Notice Number: NOT-TR-18-025

Key Dates
Release Date: May 9, 2018

NOT-TR-24-007 - Notice of Corrections to PAR-24-054 Limited Competition: High Impact Specialized Innovation Programs in Clinical and Translational Science for UM1 CTSA Hub Awards (RC2 Clinical Trials Optional)

Issued by
National Center for Advancing Translational Sciences (NCATS)

Purpose

This Notice informs the research community of the NCATS policy regarding support of phase III clinical trial activities for a rare disease or condition. The policy is designed to ensure compliance with the 21st Century Cures Act (Cures Act) as well as all pertinent statutes and existing NIH and NCATS policies; and considers the urgency of developing new treatments for rare diseases.

Background
With the signing of the 21st Century Cures Act (Cures Act) into law in December 2016, the Public Health Service (PHS) Act was amended to allow NCATS to support clinical trial (CT) activities through the end of phase II for all diseases or conditions, and through the end of phase III for a rare disease or condition (as defined here). However, the PHS Act requires NCATS to complete several steps before support can be provided for CT activities in phase III for a rare disease or condition: give public notice of its intent; determine that no other entity plans to conduct a similar clinical trial; and ensure that the Federal Government’s liability will not be increased beyond the award value of the Center’s support.

Implementation
To ensure compliance with the PHS Act, the following requirements must be satisfied prior to providing any NCATS support for a phase III CT activity for a rare disease or condition:

1. Public notification of the NCATS potential funding for phase III clinical trial activities:
Interested parties are directed to the NCATS Funding Policy website where individual notices and opportunities for public comment will be posted when NCATS contemplates providing support for a phase III clinical trial activity for a rare disease or condition.

2. Inclusion of language in funding announcements:
All NCATS solicitations, including, but not limited to, funding opportunity announcements (FOA) and requests for proposals (RFP), that provide support for phase III clinical trial activities will state the statutory requirements for NCATS support of a phase III clinical trial activity for a rare disease or condition. Such solicitations will also convey to potential applicants that NCATS is hereby authorized to publicly disclose a synopsis of the proposed clinical trial activity and any other necessary information included in the submission as part of a public notice as required for compliance with statutory requirements.

3. Ensure that the Federal government’s liability will not be increased beyond the award value of NCATS support:
The Center will address the statutory requirement that NCATS may support clinical trial activities through the end of phase III for a treatment for a rare disease or condition so long as the Center ensures that support of the clinical trial activities in phase III will not increase the Federal Government’s liability beyond the award value of the Center’s support by including the following language in solicitations and award notices providing support for phase III clinical trial activities: NCATS is not liable for nor legally obligated to provide funds or resources in support of clinical trial activities in phase III beyond the award value of the Center’s support.

4. Determination whether a proposed clinical study meets the definition of a phase III clinical trial activity for a rare disease or condition:
If NCATS receives an application or proposal that requests support for a phase III CT activity for either a rare or common disease or condition, the relevant NCATS staff, in addition to any others deemed appropriate, will make a determination regarding: a) the clinical trial phase of the proposed activity, and b) the classification of the disease or condition under study as a rare disease or condition. Potential applicants will be apprised of the results of the assessment.

5. Documentation of CT phase and rare disease or condition classification determinations:
Determination of CT phase and classification of the disease, and the rationale for such determinations, will be documented in the application or proposal record, as specified in specific solicitations. If the application or proposal is determined to request support for a phase III CT activity for a rare disease or condition, it will be subject to requirements of all applicable NIH and NCATS policies before it can be deemed compliant.

6. Public notice of and opportunity to comment on the contemplation of NCATS to support a phase III CT activity for a rare disease or condition:
If NCATS contemplates funding an application or proposal requesting support for a phase III CT activity to study a rare disease or condition, there will be a public notice with an opportunity to submit a reply for a period of at least 120 days to determine whether any public or private organization has timely plans to further the clinical trial activities or conduct clinical trials of a similar nature beyond phase II.

7. Determination whether an organization plans to conduct a similar clinical trial:
Following the public notice period, any responses to the notice will be analyzed by relevant NCATS staff, in addition to any others deemed appropriate, to ascertain whether any responses provide a credible indication that a public or private organization has timely plans to further the clinical trial activities or conduct clinical trials of a similar nature beyond phase II. If NCATS receives at least one response that is determined to provide a credible indication that a public or private organization has timely plans to further the clinical trial activities or conduct clinical trials of a similar nature beyond phase II within the specified time period for public comment, NCATS will not support the phase III CT activities proposed in the application or proposal.

Funding Considerations
If the above policy guidance is followed in full and NCATS has sufficiently fulfilled its statutory requirements, then the application or proposal will be deemed in compliance with this NCATS funding policy and eligible to spend funds in support of a phase III CT activity for a rare disease or condition, pending compliance with all other relevant NIH and NCATS policies, and programmatic prioritization.

This NCATS policy determines how the Center will support phase III clinical trial activities. As indicated in the amended PHS Act, NCATS may develop and provide infrastructure and resources for all phases of clinical trials research. The Center may also support clinical trial activities through the end of phase III for a treatment for a rare disease or condition, subject to the requirements delineated above. The Center intends to support novel and innovative clinical trials research, with an over-arching goal of addressing and improving clinical trial operations. NCATS does not intend to invest resources into supporting routine clinical trial operations that are widely and commercially available.

Prior consultation with NCATS to ascertain the applicability of the policy to a potential application or proposal is strongly encouraged.

Additional information may be found at the NCATS Funding Policy website.

Inquiries

Please direct all inquiries to:

NCATS Policy Officer
Telephone: 301-594-7328
Email: NCATS_Phase_III_CT_P@mail.nih.gov