PP452 Impact Of Patient Access Schemes On Health Technology Assessment Agency Guidance For Rare Diseases In England and Scotland

2021 ◽  
Vol 37 (S1) ◽  
pp. 38-38
Author(s):  
Karen Mark ◽  
Prachi Manchanda ◽  
Judith Rubinstein ◽  
Riza Veronica Inumerable
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Incremental Cost ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Patient Access ◽  
Cost Effectiveness Ratio

IntroductionPatient access schemes (PAS) are agreements that may enable patients to access drugs or other treatments that may not be cost effective under normal circumstances. The aim of this study was to determine whether the use of PAS by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for recommended drugs can lead to greater access to medications for rare diseases.MethodsReimbursement data for rare diseases between 2004 and 2021 from health technology assessment (HTA) agencies, namely the SMC (Scotland) and NICE (England), were included. The reviews with positive HTA decisions were considered, while those with negative decisions were excluded. Several observations were made from these data and reported.ResultsAmong the total positive reviews (n = 81), 43 included PAS. The inclusion of PAS in manufacturer submissions was more frequent for NICE than for the SMC (79% and 40% percent, respectively). Most of the drugs with PAS were included in the HTA guidance from both agencies. The positive NICE reviews contingent on PAS consisted of 20 drugs. For the same set of drugs, the SMC recommended 14 with PAS and one without PAS; five drugs were not assessed. Adalimumab was recommended by NICE with a PAS (base-case incremental cost-effectiveness ratio of GBP12,336 [EUR14,256]; GBP13,676 [EUR15,804]) and by the SMC without a PAS (base-case incremental cost-effectiveness ratio of GBP22,519 [EUR26,023]). Hence, without a PAS, the drug was costlier per quality-adjusted life-year for the National Health Service (NHS) Scotland.ConclusionsPAS submissions for rare diseases are more frequent for NICE than for the SMC. With the PAS discounts, the overall cost of the drugs is reduced, resulting in cost effectiveness. The SMC approved some drugs for which NICE required a PAS to improve the economic argument. Hence, the use of PAS for these drugs could lead to potential cost-savings to the NHS Scotland.

scholarly journals Conventional Computed Tomographic Calcium Scoring vs Full Chest CTCS for Lung Cancer Screening: A Cost-Effectiveness Analysis

2019 ◽  
Author(s):  
Boxiang Jiang ◽  
Philip A. Linden ◽  
Amit Gupta ◽  
Craig Jarrett ◽  
Stephanie G. Worrell ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cancer Screening ◽  
Literature Review ◽  
Incremental Cost ◽  
Pulmonary Nodules ◽  
Lung Cancer Screening ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Lung Field ◽  
Cost Effectiveness Ratio

Abstract Background Conventional CTCS images the mid/lower chest for coronary artery disease (CAD). Because many CAD patients are also at risk for lung malignancy, CTCS often discovers incidental pulmonary nodules (IPN). CTCS excludes the upper chest, where malignancy is common. Full-chest CTCS (FCT) may be a cost-effective screening tool for IPN. Methods A decision tree was created to compare a FCT to CTCS in a hypothetical patient cohort with suspected CAD. (Figure) The design compares the effects of missed cancers on CTCS with the cost of working up non-malignant nodules on FCT. The model was informed by results of the National Lung Screening Trial and literature review, including the rate of malignancy among patients receiving CTCS and the rate of malignancy in upper vs lower portions of the lung. The analysis outcomes are Quality-Adjusted Life Year (QALY) and incremental cost-effectiveness ratio (ICER), which is generally considered beneficial when <$50,000/QALY. Results Literature review suggests that rate of IPNs in the upper portion of the lung varied from 47-76%. Our model assumed that IPNs occur in upper and lower portions of the lung with equal frequency. The model also assumes an equal malignancy potential in upper lung IPNs despite data that malignancy occurs 61-66% in upper lung fields. In the base case analysis, a FCT will lead to an increase of 0.03 QALYs comparing to conventional CTCS (14.54 vs 14.51 QALY, respectively), which translates into an QALY increase of 16 days. The associated incremental cost for FCT is $278 ($1,027 vs $748, FCT vs CTCS respectively. The incremental cost-effectiveness ratio (ICER) is $10,289/QALY, suggesting significant benefit. Sensitivity analysis shows this benefit increases proportional to the rate of malignancy in upper lung fields. Conclusion Conventional CTCS may be a missed opportunity to screen for upper lung field cancers in high risk patients. The ICER of FCT is better than screening for breast cancer screening (mammograms $80k/QALY) and colon cancer (colonoscopy $6k/QALY). Prospective studies are appropriate to define protocols for FCT.

