Assessment of Decisions and Cost-Effectiveness Criteria Considered by Seven Health Technology Assessment Agencies

A752

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

Hwang EJ, Jang S, So S Health Insurance Review and Assessment Service in Korea, Seoul, South Korea

Objectives: Starting in September 2013, benefit enhancement policy for severe diseases(such as risk sharing scheme(RSS), upward adjustment of acceptable ICERs level) has been implemented to improve the patients’ accessibility to oncology drugs in Korea. This research aims to explore the changes of reimbursement recommendation before and after the implementation of the benefit enhancement policy for oncology drugs.  Methods: From 2007 to June 2016, All appraisal results that were assessed by DBCAC in HIRA were reviewed. The rate of reimbursement recommendation, the tendency of ICERs, proportion of RSS, etc. were assessed and compared between before and after the policy implementation for the oncology drugs.  Results: Up to June 2016, total 574 appraisals of new drugs(371 as ingredients) were assessed in HIRA. 71.3%(409/574) of them were recommended for the reimbursement(67.9%, 252/371, as ingredients). During the observation period, among total 107 appraisals of oncology drugs(69 as ingredients), the recommendation rate was 56.1%(60/107), 47.8%(33/69, as ingredients). Those drugs have been reimbursed in the 4.9 countries on the average among A7 countries. The recommendation rate was 52.5%(31/59), 44.7%(17/38, as ingredients) before the policy and 60.4%(29/48), 51.6%(16/31, as ingredients) after the policy. After the policy implementation, the acceptable ICERs levels have been raised(among 11 ingredients, before and after 4,7). After the policy implementation, 16 appraisals of oncology drugs were recommended for the reimbursement. 7 of them were recommended with RSS(43.8%), and types of RSS were refund(6/7), expenditure cap(1/7). 7 oncology drugs with RSS were assessed by CUA(n= 6), by the lowest price of A7(n= 1). 9 without RSS were assessed by weighted average price(n= 2), by CUA(n= 3), as drugs eligible for the exemption from economic assessment(n= 4). Regarding  RSS,  1 drugs for rare diseases was assessed by CUA.  Conclusions: The implementation of benefit enhancement policy for severe diseases seems to increase accessibility to oncology drugs that are clinically beneficial but having uncertainty in cost-effectiveness and having difficulty of producing clinical evidence.

PCN239 Which Types of Economic Evaluations are Considered by Health Technology Assessment Agencies? Puig-Peiró R1, Roset M2, Gilabert Perramon A1, Viayna E2, Prat A1, Gómez-Navarro V2 Health Service (CatSalut), Barcelona, Spain, 2IMS Health, Barcelona, Spain

1Catalan

Objectives: In the context of gradual implementation of economic evaluation of medicines, the Catalan Health Service (CatSalut) aimed to assess which types of EE were considered by seven Health Technology Assessment (HTA) agencies for a sample of 49 oncologic indications. Methods: The following agencies were included: HAS (France), IQWIG (Germany), NICE (United Kingdom), PBAC (Australia), pCODR (Canada), SMC (Scotland) and TLV (Sweden). Reports available online on agencies’ websites covering the target indications until October 2015 were reviewed. Information about the EE conducted was extracted and assessed: type of EE, perspective, type of costs, responsible for the model development, and whether a budged impact model (BIM) was included.  Results: 294 reports were identified across the seven agencies assessed, 74% of which included an EE and 33% included a BIM.Among agencies conducting EE (all but IQWIG), costutility analysis (CUA) was the most frequently applied (73.5%): 63% in TLV, 92% in SMC, 72% in PBAC, 97% in NICE, 54% in pCODR, and in the only EE identified in HAS. Cost-effectiveness and cost minimization analyses were observed in 11% and 10.5% of the reports, respectively. Most of the identified EE considered only direct costs; 16% and 3% of EE conducted by TLV and NICE, respectively, included indirect costs. Manufacturers participated in the development of all EE, either on their own or in collaboration with the agency or with an external committee. Only 83%, 76% and 68% of reports of PBAC, SMC and NICE, respectively, included a BIM.  Conclusions: The most common type of EE across the assessed indications for all agencies was the CUA, and only 3 agencies conducted BIMs for HTA. Indirect costs were seldom used. PCN240 Assessment of Decisions and Cost-Effectiveness Criteria Considered by Seven Health Technology Assessment Agencies Puig-Peiró R1, Planellas L2, Gilabert Perramon A1, Roset M2, Barrull C2, Prat A1, Solozabal M2 Health Service (CatSalut), Barcelona, Spain, 2IMS Health, Barcelona, Spain

1Catalan

Objectives: In the context of gradual implementation of economic evaluation of medicines, the Catalan Health Service (CatSalut) aimed to review decisions made and cost-effectiveness criteria considered by seven Health Technology Assessment (HTA) agencies for a sample of 49 oncologic indications.  Methods: Included agencies were HAS (France), IQWIG (Germany), NICE (United Kingdom), PBAC (Australia), pCODR (Canada), SMC (Scotland) and TLV (Sweden). Reports available online on agencies’ websites covering the target indications until October 2015 were reviewed in order to extract information on decisions made and criteria considered. For those reports including an EE, relationship between the type of recommendation and the estimated incremental cost-effectiveness ratio (ICER) was assessed.  Results: Overall, 294 reports were identified. TLV (97%) was the agency with a highest proportion of positive opinions, followed by HAS (96%), IQWIG (83%), pCODR (81%), PBAC (60%), SMC (55%) and NICE (45%). More than half of the recommendations made by SMC, TLV, pCODR and PBAC were linked to population restrictions or specific conditions of use. ICERs reported for indications recommended with no restriction or condition were below 50.000€  per quality adjusted life years (QALY) by NICE, PBAC, pCODR and SMC. Although there is variability on ICERs reported across agencies, there is a positive correlation between the reported ICER’s threshold and the decision’s restriction.  Conclusions: High variability in decisions and reported ICERs was observed across agencies, which could be due to both technical (different decision criteria) and strategic (binding decision and its impact in the use of drugs) reasons.

