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Objectives: Patient and public involvement (PPI) in clinical research has a well-established infrastructure in the UK, and while there has been good progress within pharmaceutical-industry-sponsored research, further improvements are still needed. This review aims to share learnings from quality assessments of historical PPI projects within Pfizer UK to inform future projects and drive PPI progress in the pharmaceutical industry., Design and Setting: Internal assessments of Pfizer UK PPI projects were conducted to identify all relevant projects across the medicines development continuum between 2017 and 2021. Five sample projects were developed into case studies., Outcome Measure: Retrospective quality assessments were performed using the Patient Focused Medicines Development (PFMD) Patient Engagement Quality Guidance (PEQG) tool. Recommendations for improvement were developed., Results: Retrospective case study analysis and quality framework assessment revealed benefits of PPI to both Pfizer UK and to external partners, as well as challenges and learnings to improve future practice. Recommendations for improvement based on these findings focused on processes and procedures for PPI, group dynamics and diversity for PPI activities, sharing of expertise, the importance of bidirectional and timely feedback, and the use of understandable language in materials., Conclusions: PPI in medicines development is impactful and beneficial but is still being optimised in the pharmaceutical industry. Using the PFMD PEQG tool to define gaps, share learnings and devise recommendations for improvement helps to ensure that PPI is genuine and empowering, rather than tokenistic. Ultimately, these recommendations should be acted on to further embed PPI as an integral part of medicines development and health research within the pharmaceutical industry. This article includes a plain language summary in the supplement., Competing Interests: Competing interests: S-AD, SB, LC, SE, SH, BP and NB are employees of Pfizer and hold Pfizer stock. AB, RD, AD, EK, GP, JP and KS have no competing interests to report., (© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Competing Interests: This Comment represents a personal viewpoint as cancer patient advocates. Some authors are patients and advocates, some are advocates as charity professionals. The views expressed in this comment do not necessarily reflect those of the authors’ affiliations. RW has received honoraria from Boehringer Ingelheim, the Health Research Authority, and Birmingham University for advice as a patient expert; has received support for conference attendance from Sarcoma UK and Sarcoma Patients Advocacy Global Network (SPAGN); is a board member of SPAGN, a member of the National Cancer Research Institute (NCRI) Consumer Forum, and a patient panel member for the Medicines and Healthcare products Regulatory Agency. EK is the founder and director of Salivary Gland Cancer UK. LEM is Chief Executive of Fight Bladder Cancer, which receives financial support from industry. AB-B is Head of Health Improvement & Influencing for Pancreatic Cancer UK. RS is Consumer Forum Chair and Consumer Lead for NCRI; is a member of patient panels for Cancer Research UK, the National Institute for Health Research, and various charities. JR has received support for attending conferences from the British Thoracic Oncology Group (BTOG), Cancer Research UK, Roy Castle Lung Cancer Foundation, and NHS England; was a lay Director of Sandwell & W Birmingham CCG (to April, 2021), and is a lay steering group member of the BTOG. KO is Chair of the International Brain Tumour Alliance (IBTA), co-Chair of the European Cancer Organisation Patient Advisory Committee, steering committee member of the European Reference Network EURACAN; and IBTA has received honoraria from industry in respect of chairing or attending meetings.

Introduction: Care for head and neck cancers is complex in particular for the rare ones. Knowledge is limited and histological heterogeneity adds complexity to the rarity. There is a wide consensus that to support clinical research on rare cancer, clinical registries should be developed within networks specializing in rare cancers. In the EU, a unique opportunity is provided by the European Reference Networks (ERN). The ERN EURACAN is dedicated to rare adults solid cancers, here we present the protocol of the EURACAN registry on rare head and neck cancers (ClinicalTrials.gov Identifier: NCT05483374)., Study Design: Registry-based cohort study including only people with rare head and neck cancers., Objectives: to help describe the natural history of rare head and neck cancers;to evaluate factors that influence prognosis;to assess treatment effectiveness;to measure indicators of quality of care., Methods: Settings and participants It is an hospital based registry established in hospitals with expertise in head and neck cancers. Only adult patients with epithelial tumours of nasopharynx; nasal cavity and paranasal sinuses; salivary gland cancer in large and small salivary glands; and middle ear will be included in the registry. This registry won't select a sample of patients. Each patient in the facility who meets the above mentioned inclusion criteria will be followed prospectively and longitudinally with follow-up at cancer progression and / or cancer relapse or patient death. It is a secondary use of data which will be collected from the clinical records. The data collected for the registry will not entail further examinations or admissions to the facility and/or additional appointments to those normally provided for the patient follow-up. Variables Data will be collected on patient characteristics (eg. patient demographics, lifestyle, medical history, health status); exposure data (eg. disease, procedures, treatments of interest) and outcomes (e.g. survival, progression, progression-free survival, etc.). In addition, data on potential confounders (e.g. comorbidity; functional status etc.) will be also collected. Statistical methods The data analyses will include descriptive statistics showing patterns of patients' and cancers' variables and indicators describing the quality of care. Multivariable Cox's proportional hazards model and Hazard ratios (HR) for all-cause or cause specific mortality will be used to determine independent predictors of overall survival, recurrence etc. Variables to include in the multivariable regression model will be selected based on the results of univariable analysis. The role of confounding or effect modifiers will be evaluated using stratified analysis or sensitivity analysis. To assess treatment effectiveness, multivariable models with propensity score adjustment and progression-free survival will be performed. Adequate statistical (eg. marginal structural model) methods will be used if time-varying treatments/confounders and confounding by indication (selective prescribing) will be present., Results: The registry initiated recruiting in May 2022. The estimated completion date is December 2030 upon agreement on the achievement of all the registry objectives. As of October 2022, the registry is recruiting. There will be a risk of limited representativeness due to the hospital-based nature of the registry and to the fact that hospital contributing to the registry are expert centres for these rare cancers. Clinical Follow-up could also be an issue but active search of the life status of the patients will be guaranteed., Competing Interests: Paolo Bossi was part of the Advisory board or conference honoraria for: Merck, Sanofi-Regeneron, Merck Sharp & Dohme, Sun Pharma, Angelini, Bristol-Myers Squibb, Nestlè Christoph Benedikt Westphalen received honoraria from Bayer, Celgene, Ipsen, F. Hoffmann-La Roche Ltd, Servier, and Taiho, served in a consulting/advisory role for BMS, Celgene, Merck, Shire/Baxalta, Rafael Pharmaceuticals, RedHill BioPharma, and F. Hoffmann-La Roche Ltd, has received travel/accommodation expenses from Bayer, Celgene, RedHill BioPharma, F. Hoffmann-La Roche Ltd, Servier, and Taiho and has received research funding from F. Hoffmann-La Roche Ltd. Lisa Licitra participated to clinical studies that received research funding intended directly to Fondazione IRCCS Istituto Nazionale dei Tumori from Astrazeneca, BMS, Boehringer Ingelheim, Celgene International, Eisai, Exelixis, Debiopharm International SA, Hoffmann – La Roche ltd, IRX Therapeutics, Medpace, Merck-Serono, MSD, Novartis, Pfizer, Roche, Buran. Also, she declares that she recived occasional payment for her participation as a speaker at conferences/congresses or as a scientific consultant for advisory boards from Astrazeneca, Bayer, MSD, Merck-Serono, AccMed, Neutron Therapeutics, Inc All the other authors declare that they have no competing interests. I confirm that this does not alter our adherence to PLOS ONE policies on sharing data and materials., (Copyright: © 2023 Trama et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.)