NFXF Research Readiness Program | NFXF (original) (raw)

NFXF Research Readiness Program

NFXF Research Readiness ProgramDany Petraska2023-08-29T13:54:31-04:00

NFXF Research Readiness Program

Our mission has always been and continues to be providing unwavering support for every family affected by Fragile X, while relentlessly pursuing a cure. As a patient advocacy organization committed to facilitating research, we wear many hats. We define our role in research as “research facilitation.”

NFXF Research Facilitation

Our research facilitation mission is to advance scientific understanding and treatment research in Fragile X-associated disorders. This is a big mission and means we need to look at research from many angles. We aim to speed up and increase the probability of getting effective FX and FX-related treatments available to the larger FX community. Treatments are not just medications, they can be language, speech, behavior, and other interventions that can be just as impactful and can change the quality of life for individuals and families living with Fragile X. Advancing treatment research can also be supporting researchers through our Research Readiness Program or advocating for federal funding for research in Fragile X-associated disorders.

Our research facilitation objective is to be the preferred point of contact and broker for any Fragile X researcher or Fragile X development leader by:

CRITICAL RESEARCH
INFORMATION FOR ALL

Our team works to facilitate research in a multitude of ways, all designed to assist in meaningful progress toward effective treatments and a cure. Learn more in our video:

Watch

Learn more about NFXF’s work in research facilitation in this presentation from the 2020 17th NFXF International Fragile X Conference Virtual Series featuring Hilary Rosselot and Dr. Craig Erickson, NFXF Scientific & Clinical Advisory and Clinical Trials committees member.

Research Facilitation

Hilary Rosselot
Director
Research Facilitation
National Fragile X Foundation

Dr. Craig Erickson
Medical Director
Fragile X Research & Treatment Center
Cincinnati Children’s Hospital

Research Facilitation Portfolio

Our Research Facilitation portfolio leverages our skills to create meaningful partnerships with all researchers, from your initial concept through the dissemination of results. We support our research facilitation mission through the NFXF Research Readiness Program, our services menu, focused projects, and advocacy:

Engagement /
Focused Projects

NFXF Research Readiness Program

Services

Research Facilitation
Services Menu

Contract Research Organizations (CROs) Support Services

Engagement / Focused Projects

Fragile X Clinical & Research Consortium

International Fragile X Premutation Registry

Advocacy

NFXF Research Readiness Program

NFXF + Researcher Partnership

Multiple Annual Touchpoints

The NFXF Research Readiness Program

The NFXF Research Readiness Program is a collaborative pipeline to support the development and execution of research in Fragile X-associated disorders. The program provides every researcher in the Fragile X field the opportunity to engage with experts and families to ensure proposed research is scientifically-sound, relevant, and patient-centric. We recommend all researchers engage in the program early for support from concept through the conclusion of the research project.

There are phases of the Research Readiness Program, designed to build relationships and bring researchers — you — into the NFXF community:

initial contact

Director of Research Facilitation Screening: Reach out to Hilary Rosselot via email or form to discuss your desired outcomes and research focus.

Review Committee Assignment: Your are then assigned to a review committee, which includes a number of Fragile X experts chosen based on interests and expertise in order to facilitate the best possible consultation.

committee reviews

Concept Reviews: You have the opportunity to do up to two concept reviews: Talking to experts and families about your concept prior to funding and IRB approval. These reviews are meant to discuss on more “big picture” aspects of the project prior to designing protocol, which gives the committee a visual on where the project is going and how they can help. All final recommendations are summarized in an anonymized report after each consultation.

Invitation to Donate: You are invited to donate or pay for the consultation services. These funds help underwrite the NFXF mission of providing unwavering support to families living with Fragile X.

Protocol Design: You then conduct a protocol design with your assigned committee. This review session is meant to continue to help you build a strong foundation to move forward in your endeavors before approaching IRB and applying for grants.

NFXF + Researcher Partnership

NFXF Certification: Once approved through committee consultations, we now feel confident in promoting your research project to the community.

Personalized Engagement Plan: NFXF then works with you to create a personalized engagement plan. Discussion topics include but are not limited to:

How to communicate details about the study on the MyFXResearch portal.
Recruitment and results dissemination plan.
How else we might provide research facilitation services in order to maximize the opportunity for both families and researchers.

Multiple Annual Touchpoints

Going Forward: From there, our partnership and collaboration continues:

We agree to keep one another up to date — as all good partnerships stem from good and open communication!
We reevaluate the personalized engagement plan over time as needed.

initial contact

Director of Research Facilitation Screening: Reach out to Hilary Rosselot via email or form to discuss your desired outcomes and research focus.

committee reviews

NFXF + Researcher Partnership

NFXF Certification: Once approved through committee consultations, we now feel confident in promoting your research project to the community.

Personalized Engagement Plan: NFXF then works with you to create a personalized engagement plan. Discussion topics include but are not limited to:

How to communicate details about the study on the MyFXResearch portal.
Recruitment and results dissemination plan.
How else we might provide research facilitation services in order to maximize the opportunity for both families and researchers.

