Reimbursement Status Research Articles

Patient Acceptance of Prescribed and Fully Reimbursed mHealth Apps in Germany: An UTAUT2-based Online Survey Study

The study aims to (1) investigate current levels of patient acceptance of mHealth in Germany; (2) determine the influencing factors of patients' intention to use, and (3) test the influence of prescription and reimbursement status on patient acceptance. Online survey with 1349 participants, of which 1051 were complete and included for statistical analysis, from a broad cross-section of the German population, addressing both users of mobile health (mHealth) applications and people without prior experience. SEM modeling based on a combination of two theoretical frameworks: the extended Unified Theory of Acceptance and Use of Technology and Health Protective Behavior Theories were used to assess acceptance. Users of mHealth in Germany are mostly patients between the ages of 30 – 50 with mental health or endocrine conditions. General willingness to use mHealth apps / DiGAs (mHealth apps fully reimbursed by social health insurance) is high at 76%, especially if they are governmentally certified, however only 27% of respondents were willing to pay out of pocket. With the exception of a spike in performance expectancy and data security, DiGAs lack a clear differentiation from mHealth apps. Perceived self-efficacy and performance expectancy are significant predictors of willingness to use digital health interventions; with age, attitude, and e-literacy being key demographic predictors. A key takeaway for regulators, providers of mHealth apps/ DiGAs, and other stakeholders involved in mHealth adoption is the importance of addressing negative beliefs early on, targeted communication around effortless usage of mHealth services across age groups and demographics, and focus on highlighting expected benefits of mHealth app/ DiGA usage.

PD16 Reassessment And Selection Strategy Of Dipeptidyl Peptidase-4 Inhibitors For Ministry Of Health Malaysia Medicine Formulary

IntroductionThe number of anti-diabetics listed in the Ministry of Health Medicine Formulary (MoHMF) and the proposal to change prescribing category for usage in primary care facilities has increased each year causing difficulty in monitoring effectiveness, safety and rational use of the treatment and budget management. This study aimed to describe the reassessment and selection strategy of Dipeptidyl Peptidase-4 Inhibitors (DPP4-i), a class of anti-diabetic medicines in the MoHMF.MethodsA literature review on the comparative effectiveness and safety of all available DPP4-i in MoHMF were conducted. Comparative treatment cost and utilization of DPP4-i were analyzed. Approved MoHMF indications were listed and compared against approved Drug Control Authority (Malaysia) indications. Information on clinical guidelines recommendations and listing or reimbursement status in non-Ministry of Health Malaysia (MoHM) public institutions and other countries were obtained. All findings were presented to MoHM drug expert committee for DPP4-i selection strategy. The final recommendation based on consensus among drug expert committee and Pharmaceutical Services Program were presented to the MoHMF Panel.ResultsThe MoHMF Panel acknowledged that the efficacy and safety profile of all DPP4-i were equivalent across therapeutic group as supported by strong evidence, hence, their selection can be made via cost-minimization strategy. The call for contract tender for single tablet was conducted based on therapeutic group (drug class) to encourage price competition and contracts were awarded to two DPP4-i which offered the lowest treatment costs. Saxagliptin and vildagliptin were awarded as contract items while sitagliptin remained as local purchase item. Prescribing category for all DPP4-i in MoHMF were streamlined accordingly. Linagliptin was disinvested due to sufficient availability of alternatives.ConclusionsSelection strategy and disinvestment has successfully reduced the number of DPP4-i listed in MoHMF thus allowing more efficient clinical and cost monitoring. Cost minimization through tender by therapeutic group was the first to be done and has efficiently provided an avenue for price competition which results in saving to MoHM.

HPR129 Reimbursement Status of European Medicines Agency Authorised Drugs in Spain

HPR129 Reimbursement Status of European Medicines Agency Authorised Drugs in Spain

HPR176 Reimbursement of Antineoplastic and Immunomodulating Agents in Turkey: Comparison With Approvals of the FDA Between 2010-2021

Antineoplastic and immunomodulating agents classification of the World Health Organization’s (WHO) ATC system (L) include drugs that alter activity of the immune system (immunomodulators) and anticancer (antineoplastic) drugs. The research and clinical trials for this group of molecules is intense due to the increasing incidence and prevalence rates of diseases covered by this group. The aim of this study is to elaborate the market approval and reimbursement status of new molecules or new indications for already available molecules in this group in Turkey in comparison to the approvals of the FDA between 2010-2021.

Primary nonadherence to drugs prescribed by general practitioners: A Dutch database study.

