Disease or Translational Resources

In this section you will have the opportunity to explore information regarding:
Patient registries,
Natural history,
Patients’ information as organizations, toolkits for patients, new-born screening programs, undiagnosed diseases programs (UDPs), EU planned cross-border treatment
Target Patient Value Profile
Databases/Tools (ODDG)
Networks
Biobanks
Biomarkers (Development, guidelines, validation, qualification, genomic, cancer, pain therapeutics)

  1. Showing 83 results for:
  2. Research and Drug Development
  3. Disease or Translational Resources
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Initiatives for undiagnosed diseases

Published by IRDiRC

Genetic ConditionGenotype and Phenotype MatchmakingPatient InformationResearch and Drug DevelopmentUndiagnosed Disease Programme

The purposes of Undiagnosed Diseases Programs (UDPs) are to provide patients with an unknown genetic condition a diagnosis and to find the correlation between genotype and phenotype; to share globally the information to facilitate the diagnosis through a matchmaking for finding possible second cases

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Advisory Committee for Therapeutics (ACT) Toolkit

Published by Newcastle University

Clinical TrialExpert AdviceNetworkNeuromuscularResearch and Translational Resource

The TREAT-NMD Advisory Committee for Therapeutics (TACT) is a unique multi-disciplinary model centred upon an international group of academic and industry drug development experts, including patient representatives. TACT gives advice on the translational and development pathway of therapeutic programs in rare Neuromuscular Diseases. Due to its success in the neuromuscular field, a toolkit to replicate the TACT model has been produced. The Advisory Committee for Therapeutics (ACT) toolkit is intended to support rare disease communities outside of Neuromuscular to establish their own ACT, by providing them with step-by-step guidance and template documentation.

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European Patients’ Academy (EUPATI) toolbox

Published by IRDiRC

EUPATIOrphan DrugPatient InformationPatient TrainingResearch and Drug DevelopmentToolboxToolkit for Patient

The EUPATI Academy is the only in-depth training program for patient experts so far in EU that lasts 14months.
It includes an on online training program supported with two weeks of F2F training. The Academy is still operated by EPF, providing high quality training in drug development to patient from EMEA.

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EURORDIS’ Community Advisory Boards

Published by IRDiRC

Community Advisory BoardEurordisMedical ResearchOrphan Drug DevelopmentPatient AdvocatePatient InformationPatient OrganisationRare DiseaseResearch and Drug Development

Patient Community Advisory Boards are groups established and operated by patient advocates. They facilitate discussions in a safe harbour setting and under strict rules, an MOU and CA on the latest developments and challenges related to medical research and procedures in a disease area with the company or body conducting the research. The CAB can cover from preclinical to post marketing topics. The CAB depending on the areas of discussion can invite scientific expert to serve as advisors.

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