scholarly journals Cost-effectiveness analysis with direct-acting antivirals in a cohort of HCV-infected inmates in Italy

ABOUTOPEN ◽  
2020 ◽  
Vol 7 (1) ◽  
pp. 103-111
Author(s):  
Roberto Ravasio ◽  
Luciano Lucania ◽  
Roberto Ranieri ◽  
Raquel Dominguez
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Medical Costs ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Direct Acting Antivirals ◽  
Cost Effectiveness Ratio ◽  
Direct Medical Costs ◽  
Direct Acting

Objective: To evaluate the cost-effectiveness of a strategy based on direct-acting antivirals (DAAs) versus no drug strategy in HCV-infected inmates, from the perspective of the Italian National Health System (iNHS). Methods: A previous Markov model was adapted to the Italian setting to evaluate the direct medical costs and health outcomes (quality-adjusted life years, QALY) throughout the life of HCV-infected inmates. Epidemiological data, patient characteristics (genotype, METAVIR classification), DAAs sustained virological response (SVR), annual likelihood of transition, treatment costs and utilities were gathered from the literature. The DAAs strategy included the administration of elbasvir/grazoprevir or sofosbuvir/velpatasvir or glecaprevir/pibrentasvir. Direct medical costs and QALYs were discounted at a 3.0% annual rate. Cost-effectiveness was evaluated as incremental cost-effectiveness ratio (€, 2019) per QALY gained. A deterministic sensitivity analysis (DSA) was performed. Results: Over a lifetime horizon, the DAAs strategy showed higher health costs per patient compared to no drugs strategy in the base-case analysis (€ 42,571 vs. € 26,119). However, it was associated with an increase of QALYs gained (21.14 vs. 15.67), showing an incremental cost-effectiveness ratio of € 3,010 per QALY. The sensitivity analysis confirmed the base-case results. Conclusions: Extending the DAAs treatment to HCV-infected inmates was estimated to be cost effective from the perspective of the Italian NHS, regardless genotype and METAVIR classification.

scholarly journals Cetuximab in first line treatment of metastatic colorectal cancer

2012 ◽  
Vol 13 (1S) ◽  
pp. 1-80
Author(s):  
Carlo Barone ◽  
Andrea Belisari ◽  
Fortunato Ciardiello ◽  
Lorenzo Mantovani ◽  
Lucia D’Angiolella ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Incremental Cost ◽  
Technology Assessment ◽  
Health Technology ◽  
Line Treatment ◽  
First Line ◽  
Kras Status ◽  
First Line Treatment

The present health technology assessment report evaluates the clinical and economic profile of cetuximab in first-line metastatic colorectal cancer (mCRC) in Italy. The first part of the report addresses the epidemiological, clinical, social and economic impact of mCRC. In the second part, evidence of efficacy, safety and cost-effectiveness of cetuximab and its available alternatives is shown. Finally, a model-based economic evaluation aimed at comparing cetuximab-based re­gimens vs. alternative therapeutic strategies indicated in mCRC in Italy is presented. The model estimates the incremental cost-effectiveness of adding cetuximab to FOLFOX-4 or FOLFIRI based on KRAS status, vs. adding bevacizumab to FOLFOX-4 or vs. FOLFOX-4 or FOLFIRI alone. A theoretical analysis vs. panitumumab has also been performed, despite panitumumab is not yet reimbursed in Italy in first-line mCRC. Survival outcomes, quality of life and costs of patient ma­nagement are estimated through a Markov model, using the Italian National Healthcare Service (NHS) perspective, over a 10 year period, taking into account KRAS status of patients. The results of the pharmaco-economic analysis show that cetuximab + FOLFOX-4 and cetuximab + FOLFIRI are associated with increased survival, increased cost and increased quality adjusted survival, compared to all other treatments currently indicated and reimbursed in Italy. Adding cetuximab to FOLFOX-4 or FOLFIRI, based on KRAS status shows favorable incremental cost-effectiveness ratio (ICER) vs. adding bevacizumab to FOLFIRI or vs. FOLFOX-4 or FOLFIRI alone. ICER of cetuximab (in combination with FOLFOX-4 or FOLFIRI), compared to currently reimbursed alternatives, is estimated between 6 and 13 thousand Euros per QALY gai­ned, depending on alternative treatment. These estimates are robust in extensive sensitivity analyses. As a final result, both clinical and economic evidence analyzed in this health technology assessment leads to recommend cetuximab as preferred option for the first-line treatment of mCRC KRAS wild-type patients in Italy.