Primary nonadherence (PNA) is defined as not filling the first prescription for a drug treatment. PNA can lead not only to poor patient outcomes but also to exposure misclassification in written prescription databases. This study aims to estimate PNA in primary care in the Netherlands and to investigate associated factors. Patients from the Nivel Primary Care Database (Nivel-PCD) who received a new prescription (>1year not prescribed) from a general practitioner in 2012 were linked to pharmacy dispensing information of consenting pharmacies based on sex, year of birth, four-digit postal code and at least 50% matching Anatomical Therapeutic Classification codes. PNA was defined as not having a prescription dispensed within 30 days from the prescribing date. PNA was assessed overall and per drug class. The associations between PNA and several patient- and prescription-related characteristics were assessed using mixed-effects logistic regression models. After matching 86 361 of 396 251 subjects (21.8%) in the Nivel-PCD records to the pharmacy records, this study included 65 877 subjects who received 181 939 new drug prescriptions. Overall, PNA was 11.5%. PNA was lowest for thyroid hormones (5.5%) and highest for proton pump inhibitors (12.8%). Several factors were associated with PNA, such as having comorbidities (odds ratio [OR] 1.46, 95% confidence interval [CI] 1.37-1.56 for >3 active diagnoses, compared to no active diagnoses) or reimbursement status (OR 2.78, 95% CI 2.65-2.92 for not reimbursed drugs compared to fully reimbursed drugs). A total of 11.5% of newly prescribed drugs were not dispensed. This can lead to overestimation of the actual drug exposure status when using written prescription databases.

Medicine shortages: Product life cycle phases and characteristics of medicines in short supply-A register study.

Introduction: Product life cycle refers to all phases of a product from development to active market phase and finally the phase in which products possibly exit the market. The product life cycle of medicines in short supply has not been studied in depth, although there is some indication of mature products and products with lower prices and profit margins being exposed to shortages more often. The aim of this study was to examine the product life cycle phases and characteristics of medicines in short supply as well as the features of medicine shortages in Finland from 2017 to 2019. Material and methods: Register data on medicine shortages of human medicinal products from 2017 to 2019 was combined with timely data on marketing authorizations and reimbursement status to gain data on product life cycle phases and characteristics (e.g., the age and the reimbursement status) of medicines in short supply and the features of medicine shortages. The data were analyzed in descriptive manner using appropriate statistical testing. Results: 3,526 shortages were reported during the 3-year study period and the number of shortages increased annually. The average duration of a shortage was 83 days and shortages affected 660 active pharmaceutical ingredients. Most often, shortages occurred with medicines affecting the nervous system, the cardiovascular system, and the genitourinary system. A majority of shortages (n = 2,689) was reported in the reimbursable medicines group, where shortages increased as the number of patients receiving reimbursements increased (p < 0.001). In the reimbursable medicines group, shortages most commonly involved medicines aged 15–19, 20–24, and 25–29, whereas with both reimbursable and non-reimbursable products the shortages most often occurred in medicines aged 50–54. The frequency of shortages differed between the groups (p < 0.001) when both age and reimbursement status were taken into account. Conclusion: Medicine shortages are common and affect commonly used medicines. Product life cycle phase has an effect on the frequency of shortages: Reimbursable medicines and medicines exposed to changes in life cycle are more likely to face a shortage. The impacts of product life cycle on the availability of medicines and medicine shortages should be studied in more detail.

The impact of the use of treatments not included in the reimbursement classes in the care of rare patients: a real world study.

Rare disease (RD) patients present complex therapeutic needs. When there are therapeutic options available, orphan drugs (OD) represent only a limited proportion of prescribed treatments. This study aims at investigating the real-world use of treatments considered not replaceable and essential for the care of RD patients, besides their reimbursement status, using data from a RD population-based registry. The study is based on data derived from the Veneto region RD registry. For the period 2019-2020, we have analyzed the prescriptions of treatments defined as essential and not replaceable, besides their reimbursement status, included in therapeutic plans issued by RD expert Centres for patients resident in the Veneto region (north-east of Italy, 4.9 million inh.). The correspondent pharmaceutical costs have been estimated as well. In the study period there have been 22.186 prescriptions, included in 9,197 therapeutic plans issued for RD patients resident in the monitored area. The plans present a high level of complexity in terms of number and type of prescribed treatments, with 11% of the plans containing 5 or more prescriptions. 3,041 medicinal products have been prescribed in the study period, of whom 41% are drugs. Although these prescriptions are distributed among all the groups of RD patients, only a limited proportion of products (n=10) is responsible of the 50% of all the costs attributable to these treatments. Overall, the annual cost attributable to essential treatments not directly reimbursed by the national health system is quantifiable in 1 million euros per million inhabitants. This real-world study offers a snapshot of the complexity of treatments defined as essential, besides their reimbursement status, in therapeutic plans issued by RD expert Centres for a consistent group of RD patients monitored by a population-based registry. It highlights the complexity of the therapeutic approaches put in place for the care of RD patients, including drugs and a variety of other treatments. Population-based registries collecting data on prescribed treatments can contribute to understand the therapeutic needs of RD patients, treatments' accessibility and the impact of prescriptions on the global pharmaceutical costs.

