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.

 

November 2017

Biological response modifier drugs for rare autoimmune diseases (NHS, UK)


The aim of the HTA Programme is to ensure that high quality research information on the effectiveness, costs and broader impact of health technology is produced in the most efficient way for those who use, manage, provide care in or develop policy for the NHS. Topics for research are identified and prioritised to meet the needs of the NHS. Health technology assessment forms a substantial portfolio of work within the National Institute for Health Research and each year about fifty new studies are commissioned to help answer questions of direct importance to the NHS. The studies include both primary research and evidence synthesis.
Research Question:
What is the clinical effectiveness of biological response modifiers (biologics) used for a range of rare autoimmune diseases and are they cost effective?

Full details of the call

DEADLINE: 30 November 2017, 1pm (London time)

 

December 2017

NeuroNEXT Clinical Trials -U01 (NINDS, 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: December 6, 2017

 

February 2018

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

USA
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.
  • NCI: Examine the unique physical and psychosocial issues related to EOLPC that may present among adults with advanced rare cancers (those with an incidence of fewer than 150 per million per year or approximately 40,000 cases or fewer) and their family/informal caregivers, including but not limited to psychological distress, prognostic uncertainty, social isolation, decisional regret, and decision-making in an emotional and ambiguous context.

Types of studies may include innovative methods or approaches such as qualitative, mixed methods, observational, quasi-experimental, and experimental with focus on individual- and family-centered outcomes. It is strongly encouraged that the investigative teams be interdisciplinary and include nurse scientists and early stage investigators.

Full details of the call

DEADLINE: February 5, 2018  then June 5, 2018 and October 5, 2018

10th Joint Call for European Research Projects on Rare Diseases (E-Rare, Europe)


The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear translational research approach. Projects shall involve a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10 000 persons in the European Community, EC associated states and Canada.

The research projects have to focus on hypothesis-driven use of multi-omic integrated approaches for discovery of disease causes and/or functional validation in the context of rare diseases. Research projects submitted must be based on novel ideas stemming from consolidated previous results and must be clearly endowed with a strong translational research orientation, i.e. studies allowing a rapid implementation into public health-related decisions or into the clinics.

Full details of the call

DEADLINES: February 6, 2018 for the pre-proposals and June 19, 2018 for selected full proposals

Project grant: Spring 2018 (CIHR, Canada)


The Project Grant program is designed to capture ideas with the greatest potential to advance health-related fundamental or applied knowledge, health research, health care, health systems, and/or health outcomes. It supports projects with a specific purpose and a defined endpoint. The best ideas may stem from new, incremental, innovative, and/or high-risk lines of inquiry or knowledge translation approaches.

The Project Grant program will:
– support a diverse portfolio of health-related research and knowledge translation projects at any stage, from discovery to application, including commercialization;
– promote relevant collaborations across disciplines, professions, and sectors; and,
– contribute to the creation and use of health-related knowledge.

Full details of the call

DEADLINES: February 6, 2018 for the letter of intent and March 6, 2018 for the application

 

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

DEADLINE: February 7, 2018 

 

Clinical Trial Readiness for Rare Neurological and Neuromuscular Diseases (U01) (NIH, USA)

USA
The purpose of this funding opportunity announcement (FOA) is to support clinical studies that will fill gaps in the design of upcoming clinical trials in rare neurological or neuromuscular diseases by validating clinical outcome measures or biomarkers, or by characterizing cohorts of relevant patients. Through the support of trial readiness studies, NINDS expects to accelerate the initiation of clinical trials for rare diseases and to increase the likelihood of success in those trials.
This FOA is intended to support clinical studies that address current obstacles to the design of upcoming clinical trials in rare neurological and neuromuscular diseases and conditions. The studies supported through this FOA should be clinical observational studies (cross-sectional or longitudinal), and no intervention studies will be supported through this FOA. Appropriate trial readiness projects can be “stand-alone” studies, or they can be ancillary to other, ongoing clinical studies.

Full details of the call

DEADLINE: February 15, 2018  then August 17, 2018

 

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

USA
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.
  • NCI: Examine the unique physical and psychosocial issues related to EOLPC that may present among adults with advanced rare cancers (those with an incidence of fewer than 150 per million per year or approximately 40,000 cases or fewer) and their family/informal caregivers, including but not limited to psychological distress, prognostic uncertainty, social isolation, decisional regret, and decision-making in an emotional and ambiguous context.

Types of studies may include innovative methods or approaches such as qualitative, mixed methods, observational, quasi-experimental, and experimental with focus on individual- and family-centered outcomes. It is strongly encouraged that the investigative teams be interdisciplinary and include nurse scientists and early stage investigators.

Full details of the call

DEADLINE: February 16, 2018  then June 16, 2018 and October 16, 2018

 

August 2018

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, 2018 for the letter of intent and October 15, 2018 for the application 

 

October 2018

Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (FDA, USA)

USA
The objective of FDA’s Orphan Products 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..

Full details of the call

DEADLINE: October 15, 2018

 

Rolling 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

 

Center for Inherited Disease Research (CIDR) High Throughput Sequencing and Genotyping Resource Access (X01) (NHGRI, USA)

USA
The Center for Inherited Disease Research (CIDR) high-throughput genotyping, sequencing and supporting statistical genetics services are designed to aid the identification of genes or genetic modifications that contribute to human health and disease or to enhance existing collections of well-phenotyped specimens by the addition of genotype or next-generation sequence data. The laboratory specializes in genomic services that cannot be efficiently carried out in individual investigator laboratories. CIDR provides the most up-to-date platforms, services and statistical genetic support. This is an NIH-wide initiative that is managed by NHGRI. Information about the current services offered can be accessed via: http://www.cidr.jhmi.edu.

Applications will be reviewed no later than 120 days after receipt of application with one meeting in each of the following windows: September 2017; November 2017; January 2018; March 2018, April-May 2018; July 2018; September 2018; November 2018; January 2019; March 2019; April-May 2019; July 2019; September 2019; November 2019; January 2020;  March 2020; April-May 2020; July 2020.

Full details of the call