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Development of evidence based practical guidance for sponsors on the use of real-world data / real-world evidence

European Comission

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Summary
16 January 2024
16 April 2024
10 October 2024
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For profit
Not for profit (incl. NGOs)
Public sector
R&D and Higher Education
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Research, Development and Innovation Medical and Health Sciences
Overview

ExpectedOutcome :

  • Industry, sponsors, and other stakeholders have access to structured, evidence-based and practical guidance and recommendations on the use of real-world data / real world evidence (RWD/RWE)1 that could be followed to support the development, and regulatory, health technologies assessment (HTA), and payer decision-making of innovative medicines and health technologies with a focus on medicinal products, medical devices, and therapeutic products that combine a medicinal product with a medical device (drug-device combinations).
  • Regulators, HTA bodies and payers will receive more structured and consistent RWD/RWE submissions to inform their decision making.

1 Real World Data (RWD) are defined as “routinely collected data relating to a patient's health status or the delivery of health care from a variety of sources other than traditional clinical trials.” Real-world evidence (RWE) is defined as the information derived from analysis of RWD. https://doi.org/10.1002/cpt.1426

Scope :

The use of real-world evidence to support decision making on the safety of medicinal products is already well established. More recently, RWE has also been used to complement evidence and support marketing authorisation, conformity assessments and HTA submissions. While high-level guidance on the use of RWD/RWE exists, the practical implementation is left up to individual sponsors. Currently, RWD/RWE submissions are usually custom-made to a specific use-case and require significant expertise and effort from the sponsor to prepare, and from the healthcare decision-maker to assess. Much knowledge exists within individual sponsors on these use-cases, but, to date, this has not been leveraged to develop practical guidance which could act as a baseline for future submissions.

To leverage the learning from individual use cases and facilitate the efficient use of RWD/RWE for regulatory, HTA, and payer submissions and to inform healthcare decision-making, structured, evidence-based, and practical guidance is needed.

To address this challenge, the action funded under this topic should:

  • Map relevant RWD/RWE initiatives across Europe and their (expected) outcomes. Where relevant, build on, align, and complement these initiatives, including the European Medicines Agency’s vision to establish the value of RWE across the spectrum of regulatory use cases by 2025 1.
  • Identify the main challenges faced by industry, sponsors, non-commercial sponsors, health professionals, prescribers, and other stakeholders in the routine use of RWD/RWE for regulatory and HTA decision-making. This is to be done by also taking into account the differences in the regulatory frameworks of medicinal products and medical devices and how stakeholders’ experiences, needs, and situations are reflected in these.
  • In collaboration with the relevant stakeholders, identify, review, and evaluate existing methodologies, guidelines, and practices for the use of RWD/RWE in healthcare decision-making.
  • Focus on an in-depth study of a broad range of use cases where RWD/RWE has been previously assessed for decision-making for medicinal products, medical devices, and combinations. This should include an analysis of methods, designs, and defining variables that enable the grouping and thereafter the utilisation of RWD/RWE sources. Particular attention should be paid to the features that enable efficient assessments.
  • Using the results of the study as a foundation, develop a draft of the practical guidance document and recommendations on the use of RWD/RWE to support submissions and decision-making processes, taking into consideration the specific needs of medicinal products and medical devices. Considerations on how RWD/RWE can be used within an ethical framework and respects EU values should be included. In addition, ensure that the guidance respects the EU data quality framework and the relevant RWD specialisation (which is currently under development).
  • Test the draft guidance in several pilots to ensure validity and broad acceptability.The precise scope of these pilots should be selected by the full consortium during preparation of the full proposal and should address multiple contexts and areas that are not already being addressed, including but not limited to: chronic serious diseases, oncology, and auto-immune diseases. They should also cover clinical development and the regulatory, HTA, and payer assessment of medicinal products and medical devices including combinations.
  • Based on the learnings from the pilots, finalise the practical guidance document and recommendations on the use of RWD/RWE to support clinical development, regulatory, HTA and payer submissions and inform decision-making processes.
  • Broadly disseminate the guidance and recommendations to the stakeholder community. Create training plans to enable dissemination.

Applicants should develop a strategy and plan for generating appropriate evidence as well as for engaging and formally consulting with regulators, HTA agencies and payers in a timely manner, in particular on the draft guidance (e.g. through national competent authorities, the EMA Innovation Task Force, qualification advice).

In addition, while the project will focus on supporting the development of a recommendation for a structured, practical and evidence based guidance, the funded project is also expected to explore synergies with complementary initiatives to advance RWD/RWD in Europe such as the GetReal Institute, REDDIE, More-EUROPA, Oncovalue, Real4Reg, RWE4Decisions, TEHDAS, QUANTUM, CORE-MD, REALM2 and projects under the ongoing call for proposals HORIZON-HLTH-2024-IND-06-08. It should also be aligned with the ambitions and guidelines set out for the European Health Data Space (EHDS)3.

1 Arlett P. et al. Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value. Clinical Pharmacology & Therapeutics 2021 https://doi.org/10.1002/cpt.2479

2 www.getreal-institute.org, www.reddie-diabetes.eu, cordis.europa.eu/project/id/101095479, oncovalue.org, www.real4reg.eu, realm-ai.eu

3 health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space_en

Expected Impact :

The action under this topic is expected to achieve the following impacts:

  • Improved access to innovations that meet the increasingly diverse needs of patients and those of the healthcare systems.
  • Better informed decision-making at different levels of the healthcare system (authorities, organisations) using RWD/RWE that will in turn contribute to a better allocation of resources towards cost-effective innovations as well as representation of different patient populations and needs.
  • Faster entry to the market of cost-effective medicinal products and devices (including combinations) developed by industry or public not-for-profit developers, which could translate to a positive effect on their R&I investments.
Eligibility

General conditions

  1. Admissibility conditions: described in Annex A and Annex E of the Horizon Europe Work Programme General Annexes

Proposal page limits and layout: described in Part B of the Application Form available in the Submission System

  • at stage 1 of a two-stage Call, the limit for RIA short proposals is 20 pages;

  • at stage 2 of a two-stage Call, the limit for RIA full proposals is 50 pages.

