2024 - KCE Trials 2024 themed call for proposals on endometriosis care

Background

Many questions in healthcare are currently not sufficiently studied in clinical trials, despite their high societal importance (e.g. comparing medicines which are commonly used but which haven’t been directly compared, comparing lifestyle changes to a medical intervention, comparing surgery to physiotherapy for specific indications or questions addressing the needs of older people). 'Comparative effectiveness' studies compare two treatment options (with ‘no treatment’ being one of the possible treatment options) that are already in use in clinical practice in a given indication, but are not yet sufficiently directly compared (i.e. which of the two treatment options works best in daily practice and is the most cost-effective). Since 2015, KCE is managing a national funding programme of such pragmatic practice-oriented clinical trials, the KCE Trials Programme. To date, over 50 clinical trials have been selected within this programme (see funded trials for more information on these trials or our dynamic dashboards for progress in recruitment). 

This year in addition to the investigator-led national call for comparative effectiveness proposals (see calls for more information on past and present calls), KCE Trials is launching a themed call focusing on endometriosis care.

Endometriosis is a chronic, oestrogen-dependent, benign, inflammatory condition characterised by the presence of tissue outside the uterus that resembles endometrium (the lining of the uterus), mainly on pelvic organs and tissues. Endometriosis can start at first menstruation and last until the menopause, or beyond. Some women with endometriosis don’t have any symptoms at all, while others suffer from various symptoms such as life-impacting pain during menstruation, sexual intercourse, bowel movements and/ or urination. Other symptoms include chronic pelvic pain, infertility, abdominal bloating, nausea, fatigue, and sometimes depression and anxiety.

Themed calls invite proposals focusing on a particular research or disease area and can be used to encourage research in an area of particular interest, to align with national priorities or for which specific research needs have been identified.

 In parallel with the publication of the KCE report on endometriosis, and due to the findings of the need for more research in this area, KCE Trials is launching a themed call for non-commercial comparative effectiveness studies in endometriosis care. This can be questions comparing diagnosis and treatments for endometriosis or suspected endometriosis.

The submitted trials should contribute to better patient care, should show clear value for money and should have the potential for return on investment. Accepted study interventions for treatment are not limited to drugs or medical devices but also include a broad range of healthcare interventions, for example psychotherapy, physiotherapy, diets, diagnostic tests or surgery. In addition, repurposing trials using off-patent drugs are eligible for funding under certain conditions. The budget available for this call amounts to € 3.0 million. 

Scope

Inclusion criteria 

The study falls within the theme of endometriosis care

• Participants in the study have either suspected or confirmed endometriosis. 

The primary aim of the study is non-commercial, this means:

• The candidate sponsor (and the Belgain Coordinating Centre 'BCC', if applicable) should be a non-profit organisation that can function as sponsor of non-commercial multicentre trials and has the ability to comply with all sponsor related obligations. 
• The holder of the intellectual property rights on the studied intervention or comparator to which the trial relates is neither directly nor indirectly the sponsor/BCC of the trial.
• The sponsor exercises the intellectual property rights to the concept of the trial, its implementation and the scientific data resulting from it.
• If there is a commercial interest in the study (e.g. the study requires the use of drugs, apps or medical devices), an exclusivity criterion comes into play. In general, the results of the study cannot bring a potential benefit to a single company only.  
• Studies on the effect of medical devices, tools (including software and apps) or on-patent drugs are only in scope, provided that there is a strong justification why the study would not be performed by the marketing authorisation holder or manufacturer. In this situation an expected high return on investment is required. 
• The funding granted under the research agreement complies with the European state aid regulations. 
• The sponsor, chief investigator and research team should have no conflict of interest in regards with the performance and possible results of the trial.

Design

• The trial is randomised (at individual level or in clusters) and multi-centric (with at least one recruiting centre in each region, unless there is a good justification not to do so).

Comparative effectiveness studies

• The trial is a comparative effectiveness trial, comparing two therapeutic or diagnostic interventions (no treatment/no test can also be an option) that are already in use in daily practice in Belgium in the studied indication. 
• Medication used off-label in daily practice is considered in scope.
• Studying an intervention that is already in use in a patient group, (e.g. adults), in another patient group, (e.g.; children, elderly, oldest old) can be considered in scope. A change of timing, e.g. first line versus second line, or change in dosing or duration of the treatment can also be considered in scope. 

Drug repurposing studies

• Repurposing of a drug(s) for an indication for which it is currently not used in daily practice may be in scope if the following 4 conditions are met:
  • The drug is off-patent and there is no secondary patent limiting the freedom to operate or implementation of the study.
  • The available evidence suggests that there is a potential for high return on investment (see Annex I).
  • There is substantial underlying peer-reviewed evidence that supports moving to a large-scale confirmatory randomised trial. 
  • The drug is authorised and commercialised in Belgium (or in use in daily practice in Belgium)

Exclusion criteria 

Started studies

• Studies for which recruitment has already started in Belgium, or which have already been submitted to a Belgian ethics committee and/or to the FAMHP (Federal Agency for Medicines and Health products) are out of scope. International studies that have already started abroad (but not in Belgium) are eligible if all other criteria are met.

Implementation studies and quality improvement studies

• If the primary aim of the studied intervention is to promote the uptake of research findings, the study is considered implementation research, which is out of scope. Interventions that aim to improve implementation of existing guidelines, health policies, programmes and practices are also out of scope, as are studies that aim to improve quality of care.

