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Upcoming call: New therapies for rare diseases

May 06 2016

Within the upcoming round of the Societal Challenges (SC1) call in the 2016-2017 Horizon2020 work programme, ttopstart has selected the 4 major topics with a pre-proposal stage in October of this year to help you prepare for this first stage deadline. Today we discuss the second: ‘PM-08-New therapies for rare diseases’.

Although current biomedical research has made great progress in gaining knowledge on the almost 6000 to 8000 rare diseases known to science, effective therapies for these often life-threatening or debilitating diseases are still lacking. This is partially due to the generally small and dispersed patient populations and the type of suggested therapy which is usually highly specialised, novel and expensive. In addition, the inclusion of early advice from regulatory authorities during development is highly recommended. Lastly, the limited market for these rare disease therapies yield a low commercial return and/or a limited access.


The topic supports clinical trials with Orphan Drug Designation (ODD) substances. Project proposals should aim to develop therapies for rare diseases by implementing clinical trials focusing on a range of interventions with an orphan designation, from small molecule to gene or cell therapy, and may include novel interventions and/or repurposing of existing interventions. There is room for a minimal element of late stage preclinical research or risk assessment complimentary to the trial. The intervention must have been granted the EU orphan designation, at the latest on the date of the full proposal call closure. Furthermore, the proposed clinical trial design takes into account recommendations from protocol assistance given by the European Medicines Agency and a clear patient recruitment strategy is presented. Concise feasibility should be proven by available previous publications on clinical and preclinical supporting data. Patient organisations should be involved.


The Commission expects proposals that advance the development of new therapeutic avenues with concrete clinical improvements for patients with rare diseases. A prerequisite for rapid advance in orphan drug development are well-prepared multi-centre clinical trials with an appropriate number of subjects. The proposal should include an early estimate of the economic potential and public health power of the new therapeutic solution. Finally, proposals should adhere to the International Rare Diseases Research Consortium (IRDiRC) objectives.

A typical consortium for this call includes clinicians, preferably representatives from multiple centres, clinical and disease related researchers and patient organisation advocates. In addition, the consortium would be exceptionally strong with the inclusion of a European SME to support decisive steps in the setup of the clinical trial towards optimal commercialisation of an orphan drug in the final stages of the clinical process. An early engagement of a specialised contract research organisation (CRO), as partner or subcontracted, will facilitate setting up a patient recruitment strategy and engaging in early EMA protocol assistance or scientific advice. The proposal should include a fully developed clinical trial annex, and with regard to the participation of the SME, the drug should at minimum have been tested in patients before and preferably be ready for a phase IIb trial.

From our elaborate expertise in these health related calls and previous successful proposals we have made a solid interpretation of these new topics which allows us to provide you with optimal support in the preparation of your proposal. If the description above relates 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.

Stage1: October 4 2016
Stage2: April 11 2017

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