Page 67 - Read Online

Rosales et al. J Transl Genet Genom 2020;4:81-90 I https://doi.org/10.20517/jtgg.2020.12 Page 85

fluid samples. Pilot grants are often a first step for junior investigators, for whom the grant application
is frequently the first formal funding application. The Pilot Core provides multiple mentoring steps for
junior investigators including feedback on initial proposals (including unfunded proposals to improve the
quality of the application), throughout the grant development process, and during project implementation.
Mentoring is done by the NAMDC PI, Statistical PI, Pilot Core PI, local site PI, NAMDC Clinical Liaison,
and additional collaborators as needed.

The NAMDC Pilot Program Committee is responsible for reviewing the pilot/new project proposals.
Applicants for Pilot Projects must be NAMDC members. The NAMDC Pilot Project Committee
recommends funding decisions that best meet the needs and priorities of the consortium and encourage its
growth. Selection for funding is based upon information from written peer review and from the NAMDC PIs
following oral presentation at the annual Face-to-Face meeting. Following the recommendation by the Pilot
Program Committee, the NAMDC Executive Committee makes the ultimate decision as to what pilot/new
projects are funded with input from the RDCRN and approval from the NIH officers. Multicenter studies
are prioritized over single-site study projects. Applicants apply for funding for projects that occur during the
12-month window of the NAMDC yearly cycle of NIH funding. This means that applicants apply for projects
that will be carried out from 1 September to 31 August of the following year. Grantees are expected to submit
a brief quarterly report, a preliminary report presentation at the annual Face-to-Face meeting, and a final
report within two months of the conclusion of the grant.

The NAMDC career enhancement program
There is a severe shortage of physicians trained to diagnose and treat pediatric and adult patients with
mitochondrial disease. The NAMDC Career Enhancement Program is designed to provide training in rare
disease with a focus on mitochondrial medicine. The program consists of the following components:

The NAMDC Fellowship Program, which offers a unique training opportunity to senior postdoctoral clinical
fellows to move on to the attending/assistant professor level in an academic setting as well-trained clinician
scientists. The focus is on translational medicine, teaching of diagnostic expertise, and the development
of clinical trials expertise. NAMDC aims to train the first of a new generation of clinician scientists who
will be well equipped to move promising new treatments for mitochondrial disease into the clinical arena.
Nine training sites are collaborating in this program. These sites - Columbia, San Diego, Seattle, Cleveland,
Hamilton, CHOP, Baylor, University of Colorado, and Mayo Clinic - are all leading institutions in the field of
mitochondrial medicine. World-renowned faculty are to be found at each site. Fellows participate in RDCRN
training courses where they present a summary of their work.

The fellowship program is unified by telemedicine conferences led by the NAMDC Career Enhancement
PI and involves all consortium sites with monthly video webinar conferencing. These conferences are well
attended by faculty, students, and NIH representatives. RDCRN trainees are also invited to participate.

A Career Enhancement program is being developed to include: (1) a didactic core lecture series on Clinical
Trial readiness to be available as an online lecture series; (2) the directors of the 10 Career Enhancement
Sites to post a comprehensive series of lectures on management of mitochondrial disease; and (3) the
Mitochondrial Medicine Society to develop a series of organ-specific mitochondrial disease lectures to be
CME-certified by the United Mitochondrial Disease Foundation (UMDF) and made available online.

Funds provided by the UMDF will be used to attract young physicians, medical students, MD/PhD students,
and recent graduates heading to medical school and the field of mitochondrial medicine. A committee of Site
Directors and UMDF grant reviewers will review proposals. In addition, an annual retreat will take place on
the day following the UMDF annual meeting where the awardees and the NAMDC fellow can interact with