Call for Supporting Early-Phase Multinational Rare Disease Studies

The ERDERA Clinical Trial Call funds multinational Phase I, Phase I/II, and Phase II interventional clinical trials that accelerate the development of treatments for rare diseases. The programme supports Good Clinical Practice (GCP)-compliant, sponsor-led studies involving international research consortia, with a strong focus on unmet medical needs, paediatric rare diseases, and collaborative clinical research across ERDERA member countries.

ERDERA Clinical Trial Call for Rare Disease Research

What is the ERDERA Clinical Trial Call?

The European Rare Diseases Research Alliance (ERDERA) Clinical Trial Call is an international funding programme that supports multinational clinical trials aimed at developing new treatments for rare diseases.

The programme funds Phase I, Phase I/II, and Phase II interventional clinical trials involving investigational medicinal products. It is designed to generate high-quality clinical evidence that can support future regulatory approval, improve patient outcomes, and accelerate the development of innovative therapies for rare diseases.

Unlike traditional joint funding programmes, the ERDERA Clinical Trial Call is centrally managed by Fondazione Telethon, allowing applicants to submit a single application under one harmonized funding framework.

Funding Overview

The Clinical Trial Call provides centralized funding for eligible multinational clinical trials.

Key funding features include:

Funding managed centrally by Fondazione Telethon on behalf of the ERDERA consortium.
Single application process for funded partners.
No parallel national applications required for ERDERA-funded participants.
Canadian participants receive funding separately through the Canadian Institutes of Health Research (CIHR).

Funding supports multinational clinical trials conducted according to internationally recognized clinical research standards.

Programme Objectives

The ERDERA Clinical Trial Call aims to:

Accelerate the development of treatments for rare diseases.
Generate robust clinical evidence.
Produce data that support future regulatory submissions.
Promote multinational clinical research collaboration.
Address unmet medical needs in rare diseases.
Improve access to innovative therapies.
Strengthen patient involvement in clinical research.
Advance translational and clinical medicine.

Priority Areas

The programme prioritizes clinical trials addressing:

Paediatric Rare Diseases

Projects focusing on rare diseases affecting children, including therapies designed specifically for paediatric patients.

Rapidly Progressive Rare Diseases

Clinical trials targeting diseases that rapidly worsen and require urgent therapeutic intervention.

Rare Diseases with No Approved Treatments

Studies investigating therapies for conditions that currently have no approved treatment options.

Rare Diseases with Significant Unmet Medical Need

Research addressing diseases where existing treatments remain insufficient despite current therapeutic options.

Eligible Clinical Trial Types

The programme supports:

Phase I clinical trials.
Phase I/II clinical trials.
Phase II clinical trials.

All studies must be:

Interventional.
Sponsor-led.
Good Clinical Practice (GCP) compliant.
Based on investigational medicinal products.

Eligible Interventions

Supported investigational medicinal products include:

Small molecules.
Repurposed drugs.
Advanced Therapy Medicinal Products (ATMPs) with GMP-validated manufacturing for Phase I/II studies.
Biologics.
New biological entities.
Repurposed biologic therapies.

Consortium Requirements

Only multinational consortia are eligible.

Each consortium must include:

At least three independent eligible partner institutions.
Partners from three different eligible ERDERA member countries.
One designated Clinical Trial Sponsor.
One designated Coordinating Investigator.
At least one funded patient partner represented by a Patient Advocacy Organisation (PAO) or another organized patient group.
Access to a qualified multinational Clinical Trial Management Organisation (CTMO).

These requirements ensure effective international collaboration and high-quality trial management.

Who is Eligible?

Eligible applicants include:

Universities.
Higher education institutions.
Research institutes.
Hospitals.
Clinical research organisations (CROs).
Healthcare institutions.
Patient Advocacy Organisations (PAOs).
Small and Medium Enterprises (SMEs).
Non-profit research organisations.
Research foundations.

Who is Not Eligible?

