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Are Criteria Commonly Used in the Benefit Assessment of Drugs also Applicable to Medical Devices? - A Systematic Literature Review
A706
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 3 4 7 – A 7 6 6
diagnostic odyssey. Complete resource use data were collected at the University Medical Center Utrecht (UMCU), Utrecht, the Netherlands. We categorized the young patients according to their WES-based diagnosis (yes, no, and uncertain), and assessed the per-patient healthcare activities and corresponding costs before (PRE) and after (POST) the diagnosis was obtained. Results: In the cohort of 371 patients, 129 patients received a genetic diagnosis (35%). In 89 (24%) patients no mutation was identified and 153 patients (41%) had an uncertain (variant-ofunknown-significance) diagnosis. We estimated implementation of WES-first would result in mean savings of € 5122 euro per patient resulting from replacement of all other genetic technologies and 50% of non-genetic diagnostic tools and consults. A decrease in costs was observed in the majority of categories POST-WES, only the costs of consults in all categories and the costs of genetic tests after not having a diagnosis (+300%) increased. Conclusions: Our study confirms that WES is a cost-effective replacement of traditional diagnostic technologies for patients with ID. Looking at the high, although similar in all WES-based diagnostic groups, costs WES could be a cost-efficient and unique option when implemented early in patient care: it could reduce health care costs and induce the amount of distinct diagnoses. On the other hand, our data demonstrate that for patients not having a diagnosis the diagnostic costs have increased after WES. This indicates a change in mindset is needed when implementing WES on a broad scale. PMD119 Substitutability of Dry Powder Inhalers: A Perspective from German Pulmonologists Redeker M1, Schwarz E1, Mohrlang C1, Hellmann A2, Hellmann A3, Hering T3, Hering T4, Andres J5, Andres J3 1GlaxoSmithKline GmbH & Co. KG, München, Germany, 2Zentrum für Pneumologie, Onkologie und Schlafmedizin am Diako, Augsburg, Augsburg, Germany, 3Wissenschaftliches Netzwerk des Bundesverbandes der Pneumologen/BdP (WinPneu), Heidenheim, Germany, 4Lungenarztpraxis Tegel, Berlin, Germany, 5MedWiss GmbH, Weinitzen, Germany
Objectives: Inhaled therapies can be substituted in German pharmacies unless prohibited through an “aut-idem-cross” by the physician. Substitution can lead to patients receiving unfamiliar inhalers resulting in problems with the application. This research aims to gain insights regarding German pulmonologists’ perspective and experience on substitutability including how often inhalers are the cause for therapy change. Methods: 90% of all German pulmonologists are affiliated to the Federal Association of Pulmonologists. All 850 were asked to participate in the survey with 304 responding. Questions involved a four- or five-point scale. The list of reasons for therapy changes is based on literature, physician- and expert-opinion. Results: 97% of all pulmonologists see patient individual selection of inhalers as “very important”. 76% report prohibiting substitution “very often” and “often”. However, 34% of pulmonologists estimate that only 9% of general practitioners use aut-idem-cross “very often” and “often”. 67% estimate that 67% of general practitioners prohibit substitution “rarely” and “very rarely”. Pulmonologists often take the therapeutical decision for medicine and inhaler but general practitioners often make follow-up prescriptions. 39% of pulmonologists report that patients visit them “very often” and “often” because they did not receive the prescribed medication due to substitution in the pharmacy. Pulmonologists report that patients had problems in applying their substituted inhaler (43% “sometimes”, 41% “often”, 11% “very often”). Pulmonologists report making on average twelve therapy changes biweekly. Most common reason for therapy changes are error rates in inhaler application. 84% report this occurring “often” to “very often”. Conclusions: Pulmonologists see patient individual selection of inhalers as crucial for a successful inhaled therapy and often exclude substitution. However, they assume that general practitioners do not commonly cross “aut-idem”. Prohibiting substitution of prescribed inhalers through national regulations may lead to less error rates and a better care for patients in Germany. Funded by GlaxoSmithKline. HO-15-16914. PMD120 Are Criteria Commonly Used in the Benefit Assessment of Drugs also Applicable to Medical Devices? - A Systematic Literature Review Zischka M, Sauer A, Günzel F, Italia N, Kulp W Xcenda GmbH, Hannover, Germany
Objectives: Since 2016 new medical devices of high-risk classes IIb and III have to undergo benefit assessment according to § 137h SGB V to be included in the DRGSystem. As of now, the same evidence criteria are applied as in drug benefit assessment. Objective of our research is whether drug specific criteria are also applicable to medical devices and to which extend. Methods: We performed a systematic literature research in Medline and Cochrane databases to identify RCTs, published between January 2015 and May 2016, using relevant MeSH and controlled terms. Since an unrestricted search for medical devices is neither practical nor feasible, we limited our search to representatives of the risk classes of interest. Therefore, catheters, surgical equipment as well as prostheses/implants, considering specific procedures, were chosen. Hits that met inclusion criteria were categorized, e.g. by sample size, medical indication, blinding, and outcomes, and study quality was assessed. Results: We identified 2,320 hits from which 1,745 hits were excluded by title and abstract. Of the remaining 575 hits more than 50 % were excluded because criteria of high-risk class was not met. 312 hits presented data on catheters (182), prostheses/implants (104), and stents (26). Compared to RCT in drugs, studies where relatively small (< 50 patients). Of the publications related to catheters, 17 were blinded, four of which were double-blinded. Similar results were obtained for prostheses/implants and stents. Conclusions: Although publications were indexed as RCT, the majority of them were neither randomized nor controlled. Compared to RCTs in drugs, study size was relatively small and methodological aspects partly differed from IQWiG-accepted approaches. From a health-policy perspective, it seems advisable to adapt drug benefit assessment criteria to device specific requirements in order to properly assess the possible added medical benefit of innovative highrisk medical devices.