scholarly journals Cost-effectiveness analysis with direct-acting antivirals in a cohort of HCV-infected inmates in Italy

ABOUTOPEN ◽  
2020 ◽  
Vol 7 (1) ◽  
pp. 103-111
Author(s):  
Roberto Ravasio ◽  
Luciano Lucania ◽  
Roberto Ranieri ◽  
Raquel Dominguez
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Medical Costs ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Direct Acting Antivirals ◽  
Cost Effectiveness Ratio ◽  
Direct Medical Costs ◽  
Direct Acting

Objective: To evaluate the cost-effectiveness of a strategy based on direct-acting antivirals (DAAs) versus no drug strategy in HCV-infected inmates, from the perspective of the Italian National Health System (iNHS). Methods: A previous Markov model was adapted to the Italian setting to evaluate the direct medical costs and health outcomes (quality-adjusted life years, QALY) throughout the life of HCV-infected inmates. Epidemiological data, patient characteristics (genotype, METAVIR classification), DAAs sustained virological response (SVR), annual likelihood of transition, treatment costs and utilities were gathered from the literature. The DAAs strategy included the administration of elbasvir/grazoprevir or sofosbuvir/velpatasvir or glecaprevir/pibrentasvir. Direct medical costs and QALYs were discounted at a 3.0% annual rate. Cost-effectiveness was evaluated as incremental cost-effectiveness ratio (€, 2019) per QALY gained. A deterministic sensitivity analysis (DSA) was performed. Results: Over a lifetime horizon, the DAAs strategy showed higher health costs per patient compared to no drugs strategy in the base-case analysis (€ 42,571 vs. € 26,119). However, it was associated with an increase of QALYs gained (21.14 vs. 15.67), showing an incremental cost-effectiveness ratio of € 3,010 per QALY. The sensitivity analysis confirmed the base-case results. Conclusions: Extending the DAAs treatment to HCV-infected inmates was estimated to be cost effective from the perspective of the Italian NHS, regardless genotype and METAVIR classification.

scholarly journals Conventional Computed Tomographic Calcium Scoring vs Full Chest CTCS for Lung Cancer Screening: A Cost-Effectiveness Analysis

2019 ◽  
Author(s):  
Boxiang Jiang ◽  
Philip A. Linden ◽  
Craig Jarrett ◽  
Stephanie G. Worrell ◽  
Vanessa P. Ho ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cancer Screening ◽  
Literature Review ◽  
Incremental Cost ◽  
Pulmonary Nodules ◽  
Lung Cancer Screening ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Lung Field ◽  
Cost Effectiveness Ratio

Abstract Background Conventional CTCS images the mid/lower chest for coronary artery disease (CAD). Because many CAD patients are also at risk for lung malignancy, CTCS often discovers incidental pulmonary nodules (IPN). CTCS excludes the upper chest, where malignancy is common. Full-chest CTCS (FCT) may be a cost-effective screening tool for IPN. Methods A decision tree was created to compare a FCT to CTCS in a hypothetical patient cohort with suspected CAD. (Figure) The design compares the effects of missed cancers on CTCS with the cost of working up non-malignant nodules on FCT. The model was informed by results of the National Lung Screening Trial and literature review, including the rate of malignancy among patients receiving CTCS and the rate of malignancy in upper vs lower portions of the lung. The analysis outcomes are Quality-Adjusted Life Year (QALY) and incremental cost-effectiveness ratio (ICER), which is generally considered beneficial when <$50,000/QALY. Results Literature review suggests that rate of IPNs in the upper portion of the lung varied from 47-76%. Our model assumed that IPNs occur in upper and lower portions of the lung with equal frequency. The model also assumes an equal malignancy potential in upper lung IPNs despite data that malignancy occurs 61-66% in upper lung fields. In the base case analysis, a FCT will lead to an increase of 0.03 QALYs comparing to conventional CTCS (14.54 vs 14.51 QALY, respectively), which translates into an QALY increase of 16 days. The associated incremental cost for FCT is $278 ($1,027 vs $748, FCT vs CTCS respectively. The incremental cost-effectiveness ratio (ICER) is $10,289/QALY, suggesting significant benefit. Sensitivity analysis shows this benefit increases proportional to the rate of malignancy in upper lung fields. Conclusion Conventional CTCS may be a missed opportunity to screen for upper lung field cancers in high risk patients. The ICER of FCT is better than screening for breast cancer screening (mammograms $80k/QALY) and colon cancer (colonoscopy $6k/QALY). Prospective studies are appropriate to define protocols for FCT.

