Pilot Study Development Policy

 

INTRODUCTION:
The Brittle Bone Disorders Consortium (BBDC) initiates and supports collaborative research on osteogenesis imperfecta (OI). In addition to a longitudinal study of OI and interventional studies, the BBDC welcomes both consortium members as well as non-BBDC investigators to propose pilot studies in OI that leverage BBDC research data or infrastructure. A pilot study is an investigation that is not part of the central, BBDC protocol but uses BBDC subjects, samples, or data collected by the BBDC.  In addition, a pilot study may involve acquisition of additional data that is not collected as part of existing BBDC studies.  A pilot study may be at only a limited number of sites or only at a single site or may not involve a site at all but rather be a non-BBD site that is performing data analysis.  Support for pilot studies may be from external sources in addition to established BBDC contract funds.

Both internal and external investigators are encouraged to propose and conduct pilot studies.  Such studies enhance the value of the BBDC and ensure the continued interest of the diverse group of investigators who are critical to the successes of the project as a whole.  These collaborative pilot studies provide an exceptional opportunity for investigators to leverage existing BBDC data or contacts to conduct new projects in a resource-efficient way.

Key Dates
Letter of Intent Due Date: September 30, 2021
Earliest Application Submission Date: October 31, 2021
Application Due Date(s): December 1, 2021
Scientific Merit Review by BBD Steering Committee:  December 6, 2021
Consortium PI Review: December 27, 2021
NIAMS review: January 17, 2022
Earliest Start Date: March 1, 2022
Funding period: April 1, 2022 – April 30, 2023
Funding amount: Up to $15,000
 
PILOT STUDY REVIEW PROCESS:
To ensure that projects align with the goals of advancing knowledge and care for individuals with OI, pilot studies are reviewed and approved by the BBDC Steering Committee. The review process is as follows:

  1. Letters of Intent (LOI) will be reviewed by The BBDC Executive Committee to ensure the proposed project aligns with the goals of the BBDC. If you would like more information on the data available in the BBDC, please contact the Project Manager at diannen@bcm.edu.

    A two-page LOI may be sent to Dianne Nguyen at diannen@bcm.edu and must include the following:
    • Descriptive title of proposed research
    • Name, address, and telephone number of the Principal Investigator(s)
    • Names of other key personnel
    • Participating institutions
    • Research proposal
      • General approach
      • Specific aims
      • Hypothesis
    • Funding source (if requesting funding from the BBDC please include a budget); note, there is no charge for data extraction and basic analyses for not-for-profit organizations
  2. The BBDC Steering Committee will review the proposal and decide whether to advance the proposal to the consortium PIs. Invitations for submission of a full application will be notified by November 1, 2021. Invited pilot study proposals should be submitted via the RDCRN Grants Portal. The project manager will perform an administrative review to ensure that all required information has been provided.
  3. The Consortium PIs will review the proposal and approval will be by majority vote.
  4. For studies involving new data collection, the proposal must then be reviewed and approved by the Program Officer (PO) for the BBDC at the NIAMS/NIH.
  5. Once all requisite approvals have been granted, letters documenting the approval will be sent from the Administrative Core on behalf of the Consortium and from the Project Officer on behalf of the NIAMS/NIH.
  6. Substantial changes to the science or scope of an approved pilot study require review by the Steering Committee.
    • The Study Lead must submit to the BBDC project manager: a revised study proposal with changes tracked, highlighted or bolded; a brief modification request memo summarizing the changes and stating the rationale for the changes. The memo may be addressed to the Steering Committee
    • Substantial changes would include requests for additional biospecimens, significant additional data, or requests to add new outcomes.
    • Formal modification requests are NOT needed for notification of a reduction in needed biospecimens or to add covariates or co-investigators, or to slightly modify the analytic approach. However, all such minor changes must still be communicated to the BBDC project manager.

 
ADDITIONAL REQUIREMENTS FOR A PILOT STUDY

Funding

Funding from the main BBDC contract may be used to support a pilot study. Awards up to $15,000 may be provided depending on funds availability.

IRB Review

  • The use of BBDC data or biological materials is not exempt from review by an Institutional Review Board (IRB). If the Pilot Study will be conducted at a non-BBDC institution, the investigator must provide the IRB approval letter to the Steering Committee before the study begins.
  • Studies that will collect new data from participants must obtain a separate informed consent from all pilot study participants.  This consent should clearly identify the pilot study as one being performed in addition to the main study and inform subjects that their participation in the pilot study is not required for them to continue in BBDC studies.  The IRB approval letter and the informed consent document must be provided to the Steering Committee before study enrollment or data transfer.
  • A singleIRB model must be used if the study involves multiple BBDC sites.

BBDC Sponsorship

  • A BBDC investigator or other approved collaborating investigator is expected to be a co-investigator or principal investigator on a pilot study in the role of BBDC sponsor. The sponsor is responsible for presenting the study to the Steering Committee, monitoring the study to assure continuing compatibility with BBDC guidelines, and serving as a liaison to the BBDC. 
  • Pilot studies from industry partners will be subject to additional agreements with more specific criteria regarding data use and financial remuneration to the OI Foundation for access to the data.
  • In addition, manuscripts and abstracts are generally expected to include a BBDC sponsor, except under circumstances that should be stated and justified as part of the original proposal.

BBDC Site Involvement
All BBDC sites designated for inclusion in the study must have agreement from the respective Principal Investigator.

Data Access and Integration

  • The data collected by the pilot study and any accompanying documentation are first to be provided to the BBDC Administrative Core for integration into the main database. This must occur before the integrated file containing data from the main study will be sent to the pilot study investigators. The pilot study Lead will be given the first and exclusive opportunity to analyze, present and publish data collected under the auspices of the pilot study.  After a reasonable time (in general, 12 months after data collection and cleaning are complete unless the Steering committee deems that the complexity of the analysis warrants extending this timeframe), the pilot study data will be made available for additional uses by other investigators. Collaboration with the pilot study investigators who collected the data is encouraged. It is the responsibility of the pilot study Lead to state in writing to the Steering Committee any special circumstances that would militate against these guidelines for data sharing.  In the spirit of encouraging collaboration, reasonable and justified requests for limiting Steering Committee access to the data will be entertained.
  • A manuscript proposal must be reviewed and approved by BBDC before data from the main study will be provided to pilot study investigators. 


Time Limit for Approval
All pilot studies have 6 months from approval to become active (with or without funding), after which they will be withdrawn and must be resubmitted for re-approval.  This time frame includes establishment of the sIRB as needed.  
 

PUBLICATIONS RESULTING FROM PILOT STUDY
All the publications from a pilot study must be reviewed and approved by the BBDC Publications Subcommittee (P&P), Steering Committee, and reviewed by NIAMS prior to submission, in accordance with BBDC publications policy. Publications must reference the BBDC and BBDC NIH funding resources as per consortium publication guidelines (see BBDC publication policy for details).

 

Revision History
20Aug2021: Adopted Y8 revised date and LOI edits
17Aug2021: Revised Y8 RFA reviewed by BBDC Exec Committee
06Nov2020: Adopted Y7 revised dates
05Nov2020: Revised Y7 dates reviewed by BBDC Exec Committee
09Dec2019: Adopted by the BBDC
04Dec2019: Reviewed by BBDC PIs
25Nov2019: Reviewed by BBDC Exec Committee