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TOPIC : Development of sensitive and validated clinical endpoints in primary Sjögren’s Syndrome (pSS)

Topic identifier: IMI2-2017-12-03
Publication date: 19 July 2017

Types of action: IMI2-RIA Research and Innovation action
DeadlineModel:
Opening date:
two-stage
19 July 2017
Deadline:
2nd stage Deadline:
24 October 2017 17:00:00
16 May 2018 17:00:00

Time Zone : (Brussels time)
  Horizon 2020 H2020 website
Pillar: Societal Challenges
Work Programme Year: H2020-JTI-IMI-2017
Work Programme Part: IMI2 Work Plan 2017
Topic Description
Specific Challenge:

Currently, published data from placebo-controlled and adequately powered clinical trials in primary Sjögren’s Syndrome (pSS) are scarce. Although specific novel, validated treatment outcome measures have been developed recently, e.g. the European League against Rheumatism (EULAR) Sjögren’s syndrome disease activity index (ESSDAI) and EULAR Sjögren’s syndrome patient reported index (ESSPRI) [4,5], their recent use in clinical trials has yielded mixed results. Important features of pSS such as swallowing difficulties, dietary problems, mental health challenges, sexual dysfunction, dental problems (including tooth loss and decay) are not (adequately) captured. Overall, the utility of the currently available measures (including sensitivity to change in Patient Reported Outcomes (PROs) and in various ESSDAI domains) in assessing efficacy and disease-modifying potential of an investigational drug is still to be determined. Moreover, no objective validated measure or functional marker of disease activity for assessing therapeutic benefits of improvement is currently available. Sensitive and validated endpoints including objective measures/biomarkers of improvement are needed to increase the likelihood of success of drug development in pSS.

Scope:

The major scope of the action generated by this topic will be the identification, development and validation of pSS-related outcome measures including clinical, PRO, laboratory, bio-behavioural activity and imaging parameters (biomarkers), applying the following step-wise approach:

  • Data generation and review. Existing data including published epidemiology data, results from interventional and non-interventional studies, and from pSS registries will be reviewed and analysed.
  • Development of new outcome measures based on the review and analysis activities.
  • Application and validation by prospectively testing of these proposed new pSS outcome measures, as well as existing ones, in (at least one) dedicated, prospective clinical trial.
  • Analysis of the outcome of the validation trial and validation of the new endpoint(s). The performance of the new outcome measures or scoring systems will be compared to that of the existing ones, with the purpose to select the most promising outcome measures for future validation.

It is anticipated that the scoring system(s) will require a combination of objective and subjective outcome measures to improve upon existing scoring systems (e.g., selected, core set of ESSDAI domains combined with ESSPRI fatigue or other key PRO items).

If industry sponsored, large e.g. Phase 3 trial(s) are conducted for novel therapies in parallel with (but independently of) the validation trial during the project, the proposed new endpoint(s) may be included as exploratory endpoints in the Phase 3 trials to increase power and robustness of the validation. The analysis of these trials may, however, occur beyond the duration of this IMI action.

Health technology Assessment (HTA) and payer views and expectations will be integrated in determining the endpoints for regulatory approval and market access requirements. Input from patient groups will also be sought and considered in the analyses to capture relevant and currently underestimated or ignored disease aspects.

While the development of the new sensitive and validated clinical endpoints are primarily intended for use in future clinical trials of adult pSS, feasibility in pediatric SS will also be cautiously evaluated for which further validation would be required as part of the project sustainability plan.

Expected Impact:

Expected impact is to enhance the development of new systemic treatments in pSS and to generate evidence for a potential new alternative for consideration by the Health Authorities. .

It is expected to result in more efficient clinical trial designs that will minimize the number of subjects required to be able to detect statistically significant and clinically meaningful differences between treatments. The optimal duration of clinical studies required to demonstrate these differences will also be characterized.

Furthermore, new relevant outcomes will have potential to optimise pSS patients’ management, and large data sets about the natural history of the disease will provide information about the clinical utility of new and innovative diagnostic and treatment interventions in pSS. Engagement of important stakeholders including regulators, payers and patient advocacy groups will help capture all aspects of pSS.

Consequently, improved and innovative therapies are expected to emerge and be available to pSS patients whose health-related quality of life and productivity will eventually improve. Selection of the optimal treatment for the right patient in a clinically and molecularly heterogeneous disease will be made possible in pSS

Topic conditions and documents

Please read carefully all provisions below before the preparation of your application.

The IMI2 12th Call for proposals topic text as well as the Call Conditions are available here

1. List of countries and applicable rules for funding: described in article 10(2) of Regulation N° 1290/2013 of 11 December 2013 laying down the rules for participation and dissemination in Horizon 2020 and in article 1 of the Commission Delegated Regulation (EU) N° 622/2014 of 14 February 2014.

2. Eligibility and admissibility conditions: described in the IMI2 Manual for evaluation, submission and grant award See also the Commission Delegated Regulation (EU) N° 622/2014 of 14 February 2014.

Proposal page limits and layout: Please refer to Part B of the standard proposal template.

3. Evaluation

Submission and evaluation process, including evaluation criteria and procedure, scoring and threshold are described in the IMI2 Manual for submission, evaluation and grant award. See also the proposal templates for your specific action in section 5, below.

4. Indicative timetable for evaluation and grant agreement:

Notification of outcomes of stage 1 evaluations: Maximum 5 months from deadline for submitting proposals.
Notification of outcomes of stage 2 evaluations: Maximum 5 months from deadline for submitting full proposals.

Signature of grant agreements: maximum 3 months from the date of informing successful applicants.

5. Provisions, proposal templates and evaluation forms:

IMI2 Research and Innovation Action (IMI2-RIA) and (IMI2-IA):

Proposal templates are available after entering the submission tool

Standard evaluation form 

IMI2 Model Grant Agreement

Clinical trial template – the Clinical Trial template is compulsory at stage 2 only !

6. Additional provisions:
Open access must be granted to all scientific publications resulting from Horizon 2020 actions, and proposals must refer to measures envisaged. Where relevant, proposals should also provide information on how the participants will manage the research data generated and/or collected during the project, such as details on what types of data the project will generate, whether and how this data will be exploited or made accessible for verification and re-use, and how it will be curated and preserved.

This topic participates per default in the open access to research data pilot which aims to improve and maximise access to and re-use of research data generated by projects:

- The pilot applies to the data needed to validate the results presented in scientific publications. Additionally, projects can choose to make other data available for open access and need to describe their approach in a Data Management Plan (to be provided within six months after the project start).

- Note that the evaluation phase proposals will not be evaluated more favourably because they are part of the Pilot, and will not be penalised for opting out of the Pilot.

- Projects can at any stage opt-out of the pilot.

The legal requirements for projects participating in this pilot are in the article 29.3 of the Model Grant Agreement.
Further information on the Open Research Data Pilot is made available in the H2020 Online Manual: http://ec.europa.eu/research/participants/docs/h2020-funding-guide/cross-cutting-issues/open-access-dissemination_en.htm 

7. Additional documents:

Summary of the most relevant provisions for participating in IMI2 actions

IMI2 Annual Work Plan 2017 

IMI2 Regulators Guidance tool for researchers 

IMI JU derogation to H2020 Rules for Participation  

Horizon 2020 Rules for Participation 

Horizon 2020 Regulation of Establishment

Horizon 2020 Specific Programme 

 

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