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P3.07-007 Compassionate Use Program for New Cancer Drugs in Israel - Shortcut for Reimbursement Approval
S1434
cancer care, there is a paucity of literature exploring preferences for the collateral damage associated with long-time side effects. We estimated the treatment preferences of lung cancer survivors and caregivers, and the time equivalents for multiple domains of long-term side effects. Methods: Through rigorous engagement of a national advisory board of lung cancer survivors, a discretechoice experiment (DCE) was developed, pretested and piloted. The DCE was administered to lung cancer survivors and caregivers at a national summit. Respondents completed 13 paired-comparison choice tasks described across six attributes: progression-free survival (PFS), short-term side effects, and physical, emotional, cognitive, and functional long-term side effects. A continuous preference model was estimated using mixed logit. Using PFS as the numeraire, the preference for avoiding sideeffects were estimated using their time equivalents by using maximum simulated likelihood. Results: Of 114 survey participants, 102 (89.4%) completed all choice tasks - although no difference was identified between those who did not complete the task (p>0.05 for all observed characteristics). All attributes were statistically different form the null (p<0.001). Respondents valued a one-unit decrease in functioning the most, valuing it equivalent to extending PFS by 3.67 months. Changes in physical (2.34) and cognitive (2.29) were valued more than a composite of all short-term side effects (1.83). Heterogeneity analyses (see figure 1) indicated that avoiding long-term side effects could be valued even more highly for some respondents. For example. the 95% confidence interval for time equivalence of functional long-term side effects ranged from 0.62 to 13.31 months.a
Journal of Thoracic Oncology
Vol. 12 No. 1S
Conclusion: Despite the limitation of this small, retrospective study, the results indicate that avoiding the long-term side effects could have significant value, especially as many patients experience moderate longterm side effects across multiple domains. Keywords: patient preference, public health, discrete choice experiment, health policy
P3.07-007 Compassionate Use Program for New Cancer Drugs in Israel - Shortcut for Reimbursement Approval Topic: Therapy and Economics Hiba Reches,1 Inna Erlich,1 Elizabeth Dudnik,1 Alona Zer,1 Daniel Goldstein,2 Laila Roisman,2 Nir Peled1 1Thoracic Cancer Unit, Davidoff Cancer Center, Petach Tiqwa/Israel, 2Davidoff Cancer Center, Petach Tiqwa/Israel Background: Drug accessibility and reimbursement remains a major challenge across the globe. The Israeli Ministry of Health (MOH) approves drugs based on previous approval by the FDA and EMA-EU. Compassionate use programs (CPU) represent the use of a compound approved by the FDA/EMA-EU before its approval by local regulatory authorities. CPU provides accelerated access to novel compounds to patients otherwise unable to get the treatment. Methods: This is a retrospective analysis of 102 patients treated with nivolumab, osimertinib, or nintedanib within a CPU in a single tertiary Israeli cancer center. Basic patient demographics, different logistic treatment aspects and the time from FDA/EMA-EU approval to reimbursement approval for these compounds in Israel were analyzed. Results: We started Nintedanib program by July 2014 when the official MOH approval was 16 months later in Nov 2015. Osimertinib program was started a year before the official approval by MOH and was approved for reimbursement 4 months prior to drug registration. Nivolumab for Non-squamous was started 6 months before approval, while for Squamous the label was approved by MOH 2 months after starting the compassionate program. Reimbursement approval was received 6 months thereafter for nivolumab (Squamous NSCLC). Two out of the three drugs in the program were approved for reimbursement, one of them even before MOH registration.
January 2017
Abstracts
S1435
we are handling advanced and metastatic adenocarcinomas in NSCLC patients.
Conclusion: Compassionate use programs allow access to new cancer drugs prior to their approval by the regulatory authorities, increases physicians’ experience with novel compounds and may affect reimbursement approval.
Results: We found 182 patients with NSCLC (adenocarcinoma), from those 72 was in stage IIIB-IV or with relapsed disease. In 37/72 (51,38%) cases some molecular testing was done (EGFR, ALK); EGFR 26 (36,1%) WT: 9/26 (34,6%), mut: 9/26 (34.6%)(in confirmation), UK (data in confirmation): 8/26 (30,7%); ALK: 10 (13,8%), w-o transloc.: 6/10 (60%); wtransloc: 1/10 (10%); UK (data in confirmation)3/10 (30%). From 72 patients, nine (12,5%) was included in clinical trials; 6/ 72 (8.33%) received TKI out of a trial. From there just 2/ 6 received TKI as first line, and 4/6 as a second line and beyond. Conclusion: It will be done after our sample, and assay tests results review. Keywords: EGFR, ALK, Public Health Service, Metastatic NSCLC
Keywords: Compassionate use program, osimertinib, lung cancer, Nivolumab
P3.07-008 METASTATIC NSCLC: Treatment Reality from 182 Cases of Lung Adenocarcinoma in a Brazilian Public Cancer Health Service Topic: Therapy and Economics Leonardo Lago,1 Bruna Da Silva,2 Gabriel Lenz,2 Fernanda Bruzzo,2 Vinicius Da Silva3 1Oncology, Hospital Sao Lucas Da Pucrs, Porto Alegre/Brazil, 2 Faculdade de Medicina Da Pucrs, Porto Alegre/Brazil, 3 Professor of Pathology, Faculdade de Medicina Da Pucrs, Porto Alegre/Brazil Background: In Brazil, lung cancer is a public health problem as in the world. The national Incidence in 2016 is estimated in 28.220 new cases.1 In Rio Grande do Sul, an etimated number of new cases is 4.240.1 Is well established molecular testing for EGFR, ALK mutations are mandatory to treat metastatic adenocarcinomas to indicate target therapy, or not. Prevalence of EGFR mutations in adenocarcinomas, for example, are estimated from 25,5% to 30,4%.2,3 Although many guidelines advocates to use a TKI as first line therapy, in Brazil is difficult because nor the drugs, nor the assays are available to public health service, except by legal measures,4 or support from pharmaceutical industry Methods: We made a cross-sectional study from January 2013 to May 2016, collecting data from medical records in our institution, to evaluate our population and how
P3.07-009 Use of Adjuvant Chemotherapy for NonSmall Cell Lung Cancer: The Real-World Clinical Practice in Taiwan Topic: Therapy and Economics Bin-Chi Liao,1 Yu-Yun Shao,1 James Chih-Hsin Yang,1 Ho-Min Chen,1 Ann-Lii Cheng,1 Mei-Shu Lai2 1 Department of Oncology, National Taiwan University Hospital, Taipei/Taiwan, 2Institute of Epidemiology and Preventive Medicine, College of Public Health, National Taiwan University, Taipei/Taiwan Background: Adjuvant chemotherapy is the standard treatment for selected patients with non-small cell lung cancer (NSCLC) following curative surgery. This study evaluated the use of adjuvant chemotherapy for these cases among the general population in Taiwan. Methods: A population-based cohort was established by searching the database of the Taiwan Cancer Registry System to identify patients newly diagnosed with NSCLC for the period covering 2005 to 2009. Our target was patients with stage I, II and IIIA NSCLC who had undergone curative surgery. Medication prescription data were retrieved from the National Health Insurance Research Database, Taiwan. Chemotherapy administered within 3 months after the surgery was defined as adjuvant chemotherapy. Results: A total of 5789 patients received curative surgery for NSCLC, and the 1277 (22.1%) who had undergone adjuvant chemotherapy were included in this study. Overall, the most common adjuvant chemotherapy
cancer care, there is a paucity of literature exploring preferences for the collateral damage associated with long-time side effects. We estimated the treatment preferences of lung cancer survivors and caregivers, and the time equivalents for multiple domains of long-term side effects. Methods: Through rigorous engagement of a national advisory board of lung cancer survivors, a discretechoice experiment (DCE) was developed, pretested and piloted. The DCE was administered to lung cancer survivors and caregivers at a national summit. Respondents completed 13 paired-comparison choice tasks described across six attributes: progression-free survival (PFS), short-term side effects, and physical, emotional, cognitive, and functional long-term side effects. A continuous preference model was estimated using mixed logit. Using PFS as the numeraire, the preference for avoiding sideeffects were estimated using their time equivalents by using maximum simulated likelihood. Results: Of 114 survey participants, 102 (89.4%) completed all choice tasks - although no difference was identified between those who did not complete the task (p>0.05 for all observed characteristics). All attributes were statistically different form the null (p<0.001). Respondents valued a one-unit decrease in functioning the most, valuing it equivalent to extending PFS by 3.67 months. Changes in physical (2.34) and cognitive (2.29) were valued more than a composite of all short-term side effects (1.83). Heterogeneity analyses (see figure 1) indicated that avoiding long-term side effects could be valued even more highly for some respondents. For example. the 95% confidence interval for time equivalence of functional long-term side effects ranged from 0.62 to 13.31 months.a
Journal of Thoracic Oncology
Vol. 12 No. 1S
Conclusion: Despite the limitation of this small, retrospective study, the results indicate that avoiding the long-term side effects could have significant value, especially as many patients experience moderate longterm side effects across multiple domains. Keywords: patient preference, public health, discrete choice experiment, health policy
P3.07-007 Compassionate Use Program for New Cancer Drugs in Israel - Shortcut for Reimbursement Approval Topic: Therapy and Economics Hiba Reches,1 Inna Erlich,1 Elizabeth Dudnik,1 Alona Zer,1 Daniel Goldstein,2 Laila Roisman,2 Nir Peled1 1Thoracic Cancer Unit, Davidoff Cancer Center, Petach Tiqwa/Israel, 2Davidoff Cancer Center, Petach Tiqwa/Israel Background: Drug accessibility and reimbursement remains a major challenge across the globe. The Israeli Ministry of Health (MOH) approves drugs based on previous approval by the FDA and EMA-EU. Compassionate use programs (CPU) represent the use of a compound approved by the FDA/EMA-EU before its approval by local regulatory authorities. CPU provides accelerated access to novel compounds to patients otherwise unable to get the treatment. Methods: This is a retrospective analysis of 102 patients treated with nivolumab, osimertinib, or nintedanib within a CPU in a single tertiary Israeli cancer center. Basic patient demographics, different logistic treatment aspects and the time from FDA/EMA-EU approval to reimbursement approval for these compounds in Israel were analyzed. Results: We started Nintedanib program by July 2014 when the official MOH approval was 16 months later in Nov 2015. Osimertinib program was started a year before the official approval by MOH and was approved for reimbursement 4 months prior to drug registration. Nivolumab for Non-squamous was started 6 months before approval, while for Squamous the label was approved by MOH 2 months after starting the compassionate program. Reimbursement approval was received 6 months thereafter for nivolumab (Squamous NSCLC). Two out of the three drugs in the program were approved for reimbursement, one of them even before MOH registration.
January 2017
Abstracts
S1435
we are handling advanced and metastatic adenocarcinomas in NSCLC patients.
Conclusion: Compassionate use programs allow access to new cancer drugs prior to their approval by the regulatory authorities, increases physicians’ experience with novel compounds and may affect reimbursement approval.
Results: We found 182 patients with NSCLC (adenocarcinoma), from those 72 was in stage IIIB-IV or with relapsed disease. In 37/72 (51,38%) cases some molecular testing was done (EGFR, ALK); EGFR 26 (36,1%) WT: 9/26 (34,6%), mut: 9/26 (34.6%)(in confirmation), UK (data in confirmation): 8/26 (30,7%); ALK: 10 (13,8%), w-o transloc.: 6/10 (60%); wtransloc: 1/10 (10%); UK (data in confirmation)3/10 (30%). From 72 patients, nine (12,5%) was included in clinical trials; 6/ 72 (8.33%) received TKI out of a trial. From there just 2/ 6 received TKI as first line, and 4/6 as a second line and beyond. Conclusion: It will be done after our sample, and assay tests results review. Keywords: EGFR, ALK, Public Health Service, Metastatic NSCLC
Keywords: Compassionate use program, osimertinib, lung cancer, Nivolumab
P3.07-008 METASTATIC NSCLC: Treatment Reality from 182 Cases of Lung Adenocarcinoma in a Brazilian Public Cancer Health Service Topic: Therapy and Economics Leonardo Lago,1 Bruna Da Silva,2 Gabriel Lenz,2 Fernanda Bruzzo,2 Vinicius Da Silva3 1Oncology, Hospital Sao Lucas Da Pucrs, Porto Alegre/Brazil, 2 Faculdade de Medicina Da Pucrs, Porto Alegre/Brazil, 3 Professor of Pathology, Faculdade de Medicina Da Pucrs, Porto Alegre/Brazil Background: In Brazil, lung cancer is a public health problem as in the world. The national Incidence in 2016 is estimated in 28.220 new cases.1 In Rio Grande do Sul, an etimated number of new cases is 4.240.1 Is well established molecular testing for EGFR, ALK mutations are mandatory to treat metastatic adenocarcinomas to indicate target therapy, or not. Prevalence of EGFR mutations in adenocarcinomas, for example, are estimated from 25,5% to 30,4%.2,3 Although many guidelines advocates to use a TKI as first line therapy, in Brazil is difficult because nor the drugs, nor the assays are available to public health service, except by legal measures,4 or support from pharmaceutical industry Methods: We made a cross-sectional study from January 2013 to May 2016, collecting data from medical records in our institution, to evaluate our population and how
P3.07-009 Use of Adjuvant Chemotherapy for NonSmall Cell Lung Cancer: The Real-World Clinical Practice in Taiwan Topic: Therapy and Economics Bin-Chi Liao,1 Yu-Yun Shao,1 James Chih-Hsin Yang,1 Ho-Min Chen,1 Ann-Lii Cheng,1 Mei-Shu Lai2 1 Department of Oncology, National Taiwan University Hospital, Taipei/Taiwan, 2Institute of Epidemiology and Preventive Medicine, College of Public Health, National Taiwan University, Taipei/Taiwan Background: Adjuvant chemotherapy is the standard treatment for selected patients with non-small cell lung cancer (NSCLC) following curative surgery. This study evaluated the use of adjuvant chemotherapy for these cases among the general population in Taiwan. Methods: A population-based cohort was established by searching the database of the Taiwan Cancer Registry System to identify patients newly diagnosed with NSCLC for the period covering 2005 to 2009. Our target was patients with stage I, II and IIIA NSCLC who had undergone curative surgery. Medication prescription data were retrieved from the National Health Insurance Research Database, Taiwan. Chemotherapy administered within 3 months after the surgery was defined as adjuvant chemotherapy. Results: A total of 5789 patients received curative surgery for NSCLC, and the 1277 (22.1%) who had undergone adjuvant chemotherapy were included in this study. Overall, the most common adjuvant chemotherapy