PATIENT REPORTED OUTCOMES (PROs)


WORKING GROUP

Objectives

The ACCELERATE Patient-Reported Outcomes (PROs) Working Group was launched in 2023 to strengthen the use of patient-reported outcomes in paediatric oncology research. Despite growing recognition of their importance, PROs remain under-used in paediatric cancer clinical trials, even though their inclusion is recommended by regulatory agencies such as the FDA and EMA.


Our shared mission is to make the inclusion of PROs in paediatric trials systematic, feasible, and meaningful, ensuring that the experiences and voices of children are reflected in research and drug development.


Within ACCELERATE, the PROs Working Group connects experts from academia, regulatory bodies, patient advocacy, and industry. Together, we collaborate across five interconnected Work Packages (WPs), each focusing on a different aspect – from mapping existing resources to shaping regulatory pathways.


Working Packages

WP-1: Review of existing resources 


Lead: Ameeta Retzer (University of Birmingham)

Team: Sarah Al-Jilaihawi (ALADDIN Fellowship), Sarah Damery (University of Birmingham)


Aim: To identify and map existing resources supporting the use of PROs in paediatric cancer clinical trials.

This comprehensive scoping review takes a systematic approach to summarising available tools, guidance, and methodological frameworks, and to identifying key gaps in the current evidence base.


Status: Review completed; manuscript writing underway.

WP-2: Identification of obstacles


Lead: David Riedl (Medical University Innsbruck)

Team: Franziska Helmberg (Medical University Innsbruck), Kelly van Bindsbergen and Anne Westerweel (Princess Máxima Center)


Aim: To explore factors that hinder or facilitate the implementation of PROs in paediatric oncology clinical trials. Using a mixed-methods design, this work combines:

  • a literature review on known barriers and facilitators,
  • an online survey with principal investigators (PIs), and
  • follow-up interviews with PIs and industry sponsors.


Status: Literature review ongoing; survey currently open; interviews planned after survey closure.


💡 We invite participation in our ongoing survey here


WP-3: Extension of existing guidelines


Lead: Madeleine Adams (Cardiff and Vale UHB)


Aim: To ensure that clinical trial guidance is inclusive of paediatric oncology by developing a paediatric-specific extension to the SPIRIT-PRO guideline.

This work draws on findings from WP-1 and includes a systematic review and Delphi consensus process.


Status: Thematic analysis of the scoping review of WP-1 completed; mapping to current guidance (SPIRIT-PRO) underway.

WP-4: Education & Dissemination


Lead: Lenneke Schrier (Princess Máxima Center)


Aim: To ensure that the knowledge generated across the work packages reaches stakeholders in an accessible and practical format.

This includes developing education and training tools for all stakeholders, especially for investigators and for patients and families (e.g., webinars, toolkits, short guidance) and providing practical guidance for implementing PROs in paediatric trials.


Status: Planned to begin as preceding WPs conclude.

WP-5: Qualification advice


Lead: Pamela Kearns (University of Birmingham)


Aim: To lay the groundwork for the regulatory qualification of PRO tools and strategies in paediatric oncology. This will be achieved through aligning findings from the other work packages to support qualification efforts and collaborating with regulators (e.g., EMA, FDA) to define criteria for PRO qualification in paediatric contexts.


Status: Planned to begin as preceding WPs conclude.

Who we are

Chairs

Chair: David Riedl (Innsbruck University)


Work Package Leads:

  • Ameeta Retzer (University of Birmingham)
  • Madeleine Adams (Cardiff and Vale UHB)
  • Lenneke Schrier (Princess Máxima Center)
  • Pamela Kearns (University of Birmingham)


Core Group

Academia

Bryce B. Reeve​ (Duke University​)

Martha Grootenhuis ​(Princess Máxima Center)

Anne-Sophie Darlington​ (University of Southampton​)

Denise Connolly (SickKids)

Sae Ishimaru (Princess Máxima Center)

Kay Koehler (CSCC)

Meg Macy (Children's Hospital Colorado)

Inês Alves (ACCELERATE)

Leona Knox (Solving Kids' Cancer UK)

Sabine Verschueren (KU Leuven)

Francois Doz (Institut Curie)

Gabriel Revon-Riviere (La Timone Children's Hospital)

Ruzanna Papyan (PCBDC of Armenia)

Donato Bonifazi (EPTRI)


Regulators

Chantal Quinten (EMA)

Elizabeth Duke (FDA)


Advocates

Vickie Buenger (CAC2)

Willemijn Plieger (Dutch Childhood Cancer Organisation)

Kelli Wright (Day One)

Nicole Scobie​ (ACCELERATE​)


Industry

Karren Jackson (United Therapeutics)

Solange Corriol-Rohou (previously AZ)

Alexandra Paúl (IQVIA)

Lara Jenkins (Eisai EMEA)

Emilia Richter (Merck Healthcare KGaA)


Extended Group

Over 30 additional experts


We are very thankful for the hands-on involvement, dedication, and enthusiasm of everyone in our group!

Related Activities

Members of the PROs Working Group are involved in a variety of related projects and initiatives that align with our mission to strengthen the role of patient-reported outcomes in paediatric oncology.

These activities further contribute to advancing the field and complement the work of our five Work Packages.


Get Involved

We welcome collaboration and input from clinicians, researchers, patient organisations, regulators and industry partners.

📩 For more information or to get involved: contact@accelerate-platform.org