Abstract NS7: Steps To An Early And Successful Adoption Of Iv Tenecteplase By A Large Integrated Medical System

Background: In 2020, a large integrated healthcare system consisting of 21 stroke centers implemented IV tenecteplase as the first line pharmaceutical treatment for acute ischemic stroke (AIS) prior to universal adoption in national clinical practice guidelines and FDA approval for stroke. Purpose: To describe the steps taken for successful implementation of IV tenecteplase as a first therapy for AIS in 21 stroke centers, part of an integrated system. Methods: Pre-implementation steps included gaining systemwide acceptance, especially from key stakeholders such as emergency department staff, patient care services, tele-stroke and neurology physicians, as well as risk management. We addressed potential impacts to related to reimbursement, stroke certification, and national award status. Inclusive changes were made to related policy and procedures, order sets, and other documentation. Standardized training and beta-testing were conducted and a sequential roll-out implemented along the real-time central monitoring. Results: Stakeholder groups were engaged prior to implementation with town halls and in-person and desktop simulation exercises. Beta-testing took place in August 2020 with three pilot sites with full system wide implementation by Nov. 2020. Since full implementation, tenecteplase is the only thrombolytic that has been ordered (n=423). Door in door out times have decreased (median 131 minutes pre- to 88 minutes post; p&lt;0.05). In addition, symptomatic ICH rates have decreased by greater than 50% (p&lt;0.05) and there are no reports of systemic bleeding or angioedema. Conclusion: Tenecteplase has been shown to be safe and feasible as a first line thrombolytic within our organization. Strong nurse and physician leads, as well as thoughtful and inclusive implementation steps were key in the successful adoption of this system-wide change.

Survey of transfemoral amputee experience and priorities for the user-centered design of powered robotic transfemoral prostheses

BackgroundTransfemoral amputees experience a complex host of physical, psychological, and social challenges, compounded by the functional limitations of current transfemoral prostheses. However, the specific relationships between human factors and prosthesis design and performance characteristics have not yet been adequately investigated. The present study aims to address this knowledge gap.MethodsA comprehensive single-cohort survey of 114 unilateral transfemoral amputees addressed a broad range of demographic and clinical characteristics, functional autonomy, satisfaction and attitudes towards their current prostheses, and design priorities for an ideal transfemoral prosthesis, including the possibility of active assistance from a robotic knee unit. The survey was custom-developed based on several standard questionnaires used to assess motor abilities and autonomy in activities of daily living, prosthesis satisfaction, and quality of life in lower-limb amputees. Survey data were analyzed to compare the experience (including autonomy and satisfaction) and design priorities of users of transfemoral prostheses with versus without microprocessor-controlled knee units (MPKs and NMPKs, respectively), with a subsequent analyses of cross-category correlation, principal component analysis (PCA), cost-sensitivity segmentation, and unsupervised K-means clustering applied within the most cost-sensitive participants, to identify functional groupings of users with respect to their design priorities.ResultsThe cohort featured predominantly younger (< 50 years) traumatic male amputees with respect to the general transfemoral amputee population, with pronounced differences in age distribution and amputation etiology (traumatic vs. non-traumatic) between MPK and NMPK groups. These differences were further reflected in user experience, with MPK users reporting significantly greater overall functional autonomy, satisfaction, and sense of prosthesis ownership than those with NMPKs, in conjunction with a decreased incidence of instability and falls. Across all participants, the leading functional priorities for an ideal transfemoral prosthesis were overall stability, adaptability to variable walking velocity, and lifestyle-related functionality, while the highest-prioritized general characteristics were reliability, comfort, and weight, with highly variable prioritization of cost according to reimbursement status. PCA and user clustering analyses revealed the possibility for functionally relevant groupings of prosthesis features and users, based on their differential prioritization of these features—with implications towards prosthesis design tradeoffs.ConclusionsThis study’s findings support the understanding that when appropriately prescribed according to patient characteristics and needs in the context of a proactive rehabilitation program, advanced transfemoral prostheses promote patient mobility, autonomy, and overall health. Survey data indicate overall stability, modularity, and versatility as key design priorities for the continued development of transfemoral prosthesis technology. Finally, observed associations between prosthesis type, user experience, and attitudes concerning prosthesis ownership suggest both that prosthesis characteristics influence device acceptance and functional outcomes, and that psychosocial factors should be specifically and proactively addressed during the rehabilitation process.

