Paediatric drug formulations for poverty-related diseases – 2019

Description

Background
Poverty-related diseases (PRDs) remain the leading causes of morbidity and mortality in sub-Saharan Africa, especially during childhood. Despite progress in other age groups, effective treatment and prevention of PRDs in paediatric populations is often lacking and/or lagging. The frequent exclusion of children and adolescents from clinical trials and the paucity of available products that target this group are factors that contribute to this population having one of the lowest health indicators. Additional challenges relate to the limited financial incentives associated with the adaption of off-patent medicines to the specific needs of paediatric populations. Therefore, concerted efforts are needed to increase access to potentially life-saving, cost-effective interventions to prevent and treat PRDs in children and to enhance the use of existing interventions in this population.

Scope
Proposals should focus on the adaptation of existing medicinal products (drugs), including off-patent products, to the specific needs of children (0-17 years of age). Proposals may address any of the PRDs within the remit of the EDCTP2 programme. Proposals should include one (or more) clinical trials conducted in sub-Saharan Africa to assess the safety, efficacy and pharmacokinetics of the drug(s), and/or the development of age-appropriate formulations. Projects must assure that the clinical trials are conducted appropriately, in line with guidelines on pharmaceutical development of medicines for paediatric use*, respecting current legislation and considering the ethical aspects and particular needs of the study subjects and their families. Applicants should seek advice on the clinical trial design from the appropriate regulatory agency(ies) before developing the application. The EDCTP Association considers that proposals for actions of between 36 and 60 months duration would allow this specific challenge to be addressed appropriately.

The clinical need for paediatric dosing should be clearly explained in the proposal. The proposal must include full details of the strategy for the clinical paediatric development, including product development milestones and go/no-go criteria for the proposed clinical trial(s) as well as specific plans for the subsequent regulatory approval process, ideally a paediatric investigation plan, which should aim at obtaining a relevant market authorisation such as the Paediatric Use Marketing Authorisation (PUMA) or equivalent.

Expected impact
The actions supported under this call should have the potential to achieve maximum impact in the field and to make a significant contribution to the objectives of the EDCTP2 programme, and in particular:

• contribute towards the development or adaptation of medicinal products for use in children;
• lead to the advancement of new drugs and/or drug combinations, with the aim of registration of new drug(s) and/or drug combinations for treatment and prevention of PRDs in children in sub-Saharan Africa and globally;
• contribute to the reduction of mortality and morbidity in sub-Saharan Africa, particularly in children and thus contribute to achieving SDG 3 ‘Ensure healthy lives and promote well-being for all at all ages’.

Eligibility
A proposal/application will only be considered eligible if:

1. its content corresponds, wholly or in part, to the topic/contest description for which it is submitted;
2. it complies with the eligibility conditions for participation set out below, depending on the type of action:
   • At least three legal entities. Two of the legal entities shall be established in two different Participating States (European Partner States)** and one of the legal entities must be established in a sub-Saharan African*** country. All three legal entities must be independent of each other;
   • ‘Sole participants’ formed by several legal entities (e.g. European Research Infrastructure Consortia, European Groupings of Territorial Cooperation, central purchasing bodies) are eligible if the above-mentioned minimum conditions are satisfied by the legal entities forming together the sole participant.

Notes

*Guideline on pharmaceutical development of medicines for paediatric use EMA, 1 August 2013

**The Participating States (European Partner States) are Austria, Denmark, Finland, France, Germany, Ireland, Italy, Luxembourg, the Netherlands, Norway, Portugal, Spain, Sweden and the United Kingdom.

***The following sub-Saharan African countries: Angola, Benin, Botswana, Burkina Faso, Burundi, Cameroon, Cape Verde, Central African Republic, Chad, Comoros, Congo (Democratic People’s Republic), Congo (Republic), Côte d’Ivoire, Djibouti, Eritrea, Ethiopia, Equatorial Guinea, Gabon, Gambia, Ghana, Guinea, Guinea-Bissau, Kenya, Lesotho, Liberia, Madagascar, Malawi, Mali, Mauritania, Mauritius, Mozambique, Namibia, Niger, Nigeria, Rwanda, São Tomé and Príncipe, Senegal, Seychelles, Sierra Leone, Somalia, South Africa, South Sudan, Sudan, Swaziland, Tanzania, Togo, Uganda, Zambia, and Zimbabwe.