Multiple Annual Touchpoints

Going Forward: From there, our partnership and collaboration continues:

We agree to keep one another up to date — as all good partnerships stem from good and open communication!
We reevaluate the personalized engagement plan over time as needed.

Questions?

If you have questions about anything research-related, we’d love to hear from you! You can reach out to Anna De Sonia directly, or submit your question or comment through our contact form below.

Hilary Rosselot headshot

Engagement / Focused Projects

MyFXResearch Portal
MyFXResearch Portal is a comprehensive list of current active research in Fragile X-associated disorders, with an easy, user-friendly filter to help interested participants find clinical trials, surveys, and studies they may be eligible for.

NFXF Data Repository™
The NFXF Data Repository is an inclusive repository of all valuable data, including NFXF survey data and NFXF Biobank data (paired clinical and biological data). The NFXF Data Repository allows research data to be readily available to the broader community for future research. This is important because we know that there are many things that lead to advancing treatment research. Having data living on past its initial collection is best for families and cost effective for researchers. The NFXF Data Repository will advance the scientific understanding and treatment research for Fragile X by leveraging previously collected data for new proof-of-concept drug treatments, outcome measure development, and other meaningful scientific work.

Fragile X Clinical & Research Consortium
There are currently over 30 clinics across the nation and a growing number of international sites dedicated to providing those living with a Fragile X condition with comprehensive evaluation and treatment recommendations. The FXCRC clinics provide medical services (including medication evaluation and consultation) supervised by a physician and supported by the latest medical, educational, and research knowledge available. Multidisciplinary services, such as genetic counseling and occupational, speech, language, and behavioral therapies, are also available either at the clinic or by referral. Many of the clinics also participate in collaborative research efforts with other Fragile X clinics and professionals.

International Fragile X Premutation Registry
This is the first international registry specific to individuals who carry the premutation. It was created through a partnership between the UC Davis MIND Institute and the National Fragile X Foundation, and is led by a group of Fragile X professionals from around the world. Registrants provide contact information and basic medical background so that they can be recruited for future research projects focused on the understanding and treatment of Fragile X premutation-associated conditions, including FXTAS (Fragile X-associated tremor/ataxia syndrome).

FORWARD
FORWARD, which stands for Fragile X Online Registry With Accessible Research Database, is a registry and database project created to increase the scientific and clinical understanding of Fragile X syndrome. Supported by the Centers for Disease Control and Prevention, the registry is open to all Fragile X disorders, while the database is open only to those affected by Fragile X syndrome. More than half of the current FXCRC clinics are participating in FORWARD.

Hilary Rosselot,
Director of Research Facilitation
research@fragilex.org
(202) 747-6207

Services

Research Facilitation Services Menu
The menu of our many services dedicated to facilitating research projects and aiding in research recruitment, including: Adding your study or trial to our MyFXResearch Portal, inclusion in our Research Roundup email newsletter + Engagement report, individual emails by state or in full + engagement report, social media posts, boosted social media posts, featured blog posts, webinars, direct mail, and targeted email.

Contract Research Organizations (CROs) Support Services
Our services available to research organizations looking to contract out some of their sponsor responsibilities. Our expertise lends itself well to support various CRO functions, including but not limited to helping with site selection, training, and meeting management.

eConsent
A virtual option for providing participant consent. We believe eConsent can provide more accessibility to research studies, and our eConsent opportunities include videos and other accessible materials to compliment the consent document. eConsent has been integrated into two newer initiatives: the International Fragile X Premutation Registry and the STX209 Reconsent projects.

Hilary Rosselot,
Director of Research Facilitation
research@fragilex.org
(202) 747-6207

Advocacy

Research Funding
Advocating for research funding is a huge part of the NFXF advocacy program. NFXF advocates helped secure approximately $400 million in federal funding for Fragile X research and programming, including research at the National Institutes of Health (NIH) and DOD, and public health programs at the Centers for Disease Control and Prevention (CDC).

Legislation
Advocating for legislation that improves the status quo for adult and families living with intellectual and developmental disabilities is always part of our advocacy work. NFXF advocates have supported legislation and policies like the HEADS Up Act, the ABLE Act, and policies that impact health insurance coverage. The NFXF also advocates for legislation to help promote drug development for rare disorders, such as the Orphan Drug Act.

Advocacy Day
NFXF Advocacy Day is the pillar of the NFXF advocacy program. At the beginning of each year, members of the Fragile X community come to Capitol Hill to advocate for Fragile X. Attendees, including families, self-advocates, friends, and professionals, descend on Capitol Hill to relate their stories about Fragile X-associated disorders, their families, and policies that support Fragile X families and other individuals with intellectual and developmental disabilities.

In-District Meetings
In-district meetings continue to build the relationship between our community and the members of Congress and their staff. Being able to advocate for Fragile X in your home districts is key!

Hilary Rosselot,
Director of Research Facilitation
research@fragilex.org
(202) 747-6207

NFXF Research Readiness Program | NFXF (original) (raw)