the Deputy Director for Intramural Research, and the potential funding Institute or Center within the NIH.

       

    3. FURTHER INFORMATION

       

      Questions regarding this process should be directed to stemcell@mail.nih.gov or call the Office of Science Policy at (301) 594-7741.