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

BackgroundWe examined if oncology drug indications with high clinical benefit, as measured by the American Society of Clinical Oncology Value Framework (ASCO‐VF) and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO‐MCBS), received public reimbursement status faster than those with lower clinical benefit from the time of pan‐Canadian Oncology Drug Review (pCODR) recommendation.MethodsOncology drug indications submitted to pCODR between July 2011 and October 2018 were examined. Included indications had a regulatory approval date, completed the pCODR review process, received a positive pCODR recommendation, and been funded by at least one province. Trials cited for clinical efficacy were used to determine the clinical benefit (per ASCO‐VF and ESMO‐MCBS) of drug indications.ResultsEighty‐four indications were identified, yielding 65 ASCO‐VF and 50 ESMO‐MCBS scores. The mean ASCO‐VF and ESMO‐MCBS scores were 44.9 (SD = 21.1) and 3.3 (SD = 1.0), respectively. The mean time to provincial reimbursement from pCODR recommendation was 13.2 months (SD = 9.3 months). Higher ASCO‐VF and ESMO‐MCBS scores had low correlation with shorter time to reimbursement, (ρ = −0.21) and (ρ = 0.24), respectively. In the multivariable analyses, ASCO‐VF (p = 0.40) and ESMO‐MCBS (p = 0.31) scores were not significantly associated with time to reimbursement. Province and year of pCODR recommendation were associated with time to reimbursement in both ASCO and ESMO models.ConclusionsOncology drug indications with higher clinical benefit do not appear to be reimbursed faster than those with low clinical benefit. This suggests the need to prioritize oncology drug indications based on clinical benefit to ensure quicker access to oncology drugs with the greatest benefits.

Health Technology Assessment (HTA) evidence, regulatory classification and reimbursement of medicine: the case of glucosamine

Abstract Objectives To examine the relationship among Health Technology Assessment (HTA) evidence, regulatory classification and reimbursement of health products using glucosamine as a case study. Data of HTA evidence, regulatory classification and reimbursement of glucosamine from 13 countries were extracted from official government websites and peer-reviewed journal articles. Role and responsibility of HTA in each country along as well as the regulatory approval process and reimbursement status of health products were reviewed. The case of glucosamine was then analysed to explore the regulatory classification, reimbursement and its HTA evidence from past to present. Key findings For regulatory classification, we found that glucosamine is classified as either medicine (9 from 13 countries) or a dietary supplement (4 from 13 countries) depends on where glucosamine is seeking its market approval. Reimbursement also differs among the countries. We summarized the key factors that could be the cause of these variations. First, the clinical evidence of glucosamine is still in question especially its efficacy and as a results its cost-effectiveness. This evidence is important for policy consideration. Secondly, different level of HTA approach in each healthcare system and country context effect on how HTA evidence is utilized and synthesized. Lastly, company’s strategic positioning is the first key stakeholder to decide whether their product would be registered as medicine or dietary supplement. Summary The variation of HTA evidence in a diverse healthcare system affects regulatory classifications and reimbursement. This can result in different levels of patient access to health products.

Early access programs and managed entry agreements for medicines in Italy: results of a Focus Group (Early Access Programs and Managed Entry Agreement).

Early access of medicines occurs with an uncertainty in the evidence even higher than the one experienced when price and reimbursement status is negotiated. Our aim is discussing the role of managed entry agreements (MEA) within early access programs (EAP) in Italy. The discussion relied on a Focus Group, participated by twelve experts, including clinicians and representatives of regulatory authorities, regional and local pharmaceutical departments, pharmaceutical companies, and an association advocating for active citizenship. The Focus Group emphasised that the topic under discussion should be embedded into a more general reform of EAP in Italy. The 648 List mostly includes mature products and indications that are rarely launched into the market afterwards. The 5% Fund is affected by an important administrative burden uncertainty of the timing of reimbursement. Starting from the discussion on MEA and EAP, the Focus Group recommended a new legislation better regulating EAP, that early access concerns specific classes of medicines selected on the grounds of the need to guarantee a rapid access and to collect real world data, that early access can be accompanied by outcome-based and population-based MEA, and that MEA are embedded into the subsequent price and reimbursement negotiation.

