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New funding opportunities for orphan drug development in Horizon 2020

June 23 2016

Are you or your company currently in the process of developing a new therapy for rare diseases? Then the EU Horizon2020 funding programme is here to boost your clinical pipeline.

Orphan drug development in Horizon 2020 - rare disease infographic

The European Commission and the International Rare Diseases Research Consortium (IRDiRC) recognise the major medical need of people affected by a rare disease (with prevalence lower than 1 per 2000), who currently lack treatment options. The number of patients that are or will be affected by a rare disease in Europe surpasses 30 million and is growing. Due to recent advancements, diagnostic tests for 3500 rare diseases are now available. Yet, effective treatment options lack behind. The route to real impact on patients’ lives is to understand the mechanisms that govern their disease and develop smart, viable and feasible interventions.

Within the Horizon 2020 programme, the European Union (EU) has for example allocated €60 million towards high-quality projects with an international scope, leveraging both research and industry developments directed specifically at rare disease treatment (PM-08; see link/table). In addition, to incentivize the development of treatment options for rare diseases the EU has reduced the regulatory burden, shortening the route-to-market.

Since 2010 an important IRDiRC goal is to have 200 new therapies for orphan diseases reach the market by 2020 by supporting an interdisciplinary approach to intervention and diagnostics. With expertise and by leveraging regulatory agencies, patient organisations and the scientific community, the IRDiRC is a strong partner for organisations that strive to bring rare disease therapies to market. The current number of successful new therapies (195 per this writing) promises to push well beyond the IRDiRC target of 200, underscoring the effectiveness of this approach.

An important factor in reaching these goals is supporting SME biotech companies developing these new therapies for rare diseases, with for example several grant opportunities of the H2020 programme outlined in the table below.


Purpose &

Funding per project

Funding rate


PM 08 - New therapies for rare diseases

Funding for your clinical Phase I/II trial on a therapy for a rare disease where Orphan
Designation has been obtained.

€ 4 - 6 million


1st stage
2nd stage

PM - 03 diagnostic characterisation of rare diseases

Development of molecular diagnoses for a large number of undiagnosed rare diseases through application of omics and/or other high-throughput approaches for molecular characterisation of rare diseases.

€ 15 million



SME instrument: Cell technologies for medical

Phase I

Funding for a feasibility study to investigate the business potential of your idea (cell technologies for medical applications only).

€ 50k



SME instrument: Cell technologies for medical

Phase II

Funding for activities including clinical validation, upscaling of manufacturing, risk assessment, design or market studies and intellectual property exploration of cell technologies for medical application.

€ 1 - 5 million



Fast Track to


Projects undertaking innovation form the demonstration stage through to market uptake – relatively mature new technologies that will achieve market entry within 3 years

€ 2 - 3 million




Early industrial R&D, preclinical phases

€ 1 – 2 million




If the descriptions above relate to your research or activities, we invite you to contact us to further discuss whether there is a good match with this call-topic, and how we can help you. ttopstart and PSR Orphan Experts make an excellent team to support you in transforming your candidate therapy into real-world value.

Because of our excellent team of expert writers, our proven methodology (FFWD Method) and our extensive network in life sciences and biotech,  ttopstart is able to maximise your funding chances. With a success rate of more than 50% in last year’s Horizon2020 programme and the 250 Million in research funding that we acquired, ttopstart truly understands what is needed to write a winning proposal for the European Commission.

Furthermore, effective CRO services (from regulatory & clinical consultancy towards full clinical execution) geared toward rare disease therapy development requires a specific orphan drug mindset. PSR Orphan Experts is highly experienced in the full clinical development life-cycle for orphan drugs, which allows us to maximise your chances of success.

Please feel free to contact us to discuss possibilities and ideas.

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