Procedure and application process

Submission and evaluation procedure
Two-stage application procedure. For the first stage, a letter of intent must be submitted by the first deadline. Successful applicants in the first stage will be invited to submit a full proposal for the second stage.

• Proposals for Stage 1 must be submitted by 10 October 2019 at 17:00:00 CET via EDCTPgrants. Evaluation results for Stage 1 are expected to be made available by 21 December 2019.
• Proposals for Stage 2 must be submitted by 16 March 2020 at 17:00:00 CET via EDCTPgrants. Evaluation results for Stage 2 are expected to be made available by 24 July 2020.

Evaluation, scoring, and thresholds
Following an admissibility and eligibility check, full proposals are evaluated by external, independent experts. Proposals are evaluated according to the criteria Excellence, Impact and Implementation (see Article 15 of the Horizon 2020 Rules for Participation Regulation No 1290/2013). Each criterion will be scored out of 5. The threshold for individual criteria will be 3. The overall threshold, applying to the sum of the three individual scores, will be 10.

For the evaluation of first-stage proposals under a two-stage submission procedure, only the criteria ‘excellence’ and ‘impact’ will be evaluated. Within these criteria, only the aspects in bold will be considered. The threshold for both individual criteria will be 4. For each indicative budget-split in the call conditions, the overall threshold, applying to the sum of the two individual scores, will be set at the level such that the total requested budget of proposals admitted to stage 2 is as close as possible to three times the available budget, and in any case, not less than two and a half times the available budget.

The actual level will, therefore, depend on the volume of proposals and funding request per proposal received. The threshold is expected to normally be set at 8 or 8.5.

For all proposals involving human participants, and/or human tissues, cells or personal data, the evaluation process will include an assessment of ethical issues.

The following aspects are considered under the evaluation criteria:

1. Excellence

• Fit with the scope and objectives of the EDCTP2 programme, the EDCTP Association strategic research agenda and the call topic description;
• Importance, relevance/pertinence, and clarity of the objectives;
• The soundness of the concept and credibility of the proposed approach/methodology.
• Importance of the question being addressed and the rationale/need for the proposed clinical trial(s) or research study now;
• Excellence and appropriateness of the clinical trial design, including the proposed location(s) of the trial;
• The extent that the proposed trial will advance the field. In particular, how it differs from or complements any relevant planned, ongoing or recently completed trials internationally;
• Appropriate consideration of interdisciplinary approaches, and where relevant, use of stakeholder knowledge and gender dimension in research and innovation content.

2. Impact

• Call specific aspects as listed under ‘expected impact’ in each individual call;
• The extent to which the outputs of the proposed work would contribute, at the European, African and/or International level, to each of the expected impacts listed in the work plan under the relevant topic;
• The likelihood to result in major advances in the field with the potential benefit of the research to the affected populations;
• Advancing the clinical development of new and improved products;
• Generalisability of the trial/study results beyond the immediate research setting in a way that will maximise the impact of the results;
• Contribution to improved disease management and prevention through changes in policy and practice, with the ultimate goal of improving public health;
• Contribution to strengthening the capacity in sub-Saharan Africa to conduct clinical trials;
• Effectiveness and quality of the proposed measures to exploit and disseminate the project results (including management of IPR) to communicate the project activities to different target audiences, and to manage research data.

3. Quality and efficiency of the implementation

• Quality and effectiveness of the work plan, including the extent to which the resources assigned to work packages are in line with their objectives and deliverables;
• Appropriateness of the management structures and procedures, including risk and innovation management, and how responsibilities for research data quality and sharing, and security will be met;
• Complementarity of the participants within the consortium, and the extent to which the consortium as whole brings together the necessary expertise;
• Appropriateness of the allocation of tasks and resources, ensuring that all participants have a valid role and adequate resources in the project to fulfil that role;
• Feasibility and appropriateness of the methods and project management to achieve the objectives within the timeframe of the grant;
• Compliance with national and international standards of research, Good Clinical Practice, ethics- and safety-related issues;
• Participants have the operational capacity, to carry out the proposed work, based on the competence and experience of the individual participant(s);
• Competence of the participants and their investigators in conducting trials according to international standards of Good Clinical Practice (ICH-GCP);
• Involvement of sub-Saharan African researchers in the scientific leadership of the clinical trial;
• Arrangements and plans to take forward clinical development of the products under evaluation (where applicable).