PMD121 New Legislative Pathway of Turkey for Assessing Reimbursement Applicatons of Medical Devices Ozdiler Copur F, Akgul T, Ozdemir Saltik Z, Can H, Askin E Medtronic, Istanbul, Turkey
Objectives: In the Turkish medical device industry, budgetary concerns usually overweight other crucial issues like quality and effectiveness of medical devices on clinical and economical outcomes. Reimbursement application and assessment principles have not been regulated until very recently by the Social Security Institution (SSI), the only competent authority for reimbursement. After productive communications and collaboration between SSI and industry, the new directive, “Procedures and Principles of Medical Device Applications” and its guideline “Medical Device Application” has been published by SSI on Apr, 20th 2016,Objective of this study is to describe reimbursement application and assessment procedures for Medical Devices and the critical points of the guideline and the directive. Methods: The Directive and Guidelines dated 20.04.2016 are analyzed. Results: Ministry of Health (MoH) registration is a prerequisite for reimbursement in Turkey. All medical devices are subject to specialty-based positive listing, consisting of generic definitions and codes with corresponding ceiling reimbursement prices set by SSI. For inclusion into the positive lists, the manufacturer/ distributor has to submit a “reimbursement dossier” to SSI. To get a new reimbursement code for innovative, first-in-it-class products (Type A) and for product differentiation in terms of size or specifications (Type B); high-level clinical evidence and local health economics and budget-impact analyses are required. C and D type applications are for matching to an existing code and getting reimbursement approval for new barcodes of the already reimbursed products. Conclusions: Local economic analysis and information on reimbursement status in reference countries may seem challenging to companies, due to lack of local data and difference between Turkish and reference countries’ healthcare systems. However, longwaited Directive regulates the decision mechanism of SSI and provides opportunity of fair competition to the industry. It may be seen as an evidence indicating the mindset shift of SSI towards the value based approach. PMD122 A New Accelerated Early Access Process for Diagnostics in France Hanna E, Azaiez C, Auquier P, Toumi M Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille Université, Marseille, France
Objectives: The fast development of precision medicine led to fast development of new diagnostic tools. New diagnostics face a complex access process delaying potential benefit to patients. France put in place an accelerated access process (AAP) for innovative diagnostics with immature data. This study objective is to describe AAP and analyse its potential impact on development and patient access to innovative diagnostics. Methods: We reviewed the French regulation for AAP called Référentiel des actes Innovants Hors Nomenclature (RIHN): Instruction N° DGOS/ PF4/2015/258, July 31st, 2015, and analysed potential implications on diagnostics manufacturers. Results: In July 2015, the General directorate of health services (DGOS) established RIHN to support ongoing development of innovative laboratory medicine and pathology diagnostics. RIHN support evaluation of innovative diagnostics in “real life” before HTA assessment and pricing is issued. Payers support manufacturers by co-funding studies to collect clinical and economic data to prove efficacy and cost-effectiveness. Early dialogue with payers helps to increase diagnostic acceptability and chances of reimbursement. In parallel to study support, RIHN ensures a temporary reimbursement for 3 years; after what, there are 3 options: positive evaluation by DGOS and dossier transmission to HAS, negative evaluation and test withdrawal, unassessable evidence due to limited data and extension of the inclusion in RIHN for 2 additional years. RIHN endorsed tests are reimbursed on yearly basis to hospitals, retrospectively according to number of tests performed. Conclusions: After the Temporary Authorisations for Use (ATU) and “forfait innovation”, RIHN constitutes the third pillar to support early access to innovation in France. It aims to remove