Development of evidence based practical guidance for sponsors on the use of real-world data / real-world evidence
Programme Category
Programme Name
Programme Description
IHI JU is based on the idea that interdisciplinary and cross-sector collaboration will enable perspective and breakthrough innovations in healthcare, including the pharmaceutical industry but also new fields such as biopharmaceuticals, medical technologies and biotechnologies.
Identifier Code
Call
Summary
This topic focuses on the development of evidence based practical guidance for sponsors on the use of real-world data / real-world evidence.
The use of real-world evidence to support decision making on the safety of medicinal products is already well established. 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.
Detailed Call Description
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.
- 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.
Call Total Budget
Financing percentage by EU or other bodies / Level of Subsidy or Loan
Maximum financial contribution per topic: €13.300.000
Thematic Categories
- Health
- Research, Technological Development and Innovation
Eligibility for Participation
- Central Government
- Large Enterprises
- Legal Entities
- Other Beneficiaries
- Private Bodies
- Researchers/Research Centers/Institutions
- Small and Medium Enterprises (SMEs)
- State-owned Enterprises
Eligibility For Participation Notes
ENTITIES ELIGIBLE FOR FUNDING
In relation to the two-stage calls for proposals covered by this Work Programme, the following provisions shall apply:
- Legal entities identified in the topic text of the call for proposals shall not be eligible for funding from IHI JU. Nevertheless:
- These entities will be entitled to provide contributions as IHI JU members other than Union or contributing partners or as constituent or affiliated entities of either.
- Legal entities participating in indirect actions selected under this type of calls for proposals shall not be eligible for funding where:
(a) they are for-profit legal entities with an annual turnover of EUR 500 million or more;
(b) they are under the direct or indirect control of a legal entity described in point (a), or under the same direct or indirect control as a legal entity described in point (a);
(c) they are directly or indirectly controlling a legal entity referred to in point (a).
The following additional condition applies:
- The applicants which are IHI JU members other than the Union, or their constituent entities and affiliated entities, and contributing partners and that are pre-identified in the topics – under the section ‘Industry consortium’ – of a call for proposals shall not apply at the first stage of the call. The applicant consortium selected at the first stage shall, in preparation for the proposal submission at the second stage, merge with the pre-identified industry consortium.
In addition, in line with Articles 11 and 119(1) and (3) of the Council Regulation (EU) 2021/2085, legal entities providing in-kind contributions as constituent entities or affiliated entities of IHI JU private members or as contributing partners that are:
- Not eligible for funding in two-stage calls for proposals; or
- Not established in a country generally eligible for funding in accordance with Part B of the General Annexes to the Horizon Europe Work Programme 2023 – 2024, may exceptionally sign the grant agreement.
This is subject to the following conditions:
- Their participation is considered essential for implementing the action by the granting authority; and
- They participate without requesting any funding.
The essentiality of non-EU legal entities for implementing the action shall be ascertained by the granting authority.
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).
The active involvement of many stakeholders working collaboratively in partnership is needed to ensure such guidance has broad applicability and adds value to the broader initiatives already underway. The diverse nature of these stakeholders, which includes patients, real world data custodians, academics, and SMEs with expertise in RWD, industry, regulators, HTA agencies, and payers, means that a public-private partnership is the ideal framework for such a collaboration.
Applicant consortium
The first stage applicant consortium is expected, in the short proposal, to address the scope and deliver on the expected outcomes of the topic, considering the expected contribution from the pre-identified industry consortium.
This may require mobilising the following expertise and/or resources:
- expertise in the technical, legal, and ethical requirements to access and use patient data in Europe;
- knowledge of medicinal product and/or medical device development regulations
- expertise in interacting with regulatory authorities, national competent authorities, HTA bodies, notified bodies and payers;
- experience with consumer-directed communications and/or patient advocacy (social media reach and expertise in health sector communications);
- expertise in managing multi-stakeholder cross-sectoral projects;
- citizens and/or patient representatives;
- real-world data sources (healthcare providers, clinical sites, contract research organisations (CROs), vendors, national/regional databases).
The applicant consortium is expected to enable effective collaboration with regulatory authorities, national competent authorities, HTA bodies, notified bodies and payers, and may consider, for instance, engaging them as consortium partners, or in an advisory capacity.
Call Opening Date
Call Closing Date
EU Contact Point
All the questions pertaining to the IHI JU Calls are to be addressed to infodesk@ihi.europa.eu.