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Guide for the Review of (R01) NINDS Pilot Clinical Studies Applications


PURPOSE

The purpose of a PILOT STUDIES FOR CLINICAL TRIALS IN NEUROLOGICAL DISORDERS grant (for brevity referred to as NINDS Pilot Studies grant) is to obtain preliminary data and conduct studies to support the rationale for a subsequent full-scale clinical trial of an intervention to treat or prevent neurological disease. For further information on this funding mechanism see the NIH GUIDE.

REVIEW CRITERIA AND FORMAT OF THE CRITIQUE

Note: Applicants will receive the reviewer's verbatim critiques. It is important to revise these to reflect any change of opinion resulting from the discussion of the application.

CRITIQUE: Please address, in five individual sections, each criterion listed below.

Review Criteria

The goals of NIH-supported research are to advance our understanding of biological systems, improve the control of disease, and enhance health. In the written comments reviewers will be asked to discuss the following aspects of the application in order to judge the likelihood that the proposed research will have a substantial impact on the pursuit of these goals. Each of these criteria will be addressed and considered in assigning the overall score, weighting them as appropriate for each application. Note that the application does not need to be strong in all categories to be judged likely to have major scientific impact and thus deserve a high priority score. For example, an investigator may propose to carry out important work that by its nature is not innovative but is essential to move a field forward.

(1) Significance: Does this study address an important problem? If the aims of the application are achieved, how will scientific knowledge be advanced? What will be the effect of these studies on the concepts or methods that drive this field?

In evaluating significance, the reviewers should also consider the state of equipoise in the medical and patient communities and the potential impact of the proposed intervention on health care and quality of life.

(2) Approach: Are the conceptual framework, design, methods, and analyses adequately developed, well-integrated, and appropriate to the aims of the project? Does the applicant acknowledge potential problem areas and consider alternative tactics? In addition to the standard evaluation criteria described above, applications will also be reviewed with respect to the following:

The adequacy of plans for sequence of clinical studies, including the proposed pilot study, that will produce a definitive clinical trial; The scientific soundness of the proposed methodology, such as translation of the clinical question into statistical hypotheses, selection of outcome measure(s), inclusion and exclusion criteria, plans for randomization and masking, secondary questions (including capacity for post hoc analyses); The completeness and quality of the protocol and standardized procedures that will be used for this pilot study; The ethical aspects of the study and the appropriateness of subject safety protections; And the adequacy of quality control procedures. Reviewers will also evaluate plans for data management and analyses. However, sophisticated data management procedures are not required for a pilot study, although this study may be used to test these procedures. Likewise, description of specific methods to be used for data analyses is not required; it is assumed that in most cases, pilot studies will provide mostly descriptive statistics. Reviewers will evaluate plans for demographic subgroup analyses, if applicable.

In evaluating plans for the sample size for the pilot, the reviewers should take into account that pilot studies need not be oriented toward detecting treatment differences; however, the data from this study should provide a basis for reliable sample size estimates for future trials.

(3) Innovation: Does the project employ novel concepts, approaches or method? Are the aims original and innovative? Does the project challenge existing paradigms or develop new methodologies or technologies?

(4) Investigator: Is the investigator appropriately trained and well suited to carry out this work? Is the work proposed appropriate to the experience level of the principal investigator and other researchers (if any)? The reviewers should consider training and expertise in the clinical problem and the proposed intervention, and training and expertise in clinical trials.

(5) Environment: Does the scientific environment in which the work will be done contribute to the probability of success? Do the proposed experiments take advantage of unique features of the scientific environment or employ useful collaborative arrangements? Is there evidence of institutional support?

OVERALL EVALUATION: In one paragraph, briefly summarize the most important points of the Critique, addressing the strengths and weaknesses of the application in terms of the five review criteria. Recommend a score reflecting the overall impact of the project on the field, weighting the review criteria, as you feel appropriate for each application.

HUMAN SUBJECTS: If Exemptions are claimed, express any comments or concerns about the appropriateness of the exemption(s) claimed (e.g., for Exemption 4, is it clear that the information will be recorded by the investigator so that subjects cannot be identified directly or indirectly?). If No Exemptions are claimed, express any comments or concerns about the appropriateness of the applicant's responses to the six required points. Discuss whether the risks to the subjects are reasonable in relation to the anticipated benefits to the subjects and/or in relation to the importance of the knowledge that may reasonably be expected to result from the research.

GENDER, MINORITY AND CHILDREN SUBJECTS: If human subjects are involved examine whether the minority and gender characteristics of the sample are scientifically acceptable and consistent with the aims of the project, using the categories of "1" to "4" as follows. Also examine whether there is appropriate inclusion of children (individuals under the age of 21). Also determine whether the research is a Phase III clinical trial.

Category

Gender (G)

Minority (M)

Children (C)

1

Both Genders

Minority & non-minority

Children & adults

2

Only Women

Only minority

Only children

3

Only Men

Only non-minority

No children included

4

Gender Unknown

Minority representation unknown

Representation of children unknown

Evaluate acceptability as "A" (acceptable) or "U" (unacceptable). If you rate the sample as "U", consider this feature a weakness or deficiency in the design of the project and reflect it in the overall score.

NOTE: To the degree that acceptability or unacceptability affects the investigator's approach to the proposed research, such comments should appear under "Approach" in the five major review criteria above, and should be factored into the score as appropriate.

In evaluating HUMAN SUBJECTS and GENDER, MINORITY AND CHILDREN SUBJECTS please refer to the enclosed information document for detailed guidance.

BIOHAZARDS: Note any materials or procedures that are potentially hazardous to research personnel and indicate whether the protection proposed will be adequate.

BUDGET: Evaluate the direct costs only. Do not focus on detail. Determine whether the total budget is appropriate for the project proposed. Provide a rationale for suggested modification in amount or duration of support. Any recommended reductions to modular budgets should be in modules of $25,000.

OTHER CONSIDERATIONS (for Administrative Notes in the Summary Statement): These comments are useful to NIH but should not influence your overall score.

FOREIGN: If the applicant organization is foreign, comment on any special talents, resources, populations, or environmental conditions that are not readily available in the United States or that provide augmentation of existing U.S. resources. In addition, indicate whether similar research is being performed in the U.S. and whether there is a need for such additional research. These aspects do not apply to applications from U.S. organizations for projects containing a significant foreign component.

Last updated November 24, 2008