PCN241 Implications of Total Budget Impact on Oncology Drug Pricing in The us And EU5 Xue Y, Degun R, Montilva J Navigant Consulting, London, UK

Objectives: To assess the impact of maintaining current pricing practices on total oncology spend in US and EU5 countries, and implications on average price of existing and pipeline therapies in the future  Methods: Number and types of pipeline oncology products, list prices for existing therapies, and analyst forecasts for prices of pipeline products were gathered to calculate the anticipated total budget impact based on status quo assumptions. Multiple scenarios for “acceptable” total budget impact were then developed, to determine ranges of corresponding unit prices for individual products  Results: Drug pricing practices is becoming the subject of increasing regulatory and political interest. Current forecast models often assume continued premium pricing of new oncology therapies, often beyond USD$100,000 per year in the US and € 70,000 per year in the EU. However, at an aggregate level, PhRMA reports that 224 medicines and vaccines are currently in phase III testing for cancer by US companies alone. Accounting for phase III success rates, likelihood of approval, and removing trials in existing indications, large numbers of new products / indications will become available in the next 3 – 5 years. Based on current analyst epidemiology and pricing assumptions, this represents a dramatic increase in total payer spend. Assuming government / payer interventions to maintain total oncology drug spend to 1) current level adjusted for inflation 2) historical increases in total healthcare budget 3) increase in population > 60, significant downward adjustments for individual unit prices would be necessary, especially in the US.  Conclusions: With increasing number of pipeline products poised to launch in the oncology space and increasing scrutiny on drug pricing across the Atlantic, a more holistic and forward looking view may be necessary for oncology pricing. PCN242 Patient Individual Therapy as Appropriate Comparator in AmnogDossiers in The Field of Oncology: Status Quo and Strategic Implications For Pharmaceutical Companies Templin C, Italia N, Damen D, Kulp W Xcenda GmbH, Hannover, Germany

Objectives: For the assessment of an added value in the AMNOG process in Germany G-BA defines appropriate comparators. These can be categorized into four classes: (1) one specific drug; (2) a list of drugs; (3) best supportive care (BSC); and (4) patient individual therapy. The latter category implies several methodological challenges for having an added benefit granted. Therefore our analysis focused on dossiers with patient individual therapy as appropriate comparator.  Methods: Information was retrieved from the G-BA website. All non-orphan AMNOG-dossiers in oncology published until the end of 2015 were screened. Data on indication, size of target population, line of therapy, the comparator utilized in the company’s dossier, and outcome (added benefit) were obtained.  Results: 41 relevant dossiers with 71 indications were identified. The appropriate comparators were distributed as follows: 26 (36.6%) specific drug, 15 (21.1%) list of drugs, 19 (26.8%) BSC and 11 (15.5%) patient individual therapy. In the last years, the latter was increasingly defined as appropriate comparator (2011-2013: 3.0% of all comparators; 2014-2015: 26.3% of all comparators). For clinical trials not containing patient individual therapy as comparator, the chance of getting an added value recognized was low (substantial added value: 4, no added value proven: 7). In 3 out of 4 dossiers with added benefit direct evidence to patient individual therapy was presented. In contrast, in 5 out of 7 indications with no added value the comparator in the relevant study deviated from patient individual therapy.  Conclusions: Patient individual care is rarely used in clinical trials. Despite this, it seems that the relevance of comparative evidence against patient individual therapy is increasing. Pharmaceutical companies should take this development into account, in order to maximize the likelihood of getting an added benefit granted. PCN243 Real World Data: Use of Novel Treatments and Biomarkers in Malignant Melanoma in European Countries von Bredow D1, Kolb N1, Bernhardt M2, Schmidt N2 1IMS Health GmbH & Co OHG, Munich, Germany, 2IMS Health GmbH & Co OHG, Frankfurt, Germany

Objectives: Since the first unravelling of complete intracellular signal transduction pathways, many key molecules have been identified causatively involved in carcinogenesis. Novel targeted therapies and immunotherapies developed in the last years offer new treatment options particularly for patients with late stage malignant melanoma. Predictive biomarkers such as BRAF mutations allow identification of patients who may benefit from specific treatments. In the study presented here, we investigated changes in treatment of malignant melanoma between 2009 and 2015 in EU5 countries, and analyzed real-world patient level treatment pathways in clinical practice.  Methods: In the analyses, 1,267 patients with late-stage (stage IIIc/IV)malignant melanoma documented in IMS® Oncology Analyzer during 2009, 2014 and 2015 were included. IMS® Oncology Analyzer contains anonymous retrospectively collected patient level data on disease and treatment history, provided by hospital- and office-based physicians. For each year, the proportion of molecules used across lines and treatment pathways of patients with more than one line of treatment were analyzed. In addition, treatment pathways were evaluated depending on BRAF status (wild-type / mutant).  Results: In 2009, most treatments across first three lines were cytotoxic regimen (49%) or interferons (41%). In 2014, 42% of treatments included BRAF inhibitors, 19% CTLA-4 antagonists. In 2015, PDL1 inhibitors were launched, resulting in 9% of patients receiving treatments affecting PD1/PDL1 pathways. In 2014 and 2015, 48% of patients with a BRAF mutation received BRAF inhibitors during first line, 46% during second line and 30% during third line.  Conclusions: Within 6 years, novel targeted therapies and immunotherapies became standard of care in treatment of late-stage melanoma. With BRAF