Does information by pharmacists convince the public to get vaccinated for pneumococcal disease and herpes zoster?

BackgroundPneumococcal diseases (PN) and herpes zoster (HZ) are preventable infections in the adult population.AimsThis study aimed to identify the vaccination rates at 1 year after pharmacist-led provision of information in the community. The objectives were to reveal the reasons for not being vaccinated and to determine opinions and awareness of PN and HZ vaccination among public.MethodsA prospective study was conducted in five social and solidarity centres in Turkey. Participants were educated by a pharmacist about PN and HZ diseases, vaccinations and reimbursement status, respectively. All participants were followed by telephone 1 year after to determine their vaccination status.ResultsA total of 155 participants (72.9% male; mean age was 68.72 ± 9.04 years) were included. With respect to PN and HZ vaccines, it was found that 40% and 12.7% of participants knew about the respective vaccines. Following the pharmacist’s educational session, 52.9% and 51.6% were willing to have the respective vaccine, but only 5.7% and 0.8% respectively got vaccinated 1 year after the educational session. Perceived disease severity, provision of information by a pharmacist, and reimbursement status of the vaccines were not associated with the vaccination rates.ConclusionsThe public obtain information on vaccines from friends and family members, which may result in misinformation and inappropriate behaviour in vaccination. Although educational sessions provided by pharmacists did not increase the actual vaccination rates for PN and HZ, public willingness to vaccination has increased.

Assessing the Accuracy of Sales Forecasts Submitted by Pharmaceutical Companies Applying for Reimbursement in Austria.

Objectives: Reimbursement decisions on new medicines require an assessment of their value. In Austria, when applying for reimbursement of new medicines, pharmaceutical companies are also obliged to submit forecasts of future sales. We systematically examined the accuracy of these pharmaceutical sales forecasts and hence the usefulness of these forecasts for reimbursement evaluations. Methods: We retrospectively analyzed reimbursement applications of 102 new drugs submitted between 2005 and 2014, which were accepted for reimbursement outside of hospitals, and for which actual reimbursed sales were available for at least 3 years. The main outcome variable was the accuracy ratio, defined as the ratio of forecasted sales submitted by pharmaceutical companies when applying for reimbursement to actual sales from reimbursement data. Results: The median accuracy ratio [95% confidence interval] was 1.33 [1.03; 1.74, range 0.15–37.5], corresponding to a median overestimation of actual sales by 33%. Forecasts of actual sales for 55.9% of all examined products either overestimated actual sales by more than 100% or underestimated them by more than 50%. The accuracy of sales forecasts did not show systematic change over the analyzed decade nor was it discernibly influenced by reimbursement status (restricted or unrestricted), the degree of therapeutic benefit, or the therapeutic area of the pharmaceutical product. Sales forecasts of drugs with a higher degree of innovation and those within a dynamic market tended to be slightly more accurate. Conclusions: The majority of sales forecasts provided by applicants for reimbursement evaluations in Austria were highly inaccurate and were on average too optimistic. This is in line with published results for other jurisdictions and highlights the need for caution when using such forecasts for reimbursement procedures.

Access to precision medicine in Thailand: a comparative study

PurposeThis study explored health insurance coverage of genetic testing and potential factors associated with precision medicine (PM) reimbursement in Thailand.Design/methodology/approachThe study employed a targeted review method. Thirteen PMs were selected to represent four PM categories: targeted cancer therapy candidate, prediction of adverse drug reactions (ADRs), dose adjustment and cancer risk prediction. Content analysis was performed to compare access to PMs among three health insurance schemes in Thailand. The primary outcome of the study was evaluating PM test reimbursement status. Secondary outcomes included clinical practice guidelines, PMs statement in FDA-approved leaflet and economic evaluation.FindingsCivil Servant Medical Benefits Scheme (CSMBS) provided more generous access to PM than Universal Coverage Scheme (UCS) and Social Security Scheme (SSS). Evidence of economic evaluations likely impacted the reimbursement decisions of SSS and UCS, while the information provided in FDA-approved leaflets seemed to impact the reimbursement decisions of CSMBS. Three health insurance schemes provided adequate access to PM tests for some cancer-targeted therapies, while gaps existed for access to PM tests for serious ADRs prevention, dose adjustment and cancer risk prediction.Originality/valueThis was the first study to explore the situation of access to PMs in Thailand. The evidence alerts public health insurance schemes to reconsider access to PMs. Development of health technology assessment guidelines for PM test reimbursement decisions should be prioritized.

