Original Title: État des pratiques - Utilisation en contexte québécois des inhibiteurs de la tyrosine kinase du récepteur du facteur de croissance épidermique (EGFR) pour le traitement du cancer du poumon

Authors' objectives: INESSS has undertaken a major three-part project to explore the potential of clinical administrative data as a lever for improving cancer care practices [INESSS, 2021]. The present work concerns the part dealing with the assessment of the value of innovative therapies in the Québec real-world context. The primary objective was to estimate overall survival in patients receiving an epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI; gefitinib, afatinib or osimertinib) for the treatment of lung cancer in three indications and to determine whether these results are similar to those reported in published studies. The project's objectives also included providing a picture of the use of EGFR-TKIs in Québec, assessing these results in relation to the clinical practice recommendations and published data, and proposing certain avenues for improving practices. Lastly, the work sought to better understand the possibilities, limitations and issues related to this type of assessment in the real-world care context.

Authors' results and conclusions: RESULTS: (#1. General overview of EGFR-TKI1 use in Québec between April 1, 2001 and March 31, 2019 ) - As at March 31, 2019, 679 patients had received at least one EGFR-TKI. • In 2018-2019, the most recent fiscal year with complete data: 4375 EGFR gene mutation tests were performed in Québec laboratories; 199 physicians prescribed at least one EGFR-TKI; 217 patients started therapy with an EGFR-TKI. (#2. Use of EGFR-TKIs in Québec for the three indications of interest) - As at the patient inclusion (March 31, 2019) and follow-up (EGFR-TKI use and survival; March 31, 2020) cut-off dates: 457 had started therapy with gefitinib as first-line palliative treatment; 80 had started therapy with afatinib with the same intent; 119 had started therapy with osimertinib, after another EGFR-TKI given as first-line palliative therapy. An EGFR-TKI was the first treatment administered after a lung cancer diagnosis in 40.7% of the patients who received first-line gefitinib and in 31.3% of those who received first-line afatinib. The median number of days of EGFR-TKI dispensed in community pharmacies per patient ranged from 274.5 to 300 days (9.0 to 9.9 months), depending on the indication. The longest number of days of EGFR-TKI dispensed (maximum) was observed with gefitinib (2400 days, or 6.6 years). Median overall survival for those who received gefitinib as first-line palliative therapy was 18.9 months (95% CI: 16.3 - 21.9). Taking the margins of error into account, this result suggests that the overall survival achieved in Québec: Was not different from that in IPASS, one of the two studies submitted to INESSS for an evaluation for coverage purposes; Was not different from that in most of the studies selected for the comparison. (#3: Assessment of the state of Québec practice and avenues of improvement) - The analyses suggest that it took about 5 years before EGFR-TKIs were fully integrated into Québec practice. However, some clinicians seem to have been particularly proactive in integrating osimertinib into their practice. A few avenues should be considered to speed up access to novel therapies, especially when the eligibility criteria are based on new paradigms: Provide service corridors to facilitate access to companion tests in all regions of Québec and resources for providing the results in a timely manner; Improve the dissemination of information on the availability of novel therapies to administrators and clinicians; Promote the dissemination of this information to patients and patient associations. (#4: Elements to be taken into consideration regarding the evaluation of therapies in the Québec real-world context) - Two main elements should be taken into consideration regarding the feasibility of evaluating a therapy in the Québec real-world context: The availability of the data required for the evaluation. These data should enable one to:  Sensitively and specifically identify those who have received the therapy and those who have received the comparator treatment, if applicable;  Calculate the decisional endpoint;  Choose a valid design and criteria for drawing conclusions. 5 Having a realistic timeframe for obtaining results with acceptable accuracy according to the purpose of the evaluation. This timeframe will depend mainly on:  The number of patients who receive the therapy and for whom data are available;  The time to the occurrence of the endpoint event;  The effect size of the experimental therapy relative to the comparator treatment, if applicable. OUTLOOK: The third part of this exploratory project is underway. Its objectives are to characterize the clinical trajectories of lung cancer patients in the Québec real-world context, to assess these trajectories relative to the clinical practice guidelines, and to identify any heterogeneous practices. In time, the most useful results of the three parts of this project can be updated on a regular basis, other questions aimed at improving care can be addressed, and the methods developed for the study of lung cancer can possibly be adapted for other types of cancer.

Authors' methods: For this purpose, a global cohort of patients who received an EGFR-TKI between April 1, 2001 and March 31, 2019 was first created. Then, three cohorts corresponding to the indications of interest were created, specifically, with the use of a classification algorithm according to the line of treatment of the first EGFR-TKI received. Median overall survival for those who received gefitinib as first-line palliative therapy was 18.9 months (95% CI: 16.3 - 21.9). Taking the margins of error into account, this result suggests that the overall survival achieved in Québec: Was not different from that in IPASS, one of the two studies submitted to INESSS for an evaluation for coverage purposes; Was not different from that in most of the studies selected for the comparison.

Project Status: Completed

URL for project: https://www.inesss.qc.ca/en/publications/publications/publication/utilisation-en-contexte-quebecois-des-inhibiteurs-de-la-tyrosine-kinase-du-recepteur-du-facteur-de-croissance-epidermique-egfr-pour-le-traitement-du-cancer-du-poumon.html

Year Published: 2022

URL for published report: https://www.inesss.qc.ca/en/publications/publications/publication/utilisation-en-contexte-quebecois-des-inhibiteurs-de-la-tyrosine-kinase-du-recepteur-du-facteur-de-croissance-epidermique-egfr-pour-le-traitement-du-cancer-du-poumon.html

English language abstract: An English language summary is available

Publication Type: Other

Country: Canada

Province: Quebec

Organisation Name: Institut national d'excellence en sante et en services sociaux

Contact Address: L'Institut national d'excellence en sante et en services sociaux (INESSS) , 2021, avenue Union, bureau 10.083, Montreal, Quebec, Canada, H3A 2S9;Tel: 1+514-873-2563, Fax: 1+514-873-1369

Contact Name: demande@inesss.qc.ca

Contact Email: demande@inesss.qc.ca

Copyright: L'Institut national d'excellence en sante et en services sociaux (INESSS)