  1. Eligible countries: described in Annex B of the Work Programme General Annexes

A number of non-EU/non-Associated Countries that are not automatically eligible for funding have made specific provisions for making funding available for their participants in Horizon Europe projects. See the information in the Horizon Europe Programme Guide .

  1. Other eligibility conditions: described in Annex B of the Work Programme General Annexes and in the ''Conditions of the Calls for proposals and Calls management rules'' section of the IHI JU Work Programme (WP)

  2. Financial and operational capacity and exclusion: described in Annex C of the Work Programme General Annexes

  3. Evaluation and award:

  • Award criteria, scoring and thresholds are described in Annex D of the Work Programme General Annexes and in the ''Conditions of the Calls for proposals and Calls management rules'' section of the IHI JU Work Programme (WP)

  • Submission and evaluation processes are described in Annex F of the Work Programme General Annexes and the Online Manual

  • Indicative timeline for evaluation and grant agreement: described in Annex F of the Work Programme General Annexes

  1. Legal and financial set-up of the grants: described in Annex G of the Work Programme General Annexes

Specific conditions

  1. Specific conditions: described in the described in the ''Conditions of the Calls for proposals and Calls management rules" section of the IHI JU Work Programme (WP)
  • specific conditions on Availability, Accessibility and Affordability (3A) do not apply to this topic

  • JU's right to object to transfer/exclusive licensing

Documents

Where relevant, templates of the reference documents and associated guidance can be found on the IHI JU website .

Regarding the application forms for submitting proposals, the relevant templates and annexes are available to download in the submission system.

The IHI JU 6th Call for proposals full topics text is available here

Evaluation form (single and two-stage Calls)

Proposal Templates Part A and Part B (Research and Innovation Actions – stage one of two-stage procedure)

  • Proposal template - Part A of the proposal is generated by the IT system in the submission environment (for more information see the HE Part A template here). In Part A of the proposal applicants insert general information on their proposal (e.g. proposal acronym), details on the participants and the overall proposal budget. Please note that only Part A of this template is applicable for this Call. For Part B, see point below.

  • IHI JU Proposal template (RIA/SP) - Part B

(applicable to the first-stage of two-stage Calls)

Proposal Annexes

  • Annex: Type of Participants

The “type of participants” is an IHI specific annex.

The excel template for:

§ short proposals (first stage of two-stage calls) can be found here and

§ full proposals (single-stage calls, and second stage of two-stage calls) can be found here

The instructions on how to fill in this template can be found here.

This is a compulsory annex, and it must be uploaded as a separate document in the submission system.

This annex is applicable to single-stage and in both stages of two-stage Calls.

  • Annex: Declaration of in-kind contribution commitment

The “Declaration of in-kind contribution commitment” is an IHI specific annex and it is applicable to the single stage and second stage of two-stage Calls.

The word document template can be found here.

This is a is a compulsory annex and it must be uploaded as a separate document in the submission system.

  • Annex: In-kind contributions to additional activities (IKAA)

The ‘’In-kind contributions to additional activities (IKAA)’’ is an IHI specific annex. The excel template can be found here and the instructions on how to fill in this template can be found here.

This is an optional annex and

it is applicable to the single stage and second stage of two-stage Calls.

  • Annex: Essential information for clinical studies

The information on clinical studies is a Horizon Europe annex.

This is a is a compulsory annex and it must be uploaded as a separate document in the submission system.

If your proposal does not include clinical studies, please upload a statement declaring your proposal does not include clinical studies.

The information on clinical studies annex can be found here:

The annex is applicable to the single stage and second stage of two-stage Calls .

  • Annex: Ethics

This is a HE annex. Ethics self-assessment should be included in proposal part A. However, in Calls where several serious ethics issues are expected, the characters limit in this section of proposal part A may not be sufficient for participants to give all necessary information. In those cases, participants may include additional information in an annex to proposal part B.

This is an optional annex and it is applicable to the single stage and second stage of two-stage Calls.

  • Annex to the budget for the Full Proposal

This is a compulsory Annex, which complements the budget figures already included in the proposal budget in PART A. Its purpose is to correctly guide the consortium in providing IHI-specific budget items (e.g. IKOP, IKAA, FC PAID, FC RECEIVED, etc.) and to comply with IHI additional eligibility criteria (e.g. 45% industry contribution).

The annex is applicable to the single stage and second stage of two-stage Calls

Model Grant Agreement (MGA)

Additional documents:

Additional documents:

Horizon Europe Reference Documents

HE Main Work Programme 2023–2024 – 1. General Introduction

HE Main Work Programme 2023–2024 – 4. Health

HE Main Work Programme 2023–2024 – 12. Missions

HE Main Work Programme 2023–2024 – 13. General Annexes

HE Programme Guide

HE Framework Programme and Rules for Participation Regulation 2021/695

HE Specific Programme Decision 2021/764

EU Financial Regulation

Rules for Legal Entity Validation, LEAR Appointment and Financial Capacity Assessment

EU Grants AGA — Annotated Model Grant Agreement

Funding & Tenders Portal Online Manual

Funding & Tenders Portal Terms and Conditions

Funding & Tenders Portal Privacy Statement

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21 June 2024