IRDiRC-Related Calls

IRDiRC does not provide centralized grants. Its members have a number of funding opportunities available in the field of rare disease research that are in compliance with IRDiRC rules and guidelines. These funding opportunities are listed below.

 

March 2017

Pre-application for the NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules – X02 (NIH, USA)

USA

The NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules [New Therapeutic Uses (NTU)] program is designed to develop partnerships between pharmaceutical companies and the biomedical research community to advance therapeutics development. This innovative program matches researchers with a selection of pharmaceutical industry assets to test ideas for new therapeutic uses with the ultimate goal of identifying promising new treatments for patients. The program was designed to enable efficient 3-way drug repositioning partnerships between academic, government, and pharmaceutical partners.

NCATS is issuing a new round of Funding Opportunity Announcements (FOAs) that support clinical testing of new therapeutic uses for experimental drugs or biologics (Assets) across a broad range of human diseases in adult and pediatric populations.

This FOA invites X02 pre-applications for the NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules.  The X02 pre-application is the first step in the application process for companion FOAs, RFA-TR-17-002 and RFA-TR-17-003; applicants should read the companion FOAs. The X02 pre-applications will be evaluated by outside experts.

Full details of the call

DEADLINES: March 17, 2017

Rare Diseases Registries – PJ-06-2016 ( European Commission)

EUflag_yellow50x34px

Patient registries and databases constitute key instruments to develop clinical research in the field of rare diseases, to improve patient care and healthcare planning. They are the best way of pooling data to achieve a sufficient sample size for epidemiological and/or clinical research. Indeed, registries serve as a recruitment tool for the launch of studies focusing on disease etiology, pathogenesis, diagnosis or therapy.

In the Council Recommendation of 8 June 2009 on an action in the field of rare diseases Member States committed themselves to consider supporting at all appropriate levels, including the EU level, for epidemiological purposes, registries and databases, whilst being aware of independent governance.

In order to support this process and in particular the interoperability of data in rare diseases registries the Commission decided to set up a European Platform on rare diseases registration.

As laid down in Article 12 of the Directive on the application of patients’ rights in cross-border healthcare registries will be a one of the objectives of the European Reference Networks to be set up. Therefore, only approved ERNs are eligible to be co-funded.

The activities to be carried out concern the creation of 3-4 new registries on rare diseases. These registries should constitute key instruments to increase knowledge on rare diseases and develop clinical research. Collaborative efforts to establish data collection and maintain them will be considered, provided that these resources are open and accessible. Registries should be built with the support and according to the standards set up by the European Platform on rare diseases registration and provide all necessary data to the Platform (taking the relevant data protection rules into account).

Full details of the call

DEADLINES: March 21, 2017

April 2017

NeuroNEXT Clinical Trials -U01 (NIH, USA)

USA
This FOA encourages applications for exploratory clinical trials of investigational agents (drugs, biologics, surgical therapies or devices) that may contribute to the justification for and provide the data required for designing a future trial, for biomarker validation studies, or for proof of mechanism clinical studies. Diseases chosen for study should be based on the NINDS’ strategic plan and clinical research interests (www.ninds.nih.gov/funding/areas/index.htm). Successful applicants will be given access to the NeuroNEXT infrastructure. Following peer review, NINDS will prioritize and order trials that are given access to the NeuroNEXT infrastructure. The NeuroNEXT Clinical Coordinating Center (CCC) will work with the successful applicant to efficiently implement the proposed study. The NeuroNEXT Data Coordinating Center (DCC) will provide statistical and data management support. The NeuroNEXT clinical sites will provide recruitment/retention support as well as on-site implementation of the clinical protocol.

Applicants do not need to be part of the existing NeuroNEXT infrastructure.

Full details of the call

DEADLINES: April 4, 2017; August 3, 2017

Diagnostic characterisation of rare diseases – SC1-PM-03-2017 (Horizon 2020, EU)

EUflag_yellow50x34px
The aim of this research should be to apply genomics and/or other –omics and/or other high-throughput
approaches for the molecular characterisation of rare diseases in view of developing molecular diagnoses for
a large number of undiagnosed rare diseases.

Expected Impact:
Providing better and faster means of high quality and clinical utility for the correct diagnosis of undiagnosed
rare diseases for which there is no or unsatisfactory diagnosis available.
• Contribute towards the IRDiRC (International Rare Diseases Research Consortium) objectives
• Foster dissemination of scientific results and knowledge exchange between stakeholders
• Develop knowledge management strategies, with the view of facilitating models of care and access to
the data gathered
• Providing better knowledge for improved family counselling as well as to improve follow-up for
patients and research initiatives
• Gather a big number of patients with similar phenotypes to facilitate match making, to avoid
duplication and to unravel a considerable number of diagnoses
• Pave the way to the development of new therapies and for a better treatment outcome in rare
disease patients

Full details of the call

DEADLINES: April 11, 2017

New therapies for rare diseases – SC1-PM-08-2017 (Horizon 2020, EU)

EUflag_yellow50x34px
Support will be provided to clinical trials on substances where orphan designation has been given by the EC, where the proposed clinical trial design takes into account recommendations from protocol assistance given by the EMA (European Medicines Agency), and where a clear patient recruitment strategy is presented.

Expected Impact:
In line with the objectives of the EU pharmaceutical legislation on orphan medicinal products, proposals shall contribute to advance the development of new therapeutic options with concrete benefits for patients
living with rare diseases.
• Rapid progress in orphan drug development due to well-prepared clinical trials and a multinational multi-centre clinical trial with an appropriate number of patients
• Develop a preliminary assessment of the potential economic and public health aspects of the new therapeutic option
• Contribute to growth of SMEs involved in drug development
• In line with the Union’s strategy for international cooperation in research and innovation, proposals shall contribute towards IRDiRC objectives.

