Purpose

The purpose of the PFDD SIG is to connect statisticians in pharmaceutical industry roles who work on progressing the inclusion of patient-reported outcomes (PROs) and clinical outcome assessments (COAs) across all phases of drug development process to share their knowledge and also lead statistical thinking and methods to solve common statistical challenges of use of COAs in drug development.

Objectives

• Advance statistical methodology for specific PFDD topics – currently (and to be expanded):
  • Pain estimands and estimators (including “missing” data)
  • Estimands and estimators for PROs assessing tolerability and safety
  • Use of PROs in early phase studies and their role in dose finding/optimization
  • Oncology estimands and estimators
  • Exploring use of SEM/latent variable modelling approaches in assessment of treatment effect estimation for COA endpoints
  • Definition of endpoint based on intensive longitudinal data, e.g. diary data/ data from wearables/sensors

• Education for industry statistician & knowledge sharing: sharing good practices and standards for PRO and COA based endpoints and are more widely disseminated to the PSI/EFSPI community and adopted in drug development across all therapy areas.
• Interact with regulators, payers, patients and the broader clinical community to obtain a better understanding of their requirements relating to PFDD
• Collaboratively partner with other SIGs in this area, either within PSI/EFSPI or in other groups, e.g., PSI/EFSPI HTA SIG, PSI/EFSPI Regulatory SIG, PSI/EFSPI Biomarkers SIG, ISOQoL Statistics SIG, SISAQoL etc.

Who we are

How to get in touch

If you have any questions relating to PFDD/PROs/COAs or need further information contact Rachael Lawrance (co-chair) or Konstantina Skaltsa (co-chair).

Events

The PFDD SIG holds monthly meetings with all members, as well as individual meetings among members of active substreams.
The PFDD SIG will host a session at PSI Conference 2024 (Monday 11-12:30).

Resources

Previous work completed (oncology estimands) as a PRO Task Force of the OncoEstimand WG: https://oncoestimand.github.io/oncowg_webpage/docs/taskforces.html

1. FDA PFDD guidance series: https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical
   1. Guidance 1: Collecting comprehensive and representative input (Final)
   2. Guidance 2: Methods to identify what is important to patients (Final)
   3. Guidance 3: Selecting, developing of modifying fit-for-purpose clinical outcomes assessments (Final)
   4. Guidance 4: Incorporating clinical outcome assessments into endpoints for regulatory decision making

1. FDA Core Patient-Reported Outcomes in cancer clinical trials guidance (2021 - Draft):  https://www.fda.gov/regulatory-information/search-fda-guidance-documents/core-patient-reported-outcomes-cancer-clinical-trials
2. Submitting patient-reported outcome data in cancer clinical trials (2023 – Final): https://www.fda.gov/regulatory-information/search-fda-guidance-documents/submitting-patient-reported-outcome-data-cancer-clinical-trials
3. EMA Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man - the use of patient-reported outcome (PRO) measures in oncology studies - Scientific guideline (2016): https://www.ema.europa.eu/en/appendix-2-guideline-evaluation-anticancer-medicinal-products-man-use-patient-reported-outcome-pro-measures-oncology-studies-scientific-guideline
4. Coens C et al (2020) International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium. https://doi.org/10.1016/S1470-2045(19)30790-9