Development and innovation studies. 

• Drugs without marketing authorisation in Belgium are out of scope except if already widely used in Belgium. Medical devices without a CE label are out of scope. 
• All types of interventions, including interventions related to organisation of care, should already be in use in practice, outside of the framework of research or development. Interventions that have been used only within the framework of clinical research or pilot testing are out of scope.
• Proof of concept studies are out of scope. There should be substantial underlying peer-reviewed evidence that supports moving to a large-scale confirmatory randomised trial

Mobile health studies (apps)

• Software and apps used as part of the intervention that are not fully developed, piloted and that do not meet all regulatory requirements on data protection are out of scope. Apps that are not used in daily practice in Belgium are also out of scope. 
• Study proposals on software and apps should also include an implementation plan which is in agreement with the non-commercial aim of the KCE Trials programme. Proposals without this implementation plan will be considered out of scope.

Who can submit to the 2024 endometriosis care call?         

This call is investigator-led, meaning that investigators submit their own research idea as a study proposal outline and should have the support of a non-commercial sponsor to perform the trial themselves. It is strongly recommended that trials are developed and conducted with the support of a clinical trial centre or equivalent. Only multicentre studies are eligible, with participating centres including at least one recruiting centre from each region (Flanders, Brussels, Wallonia), unless there is a good justification not to do so. Also, trials run in the first-line setting, for example in general practice, should have good representation from participating investigators in all three regions

The participation of Belgian centres to an international non-commercial trial within the scope of this call, is also eligible for funding. 

The aim of the study cannot be commercial, and the sponsor should be a non-commercial sponsor, in Belgium or abroad. In the latter case, KCE Trials will provide funding for the participation of Belgian centres through a Belgian coordinating centre (BCC).

A letter of support, using the template provided by KCE Trials, signed by the candidate sponsor confirming acceptance of the trial sponsor obligations and acceptance of the terms and conditions included in the template KCE-sponsor research agreement should be included in the submission. The Belgian candidate sponsor or the BCC should be a non-profit organisation that can function as sponsor of non-commercial multicentre trials and has the ability to comply with all sponsor related obligations. If the sponsor is located abroad, a signed commitment letter by the international sponsor confirming acceptance of the KCE Trials terms and conditions regarding the non-commercial nature of the trial and data sharing conditions (see below) and a signed letter of support by the BCC should be included, both using the KCE Trials templates.

If funding is requested for participants recruited abroad, justification should be provided and will need to be approved. In general, for trials with a Belgian sponsor and participants recruited abroad, participation of other funders, to cover site costs for participants recruited abroad and to contribute to the sponsor costs, will be required.

Call text

You can find the complete call text here.

How to submit ? 

• The call will be open to submissions from 21st May 2024 until 13h on 24th September 2024.
• As a first step, candidates are asked to submit a research outline. Shortlisted candidates will be invited to submit a full research proposal (FRP) (with the study protocol) in a second step. 
• We suggest to contact KCE Trials (trials”at”kce.fgov.be) in advance if you want to resubmit a proposal that was not funded in a previous call.
• All submissions must be done using the KCE Trials online submission platform. To be able to submit, you have to create an account, if you do not already have one. Please create an account as soon as possible and at least 1 week before the submission deadline to enable us to correct possible problems with the online submission platform. Once you have created the account, you can find information to start your submission under the “funding opportunities” tab.
• You may prefer to prepare your application using the application form in Word format, which is available below (section Downloads) for your convenience (no application submitted in the word template will be accepted). This document also contains guidance on how to complete your application.
• Only the chief investigator can start an application and complete the submission. However, other members of the research team can help completing the application form if added to the contacts list. You are encouraged to contact your statistician, legal advisor, health economist, clinical trial centre or equivalent early in the process and give them access to your application to complete their parts. 
• A detailed budget should be submitted using the dedicated budget tool, for which separate guidance notes are available. It is advisable to complete the budget tool in collaboration with the clinical trial centre.  
• All documents for the submission are detailed below in the “Downloads” section of the 2024 investigator-led call.
• To be deemed valid and to enter the selection process, a submission should:
• Be written in English.
• Be submitted through the online submission platform before the deadline. 
• Include details of chief investigator and candidate sponsor.
• Include a sponsor support letter using the KCE Trials template or, for international trials with a non-Belgian sponsor, a support letter from the Belgian coordinating centre and a commitment letter signed by the international sponsor, using the applicable KCE Trials templates. The letter of support needs to be signed by the legal representative of the sponsor (usually the CEO), or an official delegate.
• If there is any collaboration with a commercial partner (e.g. delivery of free/discounted medication, apps, medical devices etc.), a signed commitment letter from the commercial partner accepting the KCE Trials terms and conditions using the applicable KCE template or the contract should be included in the initial submission in order to assess the non-commercial nature of the trial. Simple purchasing under normal market condition does not require a signed commitment letter. 

Downloads and links

All downloads and links available on the webpage of the 2024 investigator-led call also apply to the 2024 themed call on endometriosis care. 

Contact and further information

For any questions, contact us at trials[@]kce.fgov.be

Information

Presentations given at the Information session on 7th June can be found on this page (please scroll to the bottom of the page).

Do you still have a specific question about your application? You can always contact the Trials team members by mail (trialsATkce.fgov.be). A zoom appointment can be made for a short discussion.