Full details of the call

DEADLINES: April 11, 2017

Human and Social Sciences & Rare Diseases – French Foundation for Rare Diseases

The French Foundation for Rare Diseases launches a fifth call for projects “Social and Human Sciences & rare diseases” in collaboration with the Caisse Nationale de Solidarité and the IRCEM Corporate Foundation. This call for proposals is intended to get a better understanding of individual consequences, family and social specifically related to the scarcity of the disease and increase knowledge on the specific impact of these diseases in terms of disability and of quality of life (activity limitations, restriction of social participation, human rights).

Full details of the call (in French)

DEADLINES: April 27, 2017

June 2017

Palliative Care Needs of Individuals with Rare Advanced Diseases and Their Family Caregivers – R21 (NIH, USA)

USA
This funding opportunity announcement (FOA) seeks to expand knowledge and increase the evidence base for palliative care (PC) in advanced rare diseases, including rare cancers, and to improve physical and psychosocial well-being and quality of life among seriously ill individuals and their family caregivers.

Full details of the call

DEADLINES: June 16, 2017; October 16, 2017

July 2017

Palliative Care Needs of Individuals with Rare Advanced Diseases and Their Family Caregivers – R01 (NIH, USA)

USA
This funding opportunity announcement (FOA) seeks to expand knowledge and increase the evidence base for palliative care (PC) in advanced rare diseases, including rare cancers, and to improve physical and psychosocial well-being and quality of life among seriously ill individuals and their family caregivers.

Research projects submitted to this FOA should focus on palliative and end-of-life care for individuals with advanced, rare diseases and their family caregivers. Studies may consider changing needs that occur across time points and disease progression, as well as ways in which EOLPC and planning can adapt to meet these changing needs. Childhood cancers are not of interest for the purposes of this FOA.

Research projects of interest include, but are not limited to those that:

  • Determine the unique EOLPC needs and preferences (including decision-making) of individuals with advanced, rare diseases and their family caregivers.
  • Contribute seminal data on how needs and preferences of individuals with rare diseases and their family caregivers change across stages of an illness, to provide evidence for future intervention-based studies.
  • Design, test, evaluate, and/or compare EOLPC interventions or models of care to support individuals living with advanced, rare diseases.
  • Design, test, and evaluate, and/or compare EOLPC interventions or models of care to support family caregivers of those living with advanced, rare diseases.
  • To establish key indicators of and appropriate measurements for quality outcomes in individuals with advanced, rare diseases and their family caregivers.
  • Identify transition points at the advanced stages of illness which result in a change in palliative or hospice care needs and develop evidence-based interventions/models of care support during these critical transition points.
  • Foster new research to determine the readiness of individuals with advanced, rare diseases and their families to engage in integrated palliative care.
  • Develop and test innovative technologies for use in palliative and/or hospice care models for individuals with rare diseases.

Full details of the call

DEADLINES: July 5, November 5, 2017

August 2017

Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant -R01 (FDA, USA)

USA
The objective of FDA’s Orphan Products Natural History Grants Program is to support studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics. The ultimate goal of these natural history studies is to support clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for natural history studies that will either assist or substantially contribute to market approval of these products. Applicants must include in the application’s Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States (US) is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the US per year), and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.  Additional information may be required upon request, for example, regarding population estimate and rationale.

Full details of the call

DEADLINES: August 31, 2017

February 2018

Clinical studies of safety and effectiveness of orphan products research project grant (FDA, USA)

USA
The goal of FDA’s OOPD grant program is to support the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the product being developed will be superior to the existing therapy. FDA provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products. Applicants must include in the application’s Background and Significance section documentation to support the assertion that the orphan disease or condition to be studied is a “rare disease or condition” and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development.

The grants are available to any foreign or domestic, public or private, for-profit or nonprofit entity (including State and local units of government). Federal agencies that are not part of the Department of Health and Human Services (HHS) may apply. Agencies that are part of HHS may not apply.  For-profit entities must commit to excluding fees or profit in their request for support to receive grant awards. Organizations that engage in lobbying activities are not eligible to receive grant awards.

Full details of the call

DEADLINES: February 7, 2018 

No deadline – applications on an ongoing basis

Canadian “Rare Diseases: Models & Mechanisms” Network (RDMM, Canada)

Canada
On behalf of the Clinical Advisory Committee of the Rare Diseases: Models and Mechanisms Network, the Co-Chairs would like to invite applications from clinicians and clinician scientists who have discovered novel genes in patients with rare diseases and wish to collaborate with a model organism investigator who is able to carry out functional analysis on an orthologous gene in a model system.

Three categories of Connection applications will be considered:

  1. Novel gene supported by genetic evidence (mutations in the same gene in unrelated patients with a similar phenotype) and functional data is being rapidly sought prior to publication.
  2. Candidate gene for a rare disease in a single patient/family/isolated population such that additional functional data is necessary to support disease-causation.
  3. Known disease gene that is of therapeutic or biological interest to a unique Canadian population or community (e.g. Hutterite, First Nation, French Canadian, patient advocacy group etc.).

Funds Available:
A maximum of $25,000 will be awarded for each catalyst grant.

Eligibility:
Applications may be submitted by any investigator associated with a Canadian Institution.

Full details of the call