The following organizations are generally not eligible to receive funding:

Private for-profit companies other than eligible SMEs.
Consortia that do not meet multinational partnership requirements.
Clinical trials outside the eligible trial phases.
Projects that fail to comply with Good Clinical Practice (GCP) standards.

Why This Funding Matters

Rare diseases affect millions of people worldwide, but many conditions still lack effective treatments due to limited patient populations and fragmented research efforts.

The ERDERA Clinical Trial Call helps:

Accelerate rare disease drug development.
Strengthen international clinical research collaboration.
Improve clinical evidence generation.
Increase patient involvement in research.
Support regulatory readiness.
Advance innovative therapies.
Improve healthcare outcomes for rare disease patients.

How the Programme Works

The funding programme follows a centralized application and management process.

Step 1: Build an International Consortium

Applicants establish a consortium involving eligible institutions from at least three ERDERA member countries.

Step 2: Appoint Key Partners

The consortium appoints:

Clinical Trial Sponsor.
Coordinating Investigator.
Patient Advocacy Organisation.
Clinical Trial Management Organisation.

Step 3: Develop the Clinical Trial

Applicants prepare a GCP-compliant protocol for an eligible Phase I, Phase I/II, or Phase II interventional clinical trial.

Step 4: Submit a Single Application

Applications are submitted directly through the ERDERA funding process without separate national submissions for funded partners.

Step 5: Conduct the Trial

Approved projects implement multinational clinical trials according to international regulatory and ethical standards.

Tips for a Strong Application

Applicants should:

Address a clearly defined unmet medical need.
Build a strong multinational consortium.
Demonstrate scientific excellence.
Include experienced clinical investigators.
Involve patient organizations from the beginning.
Present a realistic recruitment strategy.
Ensure regulatory and GCP compliance.
Develop a robust clinical development plan.

Common Mistakes to Avoid

Avoid:

Forming consortia with fewer than three eligible countries.
Excluding a patient partner.
Failing to appoint a Clinical Trial Sponsor.
Submitting trials outside the eligible phases.
Ignoring GCP requirements.
Providing weak regulatory planning.
Developing unrealistic recruitment timelines.
Submitting incomplete applications.

Frequently Asked Questions (FAQs)

1. What is the ERDERA Clinical Trial Call?

It is an international funding programme supporting multinational Phase I, Phase I/II, and Phase II interventional clinical trials for rare disease treatments.

2. Which clinical trial phases are eligible?

Eligible studies include:

Phase I.
Phase I/II.
Phase II interventional clinical trials.

3. What diseases are prioritized?

Priority is given to:

Paediatric rare diseases.
Rapidly progressive rare diseases.
Rare diseases without approved therapies.
Rare diseases with substantial unmet medical needs.

4. Who can apply?

Eligible applicants include universities, hospitals, research institutes, CROs, healthcare institutions, Patient Advocacy Organisations, SMEs, research foundations, and non-profit research organizations.

5. Are multinational partnerships required?

Yes. Every consortium must include at least three independent eligible partners from three different ERDERA member countries, along with a Clinical Trial Sponsor, Coordinating Investigator, patient partner, and Clinical Trial Management Organisation.

6. Can large private companies receive funding?

No. Private for-profit companies other than eligible SMEs are not eligible for funding under this call.

7. Why is this programme important?

The ERDERA Clinical Trial Call promotes international collaboration, accelerates rare disease therapy development, generates regulatory-quality clinical evidence, strengthens patient participation, and helps bring innovative treatments closer to people living with rare diseases.

Conclusion

The ERDERA Clinical Trial Call provides a unique opportunity for international research teams to conduct multinational Phase I, Phase I/II, and Phase II clinical trials targeting rare diseases. Through centralized funding, harmonized application procedures, strong patient involvement, and collaboration across ERDERA member countries, the programme supports the generation of high-quality clinical evidence and the development of innovative therapies for conditions with significant unmet medical needs. Eligible organizations are encouraged to form strong multinational consortia and submit scientifically rigorous proposals that can improve treatment options for people living with rare diseases.