PRO4All
Principal investigator: Oriana Ciani
Team UB/Cergas: Michela Meregaglia, Francesco Malandrini
Partners: AIOM, AIM, UNIAMO, FAVO, AISM, SIN, FICOG
Sponsor: Roche SpA
Duration: 2021 - 2024
Abstract:
The PRO4ALL working group was set up in 2021 with the aim of understanding the role of the “patient voice” in clinical research and regulatory pathways, with a specific focus to Patient Reported Outcomes (PROs) and their measurement. The working group comprises Associazione Italiana Oncologia Medica (AIOM), Associazione Italiana di Miologia (AIM), Associazione Italiana Sclerosi Multipla (AISM), Federazione Italiana delle Associazioni di Volontariato in Oncologia (FAVO), Federazione dei Gruppi Cooperativi Oncologici Italiani (FICOG), Società Italiana di Neurologia (SIN), Federazione Italiana Malattie Rare (UNIAMO) together with SDA Bocconi and the unconditional funding from Roche.
Patient-reported outcomes refer to any data on a patient's health status that comes directly from the patient, without interpretation by the clinician or others. The measurement of PROs is done by means of instruments, scales and questionnaires, collectively called patient reported outcome measures (PROMs) which allow to gather the perspective of the individual living with the disease or receiving a given treatment on physical, emotional, cognitive, social functioning, and psychological well-being and health-related quality of life.
Over the last years, the project has produced a number of output: from the evaluation of the role of PROs in EMA assessment reports between 2017 and 2022, to the analysis of the association between PROs and drug reimbursement class and innovative status assigned by AIFA. Among reports related to authorized medicines concerning neurological or oncology indications, 57% or 51% reported any use of PROMs, respectively.
In relation to this, we developed a comprehensive archive of patient-reported outcome measures (PROMs) in several different conditions and identified their main characteristics and target outcome domains.
One of the conclusions reached by the working group is that a harmonisation and standardisation of the use of PROs and PROMs in clinical research is required, together with a commitment of relevant institutions to ensure that patients are at the centre of evaluation processes at every stage of drug development.
Material produced and publications:
Valsecchi AA, Giovanardi F, Malandrini F, Meregaglia M, Servetto A, Bennati C, Pinto C, Di Maio M, Ciani O. "Breast-cancer specific comprehensive archive of Patient-Reported Outcome Measures (PROMs) for clinical research and clinical practice in oncology: Results from the PRO4All project." Breast. 2024 Sep 28;78:103817. doi: 10.1016/j.breast.2024.103817.
Malandrini F, Meregaglia M, Di Maio M, Pinto C, De Lorenzo F, Ciani O. "The development of an archive of patient-reported outcome measures (PROMs) in oncology: The Italian PRO4All project." Eur J Cancer. 2024 Aug;207:114161. doi: 10.1016/j.ejca.2024.114161.
Ciani O, Meregaglia M, Battaglia MA, Brichetto G, Conte A, Gasperini C, Sansone V. "Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017-2022." Neurol Sci. 2023 Aug;44(8):2933-2937. doi: 10.1007/s10072-023-06825-6.
Ciani O, Meregaglia M, De Lorenzo F, Perrone F, Pinto C. "Patient-Reported Outcome Measures in Oncology Drugs Approved by the European Medicines Agency, 2017-2021." JAMA Netw Open. 2023 Jan 3;6(1):e2251564. doi: 10.1001/jamanetworkopen.2022.51564.
Meregaglia M, Malandrini F, Angelini S, Ciani O. "The Assessment of Patient-Reported Outcomes for the Authorisation of Medicines in Europe: A Review of European Public Assessment Reports from 2017 to 2022." Appl Health Econ Health Policy. 2023 Nov;21(6):925-935. doi: 10.1007/s40258-023-00827-3.
Malandrini F., Borroni C., Meregaglia M., Sarra M., Ciani O. "The role of patient-reported outcomes in reimbursement decisions and drug innovation in Italy Glob Reg Health Technol Assess." 2023 Feb 9;10:12-17. Italian. doi: 10.33393/grhta.2023.2514.
Dissemination:
Manifesto PRO4all: Pazienti PROtagonisti per la Salute di tutti
Relazione AIFA Incontra 12.12.2023
Gruppo Pro4All: “Pazienti PROtagonisti per la salute di tutti”
PRO4all: Pazienti PROtagonisti per la salute di tutti
PRO4All: l’importanza crescente dei PROs in oncologia.
PRO4all - Pazienti PROtagonisti per la salute di tutti
Percorsi di cura, con i PROs si possono migliorare. Il Manifesto del Gruppo PRO4All
PRO4all - Pazienti PROtagonisti per la salute di tutti Associazione PaLiNUro
PRO4all – Pazienti PROtagonisti per la salute di tutti Farmindustria
CERGAS Seminar "Evidence generation and use of patient-reported outcomes: the PRO4all project"
"Dare voce al paziente per migliorare la Sanità. Ecco come"
Festival di Salute 2024: prendiamoci la vita
Presentations:
EuHEA 5-8 July 2022 (Oslo) – Oral presentation: “The use of patient-reported outcomes for new drugs approval in Europe: a review of European Public Assessment Reports”
ISPOR 6-9 November 2022 (Vienna) – Poster: “The Use of Patient-Reported Outcomes for New Drugs Approval in Europe: A Review of European Public Assessment Reports From 2017 to 2021”
PATH June 2022 speaker– join our future 06/2022
AIES 8-9 September 2022 (Messina) – Oral presentation: “Il ruolo degli esiti riferiti dal paziente nelle decisioni di approvazione e rimborso dei farmaci”
ISPOR 12-15 November 2023 (Copenhagen) - Poster: “The Development of a Comprehensive Archive of Patient-Reported Outcome Measures (PROMS) for Clinical Research and Clinical Practice in Oncology”
AIES 5-6 December 2023 (Rome) – Oral presentation: “The development of a comprehensive archive of Patient-Reported Outcome Measures (PROMs) for clinical research and clinical practice in oncology”
ISPOR Italy Rome Chapter 3-4 July 2023 (Milan) – oral presentation (invited speaker): “PROs - Il ruolo della qualità della vita e degli esiti riferiti dal paziente nelle decisioni di rimborso dei farmaci in Italia”
Path 19-20 October 2023- speaker - Il valore generato dai pazienti
Senato della Repubblica 30 January 2024 (Rome) – Oral presentation: “Pazienti protagonisti per la salute di tutti. La voce che può fare la differenza: il valore dei PRO per la sostenibilità del sistema salute”
ISPOR 17-20 November 2024 (Barcelona) – Poster (upcoming): “The Development of a Comprehensive Repository of Patient-Reported Outcome Measures (PROMs) for Spinal Muscular Atrophy and Duchenne Muscular Dystrophy”
Festival della salute 26-27 October 2024 Padova - La parola ai pazienti
Video: