scholarly journals Examining the association between oncology drug clinical benefit and the time to public reimbursement

2021 ◽  
Author(s):  
Sasha Thomson ◽  
Louis Everest ◽  
Noah Witzke ◽  
Tina Jiao ◽  
Seanthel Delos Santos ◽  
...  
Keyword(s):  
Clinical Benefit ◽  
Oncology Drug

Do the American Society of Clinical Oncology (ASCO) Value Framework and the European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale measure the same construct of clinical benefit?

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 6509-6509 ◽  
Author(s):  
Sierra Cheng ◽  
Erica McDonald ◽  
Matthew C. Cheung ◽  
Vanessa Sarah Arciero ◽  
Mahin Iqbal Qureshi ◽  
...  
Keyword(s):  
Clinical Oncology ◽  
European Society ◽  
Clinical Benefit ◽  
Randomized Clinical Trials ◽  
Medical Oncology ◽  
Correlation Coefficients ◽  
Rater Reliability ◽  
Oncology Drug ◽  
Life Years ◽  
American Society

6509 Background: Whether the American Society of Clinical Oncology (ASCO) Value Framework and the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) measure similar constructs of clinical benefit is unclear. It is also unclear how they relate to quality-adjusted life-years (QALYs) and funding recommendations in the UK and Canada. Methods: Randomized clinical trials (RCTs) of oncology drug approvals by the Food and Drug Administration, European Medicines Agency and Health Canada between January 2006 and August 2015 were identified and scored using the ASCO version 1 (v1) framework (August 10, 2015), ASCO version 2 (v2) framework (May 31, 2016) and ESMO-MCBS (May 30, 2015) by at least two independent reviewers. Spearman correlation coefficients were calculated to assess construct (between frameworks) and criterion validity (against incremental QALYs from the National Institute of Clinical Excellence (NICE) and the pan-Canadian Oncology Drug Review (pCODR)). Associations between scores and NICE/pCODR recommendations were examined by logistic regression models. Inter-rater reliability was assessed using intra-class correlation coefficients. Results: From 109 included RCTs, 108 ASCOv1, 111 ASCOv2 and 83 ESMO scores were determined. Correlation coefficients for ASCOv1 vs. ESMO, ASCOv2 vs. ESMO, and ASCOv1 vs. ASCOv2 were 0.36 (95% CI 0.15-0.54), 0.17 (95% CI -0.06-0.37) and 0.50 (95% CI 0.35-0.63), respectively. Compared with NICE QALYs, correlation coefficients were 0.45 (ASCOv1), 0.53 (ASCOv2) and 0.46 (ESMO); with pCODR QALYs, coefficients were 0.19 (ASCOv1), 0.20 (ASCOv2) and 0.36 (ESMO). None of the frameworks were significantly associated with NICE/pCODR recommendations. Inter-rater reliability was good for all frameworks. Conclusions: The weak-to-moderate correlations between the ASCO frameworks and ESMO-MCBS, with QALYs, and with NICE/pCODR funding recommendations suggest different constructs of clinical benefit measured. Construct convergent validity with the ESMO-MCBS in fact did not increase with the updated ASCO framework.

Use of single-arm trials to support malignant hematology and oncology drug and biologic approvals: A 20-year FDA experience.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e13572-e13572
Author(s):  
Sundeep Agrawal ◽  
Shaily Arora ◽  
Jonathon Joseph Vallejo ◽  
Thomas Gwise ◽  
Meredith Kathleen Chuk ◽  
...  
Keyword(s):  
Drug Development ◽  
Clinical Benefit ◽  
Randomized Clinical Trials ◽  
Kinase Inhibitors ◽  
Response Rate ◽  
Checkpoint Inhibitors ◽  
Unmet Need ◽  
Cancer Drug ◽  
Cancer Therapies ◽  
Oncology Drug

e13572 Background: Improved understanding of the underlying biology of cancer has led to a paradigm shift in cancer drug development and has paved the way for many products to receive accelerated or regular approval based on non-randomized/single arm trials (SATs). Given the high unmet medical need of cancer patients, challenges with lengthy and confounded survival endpoints, and difficulty enrolling rare biomarker-defined subsets of disease, SATs have been used to evaluate a variety of cancer therapies. Unlike time to event endpoints, the objective and clinically relevant endpoint of response rate (RR) and duration of response is interpretable in SATs, as spontaneous tumor shrinkage is not expected. Methods: A search of FDA databases identified all drugs and biologics approved for malignant hematology and oncology indications from January 1, 2001, to December 31, 2020 based on SATs. Data sources included approval letters, U.S. prescribing information, and clinical review documents. The definition of response varied by setting and time period (e.g. RECIST, WHO, IWG, etc.). Results: Between January 1, 2001 and December 31, 2020, FDA granted 153 new indications based on SATs, including 102 accelerated approvals (AAs) and 51 regular approvals (RAs). Overall, 69 approvals (45%) were for new molecular entities and 84 (55%) were expanded indications. Response rate was the most common endpoint used in the trial providing substantial evidence of efficacy to support approval [120/153, (78%)]. The durability of response was also considered to support evidence of clinical benefit. Of the 102 AAs, 38 (37%) have fulfilled their post-marketing requirement (PMR) to verify clinical benefit, 59 (58%) are pending verification of benefit, and 5 (5%) have been withdrawn from the market. Of note, 88% (52/59) of AAs pending verification of benefit occurred in the last 5 years alone (22 AAs in 2020, 8 in 2019, 8 in 2018, 12 in 2017, and 2 in 2016). Between 2001-2020, 58 (38%) new indications were granted for kinase inhibitors, 34 (22%) for immune checkpoint inhibitors (CPIs), and 61 (40%) for drugs with other mechanisms of action including but not limited to antibody-drug conjugates, cytotoxic drugs, and non-CPI monoclonal antibodies. Conclusions: In the last two decades, SATs have been effectively used to study anti-cancer therapies in well-defined patient populations using durable RR as an objective and interpretable clinical endpoint. Although randomized clinical trials remain the gold standard in clinical research, SATs have allowed for rapid advancements in oncology drug development and will continue to serve an important role in bringing new therapies to patients with unmet need.

Do the American Society of Clinical Oncology Value Framework and the European Society of Medical Oncology Magnitude of Clinical Benefit Scale Measure the Same Construct of Clinical Benefit?

2017 ◽  
Vol 35 (24) ◽  
pp. 2764-2771 ◽  
Author(s):  
Sierra Cheng ◽  
Erica J. McDonald ◽  
Matthew C. Cheung ◽  
Vanessa S. Arciero ◽  
Mahin Qureshi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
European Society ◽  
Clinical Benefit ◽  
Randomized Clinical Trials ◽  
Medical Oncology ◽  
Intraclass Correlation ◽  
Correlation Coefficients ◽  
Rater Reliability ◽  
Oncology Drug ◽  
Life Years

Purpose Whether the ASCO Value Framework and the European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale (MCBS) measure similar constructs of clinical benefit is unclear. It is also unclear how they relate to quality-adjusted life-years (QALYs) and funding recommendations in the United Kingdom and Canada. Methods Randomized clinical trials of oncology drug approvals by the US Food and Drug Administration, European Medicines Agency, and Health Canada between 2006 and August 2015 were identified and scored using the ASCO version 1 (v1) framework, ASCO version 2 (v2) framework, and ESMO-MCBS by at least two independent reviewers. Spearman correlation coefficients were calculated to assess construct (between frameworks) and criterion validity (against QALYs from the National Institute for Health and Care Excellence [NICE] and the pan-Canadian Oncology Drug Review [pCODR]). Associations between scores and NICE/pCODR recommendations were examined. Inter-rater reliability was assessed using intraclass correlation coefficients. Results From 109 included randomized clinical trials, 108 ASCOv1, 111 ASCOv2, and 83 ESMO scores were determined. Correlation coefficients for ASCOv1 versus ESMO, ASCOv2 versus ESMO, and ASCOv1 versus ASCOv2 were 0.36 (95% CI, 0.15 to 0.54), 0.17 (95% CI, −0.06 to 0.37), and 0.50 (95% CI, 0.35 to 0.63), respectively. Compared with NICE QALYs, correlation coefficients were 0.45 (ASCOv1), 0.53 (ASCOv2), and 0.46 (ESMO); with pCODR QALYs, coefficients were 0.19 (ASCOv1), 0.20 (ASCOv2), and 0.36 (ESMO). None of the frameworks were significantly associated with NICE/pCODR recommendations. Inter-rater reliability was good for all frameworks. Conclusion The weak-to-moderate correlations of the ASCO frameworks with the ESMO-MCBS, as well as their correlations with QALYs and with NICE/pCODR funding recommendations, suggest different constructs of clinical benefit measured. Construct convergent validity with the ESMO-MCBS did not increase with the updated ASCO framework.

Examining the relationship between the clinical benefit of oncology drug indications and the time from pan-Canadian Oncology Drug Review (pCODR) recommendation to public reimbursement.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19360-e19360
Author(s):  
Sasha Thomson ◽  
Louis Everest ◽  
Noah Witzke ◽  
Seanthel Delos Santos ◽  
Matthew C. Cheung ◽  
...  
Keyword(s):  
Clinical Benefit ◽  
Multivariable Analysis ◽  
Cancer Drug ◽  
Cancer Type ◽  
Inclusion Criteria ◽  
Oncology Drug ◽  
The Mean ◽  
American Society ◽  
The Relationship ◽  
Drug Review