PP355 Evolution Of Health Technology Assessment For Rare Diseases In Asia

2020 ◽  
Vol 36 (S1) ◽  
pp. 30-31
Author(s):  
Tarveen Jandoo ◽  
Baris Deniz ◽  
Shuo Yang
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Risk Sharing ◽  
National Policy ◽  
Health Technology ◽  
A Value ◽  
Access And Affordability ◽  
Improve Access

IntroductionWe reviewed the health technology assessment (HTA) guidelines for therapies targeting orphan conditions in four countries/regions in Asia.MethodsA pragmatic literature search was conducted to identify and review key documents outlining reimbursement, pricing, and coverage policies in China, Taiwan, Korea, and Japan.ResultsTherapies for rare diseases in Japan and those for ultra-rare diseases in Korea are exempt from cost-effectiveness evaluations. Taiwan provides full financial coverage for rare disease therapies. China has no special considerations for rare diseases. Drugs included in the medical insurance list are reimbursed at varying levels depending on the “class” of the listing. Unlike prior variations at provincial levels for coverage of off-the-list drugs, new national policy has introduced consistency in coverage.ConclusionsAccess and reimbursement processes vary between markets in Asia. New HTA guidelines in Japan allow for easier access to therapies targeting rare diseases by eliminating cost-effectiveness analysis for price determination. On the other hand, a value dossier including an economic evaluation is necessary for rare diseases in Korea. However, manufacturers can provide risk-sharing schemes for rare diseases. China has not yet introduced any specific evaluations or reimbursement criteria for therapies targeting rare diseases. Policies for rare diseases are evolving rapidly to improve access and affordability.

scholarly journals Conventional Computed Tomographic Calcium Scoring vs Full Chest CTCS for Lung Cancer Screening: A Cost-Effectiveness Analysis

2019 ◽  
Author(s):  
Boxiang Jiang ◽  
Philip A. Linden ◽  
Amit Gupta ◽  
Craig Jarrett ◽  
Stephanie G. Worrell ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cancer Screening ◽  
Literature Review ◽  
Incremental Cost ◽  
Pulmonary Nodules ◽  
Lung Cancer Screening ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Lung Field ◽  
Cost Effectiveness Ratio

Abstract Background Conventional CTCS images the mid/lower chest for coronary artery disease (CAD). Because many CAD patients are also at risk for lung malignancy, CTCS often discovers incidental pulmonary nodules (IPN). CTCS excludes the upper chest, where malignancy is common. Full-chest CTCS (FCT) may be a cost-effective screening tool for IPN. Methods A decision tree was created to compare a FCT to CTCS in a hypothetical patient cohort with suspected CAD. (Figure) The design compares the effects of missed cancers on CTCS with the cost of working up non-malignant nodules on FCT. The model was informed by results of the National Lung Screening Trial and literature review, including the rate of malignancy among patients receiving CTCS and the rate of malignancy in upper vs lower portions of the lung. The analysis outcomes are Quality-Adjusted Life Year (QALY) and incremental cost-effectiveness ratio (ICER), which is generally considered beneficial when <$50,000/QALY. Results Literature review suggests that rate of IPNs in the upper portion of the lung varied from 47-76%. Our model assumed that IPNs occur in upper and lower portions of the lung with equal frequency. The model also assumes an equal malignancy potential in upper lung IPNs despite data that malignancy occurs 61-66% in upper lung fields. In the base case analysis, a FCT will lead to an increase of 0.03 QALYs comparing to conventional CTCS (14.54 vs 14.51 QALY, respectively), which translates into an QALY increase of 16 days. The associated incremental cost for FCT is $278 ($1,027 vs $748, FCT vs CTCS respectively. The incremental cost-effectiveness ratio (ICER) is $10,289/QALY, suggesting significant benefit. Sensitivity analysis shows this benefit increases proportional to the rate of malignancy in upper lung fields. Conclusion Conventional CTCS may be a missed opportunity to screen for upper lung field cancers in high risk patients. The ICER of FCT is better than screening for breast cancer screening (mammograms $80k/QALY) and colon cancer (colonoscopy $6k/QALY). Prospective studies are appropriate to define protocols for FCT.

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