Form 1 NICHD 2022 BPCA Prioritization Nomination survey

Overview & Background
OMB # 0925-0766
Expiration Date: 4/2023
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OVERVIEW
The Eunice Kennedy Shriver National Institute of Child Health and Human
Development (NICHD), the National Institutes of Health (NIH), and the U.S.
Department of Health and Human Services (HHS) invite all stakeholders, including
physicians, clinicians, researchers, pediatric organizations and associations,
patient representatives and other interested parties to submit nominations for
pediatric research as it begins the Best Pharmaceuticals for Children Act (BPCA)
annual prioritization process. NICHD is gathering nominations for a Therapeutic
Area (e.g., pediatric condition, subpopulation or setting of care) that requires
further study in children as well as the particular drug, biologic, or medical device
that is being recommended for study in that area.
BACKGROUND
Nominations for the BPCA Priority List of Needs in Pediatric Therapeutics are
solicited as a part of fulfilling NIH’s authority and responsibility to establish a
program for pediatric drug testing and development as outlined in the BPCA
legislation. The Priority List consists of key therapeutic needs in the medical
treatment of children and adolescents; it is organized by Therapeutic Area, which
can be a group of conditions, a subgroup of the population, or a setting of
care. Each Priority List targets a few Therapeutic Areas for discussion and further
prioritization.
For your reference, the 2020 BPCA Priority List of Needs in Pediatric Therapeutics
is located on the BPCA website:

https://www.nichd.nih.gov/research/supported/bpca/prioritizing-pediatrictherapies. NICHD has established a prioritization process through which all
nominations will be considered and evaluated by leading pediatric experts and
stakeholder representatives. The final Priority List is published in the Federal
Register, and sets the agenda for NICHD’s focus on future BPCA-related activities.

Dates & Evaluation Criteria
DATES
There will be two opportunities for public input in 2022:
1) This nomination form will be accepted through June 1, 2022. All responses will
be compiled and reviewed by a committee of stakeholder representatives. The
review will result in a preliminary priority list.
2) There will be an annual stakeholders meeting in December 2022 to review the
final priority list and provide updates on the BPCA drug development program.
EVALUATION CRITERIA
All nominations for research will be reviewed and evaluated on six key criteria, as
follows:
1) Relevance to BPCA mission & goals
2) No disqualifying ethical concerns
3) Feasibility: consideration of the resources available to conduct the study
4) Impact: potential effect on children, society, and delivery of care
5) Population: consideration of the different populations that may benefit from the
research
6) Evidence: consideration of the level of evidence available and current gaps
For further detail on these criteria, see the Request for Information (RFI) in the
National Institutes of Health Guide at [LINK PENDING].

Instructions
On the following pages, you have the opportunity to submit a nomination for
consideration in the BPCA Priority List of Needs in Pediatric Therapeutics. This
nomination form is the primary vehicle by which nominations will be collected.
The form corresponds to one nomination; a party may complete the form multiple
times if interested in submitting more than one nomination.
Nominations must specify the drug, biologic, or medical device to be studied and
the Therapeutic Area in which it will be studied. Nominations will be categorized
by Therapeutic Areas when reviewed.
Please contain all open-ended responses to 100 words or less.

Nomination Submission Form
* 1. Please identify and list the proposed drug, biologic, or medical device to be
investigated, along with the proposed pediatric indication of concern.
Drug
Biologic
Medical Device

2. Please list the proposed Therapeutic Area in which the proposed drug, biologic
or medical device is to be investigated and the potential impact of a study in this
area.

A Therapeutic Area may be a group of conditions, a subgroup of the population, or
a setting of care. For example, the 2009 priority list includes the use of lorazepam
for sedation in the intensive care unit. The indication is Sedation, and the
Therapeutic Area would be Intensive Care.
Please consider the following in your response:
Prevalence of the condition/size of population subgroup/use in setting of care
Morbidity of the underlying condition
Severity, as indicated by mortality rate, hospital length of stay (LOS), and/or
the presence of comorbidities
Frequency of use of the drug, biologic, or medical device for proposed
indication
Availability of alternative treatments, if any
Potential for research results to have multiplicative effects across
Therapeutic Areas or other indications

3. Please submit a single sentence that frames your research question about the
use of the drug, biologic, or medical device. Be sure to include the specific
indication and outcome measure to be investigated.

