The Aparito Digital Endpoints Patient Group Accelerator Programme



For a medicine to be authorized, clinical efficacy and safety need to be established, usually through robust and relevant clinical trial data. There are several unique challenges in the development of orphan diseases treatments. Low patient numbers, an incomplete understanding of the disease pathology, phenotypic heterogeneity, and a lack of established endpoints are barriers to efficient and effective clinical trials.

A key aspect is the choice and selection of the primary and key secondary efficacy endpoints. A reliable, valid, sensitive, interpretable and meaningful endpoint is vital to a well-designed study. This is necessary both as the basis to demonstrate the clinical impact of any therapeutic intervention and to stand up to regulatory scrutiny.

Through the launch of the Aparito Digital Endpoints Patient Group Accelerator programme we are hoping to address the high unmet needs of patient communities by co-developing new digital endpoints with patient groups. Utilizing our patient facing mobile application to collect the data, those patient groups selected to join our programme will benefit from a purpose built technology solution.

the aim

Together, we will collaborate with selected patient groups to:

  • identify the disease hallmarks that are specific to that disease community

  • identify the best way to capture and measure those disease hallmarks

  • address any gaps in the development of new medicines or repurposed drugs 


endpoint design

As part of the programme we will conduct qualitative research in the form of interviews and focus groups with patients, carers and key opinion leaders to ensure that the selected endpoint(s) collect data is meaningful, important and relevant. In addition, we will deploy / seek some user-testing from the patients and families to ensure that the technology is feasible and acceptable in the context intended. 


Ultimately, these digital endpoints will be designed and validated to help solve many regulatory and reimbursement challenges in rare disease drug development and support the availability of new treatments for patients with rare diseases. Where appropriate, we will bring in representatives from pharma to seek their input on the project. 

Selection criteria

To be eligible for the Aparito Digital EndpointsPatient Group Partnership programme your patient group must fulfil the following: ​

  • Be legally established with an existing governance structure

  • Have on-going engagement and/or access to an international disease community 

  • Be familiar with the drug development pathway and clinical research activities

  • Have a reasonable documentation of the natural history of their disease 

  • Be collaborative, willing and have resources to actively participate at each phase of the project in close partnership with Aparito 

  • Must have engaged KOLs 

  • Must have a relationship with pharma and/or academic partners with a pipeline of medicine that might be close to starting / already started clinical trials (you will be asked details on this)



To apply, please register your interest here. We encourage you to ask for support in completing the application from experts you know to make sure the information is as complete as possible. We encourage you to submit your application even if your group does not have access to all of the information or resources requested.  

The two successful Patient Groups will benefit from the following aspects: 

  • Access to dedicated experts in regulatory science, clinical and patient advocacy 

  • Qualitative interviews to identify important disease hallmarks

  • User-testing of technology that might support capturing and measuring endpoints

  • Initial steps towards protocol design for the validation of such an endpoint 

  • Shared ownership of any new digital solutions co-developed 

If you have any questions about the Patient Digital Endpoints Group Accelerator Programme please get in touch.

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