hurdles and facilitate access of innovative laboratory medicine and pathology tests onto French market by providing temporary funding. French payers are moving from a “see-to-pay” to “pay-to-see” strategy. France might now be considered an attractive market for launching new and innovative diagnostics. PMD123 Cost-Effectiveness Analysis of Screening Strategies for Atrial Fibrillation in England and Wales Thom H1, Hollingworth W1, McAleenan A1, Davies P1, Higgins J1, Okoli GN1, Sterne J1, Feder G1, Eaton D2, Hingorani A3, Fawsitt C1, Lobban T4, Bryden PA5, Richards A1, Sofat R3, Welton NJ1 1University of Bristol, Bristol, UK, 2Anticoagulation Europe, Kent, UK, 3University College London, London, UK, 4Atrial Fibrillation Association, Warwickshire, UK, 5Roche, Basel, Switzerland
Objectives: To determine the cost-effectiveness of primary care screening strategies for atrial fibrillation in England and Wales. Methods: We built a decision model to compare no screening with opportunistic or systematic screening using 12 permutations of screening tests (modified blood pressure monitors (MBP), photoplethysmography (PP), pulse palpation, or electrocardiograms (ECG)) and interpreters (automatic, nurse, GP, cardiologist). Positive tests at screening were assumed to be confirmed by diagnostic 12-lead ECG interpreted by cardiologist. Long term costs and quality of life for true positive AF cases treated with Apixaban or aspirin were modelled using a Markov model. Model inputs were identified using systematic reviews. We calculated incremental net benefit (INB at £20,000 per QALY), compared to no screening, of screening at ages 55, 60, 65, 70, 75, and 80 and repeat screening strategies with 5-yearly intervals starting at these ages and continuing to age 80. Results: Screening was more cost-effective than no screening. Provided GP uptake is high, opportunistic screening was more likely to be cost-effective than systematic screening. PP (based on one study), MBP and pulse-palpation by a nurse were most likely to be cost-effective. Screening at higher ages was most
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 3 4 7 – A 7 6 6
diagnostic odyssey. Complete resource use data were collected at the University Medical Center Utrecht (UMCU), Utrecht, the Netherlands. We categorized the young patients according to their WES-based diagnosis (yes, no, and uncertain), and assessed the per-patient healthcare activities and corresponding costs before (PRE) and after (POST) the diagnosis was obtained. Results: In the cohort of 371 patients, 129 patients received a genetic diagnosis (35%). In 89 (24%) patients no mutation was identified and 153 patients (41%) had an uncertain (variant-ofunknown-significance) diagnosis. We estimated implementation of WES-first would result in mean savings of € 5122 euro per patient resulting from replacement of all other genetic technologies and 50% of non-genetic diagnostic tools and consults. A decrease in costs was observed in the majority of categories POST-WES, only the costs of consults in all categories and the costs of genetic tests after not having a diagnosis (+300%) increased. Conclusions: Our study confirms that WES is a cost-effective replacement of traditional diagnostic technologies for patients with ID. Looking at the high, although similar in all WES-based diagnostic groups, costs WES could be a cost-efficient and unique option when implemented early in patient care: it could reduce health care costs and induce the amount of distinct diagnoses. On the other hand, our data demonstrate that for patients not having a diagnosis the diagnostic costs have increased after WES. This indicates a change in mindset is needed when implementing WES on a broad scale. PMD119 Substitutability of Dry Powder Inhalers: A Perspective from German Pulmonologists Redeker M1, Schwarz E1, Mohrlang C1, Hellmann A2, Hellmann A3, Hering T3, Hering T4, Andres J5, Andres J3 1GlaxoSmithKline GmbH & Co. KG, München, Germany, 2Zentrum für Pneumologie, Onkologie und Schlafmedizin am Diako, Augsburg, Augsburg, Germany, 3Wissenschaftliches Netzwerk des Bundesverbandes der Pneumologen/BdP (WinPneu), Heidenheim, Germany, 4Lungenarztpraxis Tegel, Berlin, Germany, 5MedWiss GmbH, Weinitzen, Germany