4. For the proposed nomination, what study design would be most effective in
providing the needed evidence in pediatrics?
Synthesis of existing evidence (e.g., systematic review, meta-analysis)
Primary research using prospective data collection without randomization (e.g.,
observational study)
Primary research through a prospective randomized trial
Other (please describe)

5. Understanding that there is the potential for ethical concerns with research
involving pediatric populations, please describe any potential ethical
considerations relevant to the nominated research study. Please describe
potential ethical considerations with respect to both the Therapeutic Area as well
as the proposed drug, biologic, or medical device.

6. Please describe any existing evidence available regarding the proposed
research question and the feasibility of the proposed research question, as it
relates to the following considerations:
Existing evidence: Existence of an unmet need or gap in the available
evidence; relevance to uncertainties for physicians, such as variations in
clinical care or controversy over what constitutes appropriate clinical care;
any economic analyses allowing calculation of health utilities; any existing
efforts underway to conduct research in this area
Feasibility: Resources required to conduct the study, taking into account the
availability of patients, sites, and principal investigators. Please acknowledge
any existing networks or resources available.

7. Please describe any other information you would like to share that supports
your nomination.

Areas for Continued Discussion/Collaboration
In addition to submitting therapeutic priorities for consideration, the BPCA
program is also interested in gathering data on areas that may impact the success
of clinical research. The following is a short list of areas for continued discussion
and potential areas ripe for collaborative actions.
Please indicate if you have expertise or information to assist in advancing discussion and
collaboration in the following areas:

8. Models for collaborative workforce development in pharmacology research
Yes
No
If yes, please specify.

9. Existing resources of clinical and translational biomarkers that can be
considered for validation
Yes
No
If yes, please specify.

10. Existing models for multi-dimensional inclusion of and dissemination practices
to underserved populations (including children, women, and underrepresented
minorities) in clinical drug trials
Yes
No
If yes, please specify.

11. Best practices for widespread dissemination of clinical research data to a broad
audience of researchers, clinicians, and patients/parents
Yes
No
If yes, please specify.

12. Please rank the following 4 areas for continued discussion in terms of priority
with 1 being highest priority and 4 being lowest priority.
´
Models for collaborative workforce development in pharmacology research
´
Existing resources of clinical and translational biomarkers that can be considered for
validation
´
Existing models for multi-dimensional inclusion of and dissemination practices to
underserved populations (including children, women, and underrepresented minorities) in
clinical drug trials
´
Best practices for widespread dissemination of clinical research data to a broad audience of
researchers, clinicians, and patients/parents

13. Please indicate if each of the following areas are high priority, medium priority,
or low priority:
High Priority

Models for
collaborative
workforce
development in
pharmacology
research
Existing
resources of
clinical and
translational
biomarkers that
can be considered
for validation
Existing models
for multidimensional
inclusion of and
dissemination
practices to
underserved
populations
(including
children, women,
and
underrepresented
minorities) in
clinical drug
trials
Best practices for
widespread
dissemination of
clinical research
data to a broad
audience of
researchers,
clinicians, and
patients/parents

Medium Priority

Low Priority

14. Are you interested in joining a public assembly to discuss advancement in any
of the areas above? If so, select the box(es) next to your preferred assemblies.

Models for collaborative workforce development in pharmacology research
Existing resources of clinical and translational biomarkers that can be considered for
validation
Existing models for multi-dimensional inclusion of and dissemination practices to
underserved populations (including children, women, and underrepresented
minorities) in clinical drug trials
Best practices for widespread dissemination of clinical research data to a broad
audience of researchers, clinicians, and patients/parents

Nominator Information
Completion of the following information is optional. If you would like to receive
future BPCA communication, please provide contact information below. The
answers you give will not influence the progress of your submission.
15. Please select a description that best describes your role or perspective:
Employer
Government - Programs (e.g., Medicare, Medicaid)
Government - Research
Health Care Provider
Health Plan/Insurance Carrier
Manufacturer (Device)
Manufacturer (Drug or Biologic)
Medical Administrator
Nonprofit/Policy Institute
Patient/Family (including family caregiver)
Professional Association
Public/Consumer
Researcher

16. Who are you representing with your response?
Self
Organization

17. How did you hear about the opportunity to submit a nomination for the BPCA
Priority List of Needs in Pediatric Therapeutics?

18. If you are not already receiving BPCA communications, would you like to be
added to the BPCA Stakeholders Distribution List?
Yes
No
Please note that this survey is anonymous so we cannot respond to any questions/comments
unless you provide your contact details. If you are interested in joining a public assembly or
you would like to be added to the BPCA Stakeholders distribution list, please complete the
section below. Otherwise, contact information is optional.

19. Contact Information
Name
Organization
Title
Email address
Phone number