e19360 Background: We examined if publicly reimbursed oncology drug indications with evidence of high clinical benefit, as measured by the American Society of Clinical Oncology Value Framework v2 (ASCO-VF), and European Society for Medical Oncology Magnitude of Clinical Benefit Scale v1.1 (ESMO-MCBS), received reimbursement status faster than those with lower clinical benefit from the time of pCODR recommendation. Methods: Oncology drug indications that received pCODR recommendations between Jan 2012 and July 2018 were identified. Indications that did not receive provincial reimbursement, without notice of compliance, or received a negative pCODR recommendation were excluded. The relationship between clinical benefit, as measured by ASCO-VF and ESMO-MCBS, and the time to reimbursement was evaluated using Spearman correlation coefficient, univariable, and multivariable linear regression analyses. Results: Overall, 84 indications met inclusion criteria yielding 80 ASCO-VF and 66 ESMO-MCBS scores. The mean ASCO-VF and ESMO-MCBS scores were 38.8 (SD = 23.8) and 3.0 (SD = 1.1) respectively. Higher ASCO-VF and ESMO-MCBS scores had low correlation with shorter time to provincial funding, (rho = -0.15, 95%CI -0.24, -0.06) and (rho = -0.25, 95%CI -0.34, -0.16) respectively. Univariable analyses showed that manufacturer reported incremental cost effectiveness ratio (ICER) values, year of pCODR recommendation, province and cancer type were associated with time to public reimbursement (all p < 0.0001). After adjusting for potential confounders in the respective multivariable analysis, ASCO-VF (p = 0.29) and ESMO-MCBS (p = 0.15) scores were not significantly associated with time to public reimbursement. Year of pCODR recommendation remained associated with time to public reimbursement (p < 0.001). Earlier years (2012-2014) had a shorter time to reimbursement (mean = 10.4 months) than later years (2015-2018) (mean = 14.5 months). Other factors that were associated with time to reimbursement in multivariable analysis were province (p < 0.001) and cancer type (p < 0.001). Conclusions: Currently, oncology drug indication with evidence of high clinical benefit do not appear to be funded faster than those with low clinical benefit. This suggests the need to prioritize cancer drug indications based on clinical benefit in order to allow for timely public reimbursement of cancer drugs with higher clinical benefit to patients.

VP02 Real-World Evidence (RWE) And CADTH Pan-Canadian Oncology Drug Review

2018 ◽  
Vol 34 (S1) ◽  
pp. 159-160
Author(s):  
Dolly Han ◽  
Missale Tiruneh ◽  
Alexandra Chambers ◽  
Adam Haynes
Keyword(s):  
Cost Effectiveness ◽  
Data Collection ◽  
Real World ◽  
Clinical Benefit ◽  
Cancer Drug ◽  
Duration Of Treatment ◽  
Expert Review ◽  
Oncology Drug ◽  
Real World Evidence ◽  
Drug Review

Introduction:The pan-Canadian Oncology Drug Review (pCODR) program was established by Canada's provincial and territorial Ministries of Health (except Quebec) to assess cancer drug therapies and make recommendations to guide drug reimbursement decisions. The pCODR Expert Review Committee (pERC) makes reimbursement recommendations, providing a rationale for the recommendation and next steps for stakeholders. The objective of this analysis was to identify reviews and reasons pERC has requested real-world evidence (RWE) data collection.Methods:A retrospective analysis of pERC Final Recommendations (January 2012 – May 2017) was conducted. pERC Final Recommendations include drug information, reimbursement recommendation, rationale for recommendation following pERC's Deliberative Framework (clinical benefit, patient-based values, economic evaluation, and adoption feasibility), next steps for jurisdictions to consider to support their funding decisions, summary of deliberations, and evidence in brief. Reviews were included if there was a next step advising the collection of RWE to reduce uncertainty in the drug under review.Results:Out of eighty-four reviews, forty-one (forty-eight percent) included a next step to collect RWE to address a gap in the available evidence. Reasons for RWE data collection, in descending order of frequency, were to inform: sequencing of available therapies; magnitude of clinical benefit and cost-effectiveness or the true cost-effectiveness; duration of treatment and cost-effectiveness; defining the population or disease progression; quality of life; and dosage.Conclusions:In almost half of pERC's recommendation there is an indication that there is a gap in the existing evidence that could potentially be addressed through the collection of RWE. This reflects the rising number of new cancer drugs, limited evidence supporting submissions (for example non-comparative studies), and newer drugs such as immunotherapies which may not have a fixed treatment duration. Further research includes development of mechanisms for RWE data collection to help inform pERC recommendations and assist stakeholders with adoption feasibility of reviewed drugs.

918: Evaluation of the Clinical Benefit of Permixon and Tamsulosin in Severe BPH Patients – Permal Study Sub-Set Analysis

2004 ◽  
Vol 171 (4S) ◽  
pp. 243-244 ◽  
Author(s):  
Frans Debruyne ◽  
Peter Boyle ◽  
Remigio Vela-Navarrete ◽  
Fernando Calais Da Silva ◽  
Pierre Teillac ◽  
...  
Keyword(s):  
Clinical Benefit

Erythropoietin for HCV May Lack Clinical Benefit

2008 ◽  
Vol 41 (9) ◽  
pp. 52
Author(s):  
MICHELE G. SULLIVAN
Keyword(s):  
Clinical Benefit

Traditional and Enhanced Parent Training Show Similar Levels of Clinical Benefit in Single Mothers

2015 ◽  
Author(s):  
E. Rajwan ◽  
A. Chacko ◽  
B.T. Wymbs ◽  
F.A. Wymbs
Keyword(s):  
Parent Training ◽  
Single Mothers ◽  
Clinical Benefit
Sign in / Sign up

Export Citation Format

Share Document