Objectives: Inhaled therapies can be substituted in German pharmacies unless prohibited through an “aut-idem-cross” by the physician. Substitution can lead to patients receiving unfamiliar inhalers resulting in problems with the application. This research aims to gain insights regarding German pulmonologists’ perspective and experience on substitutability including how often inhalers are the cause for therapy change. Methods: 90% of all German pulmonologists are affiliated to the Federal Association of Pulmonologists. All 850 were asked to participate in the survey with 304 responding. Questions involved a four- or five-point scale. The list of reasons for therapy changes is based on literature, physician- and expert-opinion. Results: 97% of all pulmonologists see patient individual selection of inhalers as “very important”. 76% report prohibiting substitution “very often” and “often”. However, 34% of pulmonologists estimate that only 9% of general practitioners use aut-idem-cross “very often” and “often”. 67% estimate that 67% of general practitioners prohibit substitution “rarely” and “very rarely”. Pulmonologists often take the therapeutical decision for medicine and inhaler but general practitioners often make follow-up prescriptions. 39% of pulmonologists report that patients visit them “very often” and “often” because they did not receive the prescribed medication due to substitution in the pharmacy. Pulmonologists report that patients had problems in applying their substituted inhaler (43% “sometimes”, 41% “often”, 11% “very often”). Pulmonologists report making on average twelve therapy changes biweekly. Most common reason for therapy changes are error rates in inhaler application. 84% report this occurring “often” to “very often”. Conclusions: Pulmonologists see patient individual selection of inhalers as crucial for a successful inhaled therapy and often exclude substitution. However, they assume that general practitioners do not commonly cross “aut-idem”. Prohibiting substitution of prescribed inhalers through national regulations may lead to less error rates and a better care for patients in Germany. Funded by GlaxoSmithKline. HO-15-16914. PMD120 Are Criteria Commonly Used in the Benefit Assessment of Drugs also Applicable to Medical Devices? - A Systematic Literature Review Zischka M, Sauer A, Günzel F, Italia N, Kulp W Xcenda GmbH, Hannover, Germany
Objectives: Since 2016 new medical devices of high-risk classes IIb and III have to undergo benefit assessment according to § 137h SGB V to be included in the DRGSystem. As of now, the same evidence criteria are applied as in drug benefit assessment. Objective of our research is whether drug specific criteria are also applicable to medical devices and to which extend. Methods: We performed a systematic literature research in Medline and Cochrane databases to identify RCTs, published between January 2015 and May 2016, using relevant MeSH and controlled terms. Since an unrestricted search for medical devices is neither practical nor feasible, we limited our search to representatives of the risk classes of interest. Therefore, catheters, surgical equipment as well as prostheses/implants, considering specific procedures, were chosen. Hits that met inclusion criteria were categorized, e.g. by sample size, medical indication, blinding, and outcomes, and study quality was assessed. Results: We identified 2,320 hits from which 1,745 hits were excluded by title and abstract. Of the remaining 575 hits more than 50 % were excluded because criteria of high-risk class was not met. 312 hits presented data on catheters (182), prostheses/implants (104), and stents (26). Compared to RCT in drugs, studies where relatively small (< 50 patients). Of the publications related to catheters, 17 were blinded, four of which were double-blinded. Similar results were obtained for prostheses/implants and stents. Conclusions: Although publications were indexed as RCT, the majority of them were neither randomized nor controlled. Compared to RCTs in drugs, study size was relatively small and methodological aspects partly differed from IQWiG-accepted approaches. From a health-policy perspective, it seems advisable to adapt drug benefit assessment criteria to device specific requirements in order to properly assess the possible added medical benefit of innovative highrisk medical devices.
