Lung cancer patients treated with a new PD-1 IMP: A longitudinal, prospective, observational, multicentric study in France
CASE STUDY TYPE
Non-interventional, multicenter, single-cohort study
BACKGROUND
This is the most common histological subtype of lung cancer, accounting for approximately 85% of all cases. It is frequently diagnosed at an advanced stage and the 5-year survival rate varies from 9% when distant to 65% when localized.
Several clinical trials showed that the new PD-1 IMP provided best response rates with durable response, improved overall survival (OS) and health-related quality of life (QoL) than a specific chemotherapy medication.
The key objectives of this study were to describe the demographic and clinical characteristics of patients with this type of lung cancer (N ≈ 1,500) initiating treatment with this new PD-1 IMP as well as the survival outcomes over 36 months in a real-life setting in France.
METHODS
This was an international non-interventional, multicenter, single-cohort study. Data were collected retrospectively at the time of initiation and prospectively during treatment (ambispective data collection). No interventions, extra procedures, or extra visits were required.
OBJECTIVES
CO-PRIMARY OBJECTIVES
- To describe socio-demographic and clinical characteristics of patients
- To describe treatment effectiveness (i.e OS) at 36 months
EXPLORATORY OBJECTIVES
To examine the prognostic association between the OS at 36 months and the characteristics of the patients
SECONDARY OBJECTIVES
- To estimate the OS at 12 and 24 months
- To evaluate the incidence, severity and management of IMP adverse event (AE) and adverse drug reactions (ADR) at 36 months
- To estimate intermediate endpoints (progression-free survival and objective response rate) at several timepoints
- To describe QoL and working status at several timepoints
- To describe the management of patients, treatment patterns, and variations in treatment patterns at several timepoints
- To describe the evolution over the 36-month follow-up period of major clinical parameters
SCIENTIFIC and DATA vALORISATION
- Three poster presentations at major conferences
- Two publications
- Excellent recognition of the Scientific Community regarding the extend and quality of the study database, useful for future research.
Key factors of success
- Strong and long-term involvement of ICTA’s collaborators (58 in total)
- Strong involvement of the Scientific Committee
- Regular communication: internally, with the centres and the Sponsor
- ICTA’s ability to develop specific iTools in a short time upon Sponsor’s request
- ICTA’s expertise in data management and AE management
- Flexibility and adaptability to deadlines by ICTA.
LESSONS LEARNED to speed up the start-up phase of the next study
- The value of close communication between ICTA, the Sponsor and any third parties (e.g. Scientific Committee and regional expert physicians) with regular teleconference meetings.
- To well define the Sponsor’s expectations in terms of objectives, statistical analyses and results presentation.
- The importance to validate all study documents and make sure that they were perfectly understood by the Sponsor and the Scientific Committee.
CHALLENGES
| KEY CHALLENGES | ICTA’s SOLUTIONS | ICTA’s ACHIEVEMENTS |
|---|---|---|
| . High number of physicians to be contacted (±2,500) and enrolled (± 250) in order to recruit 1,500 patients | . Involvement of regional expert physicians for site initiation and eCRF training . Close follow-up of sites by CRO | · 205 sites and 261 physicians agreed to participate and 146 and 186 respectively were active · 1,495 patients were recruited ▶ Recruitment ended 5 months earlier than the Sponsor’s expectations |
| . High amount of data to be collected/managed In particular high number of AE/ADRs to be handled and reconciled with Sponsor PV | . Development of a customed eCRF restricted to “must-have” data for an easier data collection and reduced burden . Hotline for site support . Close follow-up of sites by CRO . Close follow-up of AEs by both vigilance departments | . 12,900 visits checked remotely . >6,700 questionnaires entered in the database . >16,500 treatments coded . ±12,400 AE forms collected and treated |
| . Many different statistical analyses (planned in the statistical analysis plan (SAP) . Several requests of additional analyses (not planned in the SAP) . Multiple Sponsor reviews of the SAP, TFLs and CSR | . Anticipation and organization . SAP completed with additional analyses . Update of the statistical program and associated QC . Timing management of each 5 interim analyses and final analysis (update of the SAP, new version of TFLs) . Several teleconference meetings to understand the Sponsor’s expectations | . Adaptability and flexibility of ICTA to keep the timelines & deadlines . TFLs of > 4,000 pages . TFLs addendum of > 600 pages . Enabling data valorisation through a clinical report study (CSR) . First exhaustive database providing long-term real life data in this disease |