For this call for proposals, the Coordinator has a ‘right to reply’ to the expert assessments (rebuttal procedure).

Financial provisions

• The call budget is EUR 18 million;
• The funding level is 100% of eligible costs.

Grant agreement
Participants in actions resulting from this Call for Proposals will be required to conclude a consortium agreement prior to the conclusion of the General EDCTP2 grant agreement (multi-beneficiary).

Application process

• The application must be submitted online via EDCTPgrants;
• Only registered users of EDCTPgrants system can apply for grants and therefore you are advised to register on the system as soon as possible.

More information
• EDCTP2 Frequently Asked Questions about calls for proposals
• For specific questions related to this call for proposals, please contact: Dr Debora Bade at bade@edctp.org.
• For questions and issues about EDCTPgrants and the online application submission please contact EDCTP via edctpgrants@edctp.org
• For guidance on applying to EDCTP, please refer to the Templates, guides and policies section of the EDCTP website
• The EDCTP2 programme runs until the end of December 2024. Applicants should take into consideration the programme end date when planning their proposals.

Projects awarded under this call

ACOZI-KIDS
Towards a pediatric indication for children between 1 and 14 years of age for a safe, effective, all-oral single-dose treatment formulation of acoziborole for first and second stage gambiense human African trypanosomiasis (g-HAT)

Drugs for Neglected Diseases initiative, Geneva, Switzerland, with partners from the Democratic Republic of the Congo, France, Guinea, and Portugal 
Project coordinator: Dr Antoine Tarral
Starting date: 01 October 2020
Duration: 60 months
EDCTP grant amount: EUR 2,999,107
Grant agreement: RIA2019PD-2890

DPP
Developing paediatric primaquine

Chancellor, Masters and Scholars of the University of Oxford, Oxford, United Kingdom, with partners from Burkina Faso, Ethiopia, and France
Project coordinator: Dr W.R.J. Taylor
Starting date: 01 September 2020
Duration: 56 months
EDCTP grant amount: EUR 3,489,896
Grant agreement: RIA2019PD-2893
Project website

MiniMox
Treatment for all: Developing a paediatric formulation moxidectin for neglected infectious diseases

Luxembourg Institute of Health, Luxembourg, Luxembourg, with partners from Cameroon, Germany, Ghana, and the United Kingdom
Project coordinator: Dr Michel Vaillant
Starting date: 01 January 2021
Duration: 42 months
EDCTP grant amount: EUR 2,112,690
Grant agreement: RIA2019PD-2880

PedMAb
Phase I/II study to determine Safety and Pharmacokinetics of subcutaneous administration of potent and broad anti HIV-1 neutralizing monoclonal antibodies, given to HIV-1 exposed neonates and infants

Ospedale San Raffaele SRL, Italy, with partners from France, Norway and South Africa
Project coordinator: Dr Gabriella Scarlatti
Starting date: 1 May 2021
Duration: 42 months
Grant amount: EUR 3,965,161
Grant agreement: RIA2019PD-2887

UNIVERSAL
Pharmacokinetic and safety studies of new antiretroviral formulations: expediting UNIVERSAL first and second line regimens for all children living with HIV in Africa

Fondazione PENTA ONLUS (PENTA), Italy, with partners from Cameroon, Cote d’Ivoire, France, Mali, the Netherlands, Senegal, Spain, Switzerland, Uganda, United Kingdom, United States and Zimbabwe
Project coordinator: Professor Carlo Giaquinto
Starting date: 1 January 2021
Duration: 60 months
Grant amount: EUR 3,999,818.75
Grant agreement: RIA2019PD-2882