PMD121 New Legislative Pathway of Turkey for Assessing Reimbursement Applicatons of Medical Devices Ozdiler Copur F, Akgul T, Ozdemir Saltik Z, Can H, Askin E Medtronic, Istanbul, Turkey
Objectives: In the Turkish medical device industry, budgetary concerns usually overweight other crucial issues like quality and effectiveness of medical devices on clinical and economical outcomes. Reimbursement application and assessment principles have not been regulated until very recently by the Social Security Institution (SSI), the only competent authority for reimbursement. After productive communications and collaboration between SSI and industry, the new directive, “Procedures and Principles of Medical Device Applications” and its guideline “Medical Device Application” has been published by SSI on Apr, 20th 2016,Objective of this study is to describe reimbursement application and assessment procedures for Medical Devices and the critical points of the guideline and the directive. Methods: The Directive and Guidelines dated 20.04.2016 are analyzed. Results: Ministry of Health (MoH) registration is a prerequisite for reimbursement in Turkey. All medical devices are subject to specialty-based positive listing, consisting of generic definitions and codes with corresponding ceiling reimbursement prices set by SSI. For inclusion into the positive lists, the manufacturer/ distributor has to submit a “reimbursement dossier” to SSI. To get a new reimbursement code for innovative, first-in-it-class products (Type A) and for product differentiation in terms of size or specifications (Type B); high-level clinical evidence and local health economics and budget-impact analyses are required. C and D type applications are for matching to an existing code and getting reimbursement approval for new barcodes of the already reimbursed products. Conclusions: Local economic analysis and information on reimbursement status in reference countries may seem challenging to companies, due to lack of local data and difference between Turkish and reference countries’ healthcare systems. However, longwaited Directive regulates the decision mechanism of SSI and provides opportunity of fair competition to the industry. It may be seen as an evidence indicating the mindset shift of SSI towards the value based approach. PMD122 A New Accelerated Early Access Process for Diagnostics in France Hanna E, Azaiez C, Auquier P, Toumi M Faculté de Médecine, Laboratoire de Santé Publique, Aix-Marseille Université, Marseille, France
Objectives: The fast development of precision medicine led to fast development of new diagnostic tools. New diagnostics face a complex access process delaying potential benefit to patients. France put in place an accelerated access process (AAP) for innovative diagnostics with immature data. This study objective is to describe AAP and analyse its potential impact on development and patient access to innovative diagnostics. Methods: We reviewed the French regulation for AAP called Référentiel des actes Innovants Hors Nomenclature (RIHN): Instruction N° DGOS/ PF4/2015/258, July 31st, 2015, and analysed potential implications on diagnostics manufacturers. Results: In July 2015, the General directorate of health services (DGOS) established RIHN to support ongoing development of innovative laboratory medicine and pathology diagnostics. RIHN support evaluation of innovative diagnostics in “real life” before HTA assessment and pricing is issued. Payers support manufacturers by co-funding studies to collect clinical and economic data to prove efficacy and cost-effectiveness. Early dialogue with payers helps to increase diagnostic acceptability and chances of reimbursement. In parallel to study support, RIHN ensures a temporary reimbursement for 3 years; after what, there are 3 options: positive evaluation by DGOS and dossier transmission to HAS, negative evaluation and test withdrawal, unassessable evidence due to limited data and extension of the inclusion in RIHN for 2 additional years. RIHN endorsed tests are reimbursed on yearly basis to hospitals, retrospectively according to number of tests performed. Conclusions: After the Temporary Authorisations for Use (ATU) and “forfait innovation”, RIHN constitutes the third pillar to support early access to innovation in France. It aims to remove hurdles and facilitate access of innovative laboratory medicine and pathology tests onto French market by providing temporary funding. French payers are moving from a “see-to-pay” to “pay-to-see” strategy. France might now be considered an attractive market for launching new and innovative diagnostics. PMD123 Cost-Effectiveness Analysis of Screening Strategies for Atrial Fibrillation in England and Wales Thom H1, Hollingworth W1, McAleenan A1, Davies P1, Higgins J1, Okoli GN1, Sterne J1, Feder G1, Eaton D2, Hingorani A3, Fawsitt C1, Lobban T4, Bryden PA5, Richards A1, Sofat R3, Welton NJ1 1University of Bristol, Bristol, UK, 2Anticoagulation Europe, Kent, UK, 3University College London, London, UK, 4Atrial Fibrillation Association, Warwickshire, UK, 5Roche, Basel, Switzerland
Objectives: To determine the cost-effectiveness of primary care screening strategies for atrial fibrillation in England and Wales. Methods: We built a decision model to compare no screening with opportunistic or systematic screening using 12 permutations of screening tests (modified blood pressure monitors (MBP), photoplethysmography (PP), pulse palpation, or electrocardiograms (ECG)) and interpreters (automatic, nurse, GP, cardiologist). Positive tests at screening were assumed to be confirmed by diagnostic 12-lead ECG interpreted by cardiologist. Long term costs and quality of life for true positive AF cases treated with Apixaban or aspirin were modelled using a Markov model. Model inputs were identified using systematic reviews. We calculated incremental net benefit (INB at £20,000 per QALY), compared to no screening, of screening at ages 55, 60, 65, 70, 75, and 80 and repeat screening strategies with 5-yearly intervals starting at these ages and continuing to age 80. Results: Screening was more cost-effective than no screening. Provided GP uptake is high, opportunistic screening was more likely to be cost-effective than systematic screening. PP (based on one study), MBP and pulse-palpation by a nurse were most likely to be cost-effective. Screening at higher ages was most