Analysis of patient access to orphan drugs in Turkey

BackgroundRare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals.MethodsThe drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey.ResultsBased on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents.ConclusionAn orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

Adventures in getting paid

Adventures in getting paid

Factors influencing adjuvant treatment decision making among Chinese patients with hepatocellular carcinoma (HCC): Results of a patient survey.

346 Background: This study aimed to understand patient decision-making factors for post-operative adjuvant treatment after resection of hepatocellular carcinoma (HCC) in China. Methods: Patients who had received surgical resection of HCC in the last 2 years at 27 hospitals in China were surveyed using a questionnaire (https://pro.wenjuan.com/t/jUZFU). Feedback was collected by telephone or online. The survey covered three domains: adjuvant treatment decision making, selection of treatment regimen and the influence of recurrence risk on willingness to receive adjuvant treatment. For each domain, participants were asked to identify decision makers and to score decision-making factors from 1 (no influence) to 7 (high influence). All factors with an average score of 6 or 7 points were considered impactful, and those with scores ≤5 were not considered impactful. Descriptive statistics were used to summarize the findings. This study was approved by the ethics committee of Zhongshan hospital and patients provided informed consent. Results: In total, 2,220 valid responses were collected (2,183 patients from Zhongshan Hospital, 37 from 26 other hospitals); 75% of respondents were male, 60% were aged 50-70 years, and 50% had a monthly family income ≤5,000 Chinese Yuan (~$740 USD). Among respondents, 62% had received adjuvant treatment: 56% received systemic therapy (chemotherapy, tyrosine kinase inhibitors, immune therapy and traditional Chinese medicine), 27% transarterial chemoembolization (TACE) plus systemic therapy and 17% TACE alone. The majority of respondents (75%) identified physicians as the decision makers for use of adjuvant therapy and selection of treatment regimen. Of the 38% of respondents who did not receive adjuvant treatment, 90% gave ‘physician recommendation’ as the reason. When patients were the decision makers, factors influencing decisions to use adjuvant treatment and selection of treatment regimen, ranked from most to least influential, were efficacy (for prevention of tumor recurrence), reimbursement status, treatment side effects and cost. Patient willingness to receive adjuvant treatment was influenced by predicted risk of recurrence; among respondents with an estimated 2-year recurrence risk &gt; 10% and &gt; 30%, 40% and 56%, respectively, believed they should receive adjuvant therapy. Conclusions: A majority of patients surveyed received adjuvant therapy, the most common of which were systemic therapy and TACE. Patients who underwent resection had a strong desire to reduce postoperative recurrence by using adjuvant therapy. Physician opinion was identified as the most important factor in treatment decision making, followed by economic factors. When patients were the decision maker, the most influential factors were treatment effectiveness followed by reimbursement status.

The Expiry of Humira® Market Exclusivity and the Entry of Adalimumab Biosimilars in Europe: An Overview of Pricing and National Policy Measures.

Background: From October 2018, adalimumab biosimilars could enter the European market. However, in some countries, such as Netherlands, high discounts reported for the originator product may have influenced biosimilar entry. Objectives: The aim of this paper is to provide a European overview of (list) prices of originator adalimumab, before and after loss of exclusivity; to report changes in the reimbursement status of adalimumab products; and discuss relevant policy measures. Methods: Experts in European countries received a survey consisting of three parts: 1) general financing/co-payment of medicines, 2) reimbursement status and prices of originator adalimumab, and availability of biosimilars, and 3) policy measures related to the use of adalimumab. Results: In May 2019, adalimumab biosimilars were available in 24 of the 30 countries surveyed. Following introduction of adalimumab biosimilars, a number of countries have made changes in relation to the reimbursement status of adalimumab products. Originator adalimumab list prices varied between countries by a factor of 2.8 before and 4.1 after loss of exclusivity. Overall, list prices of originator adalimumab decreased after loss of exclusivity, although for 13 countries list prices were unchanged. When reported, discounts/rebates on originator adalimumab after loss of exclusivity ranged from 0% to approximately 26% (Romania), 60% (Poland), 80% (Denmark, Italy, Norway), and 80–90% (Netherlands), leading to actual prices per pen or syringe between €412 (Finland) and €50 – €99 (Netherlands). To leverage competition following entry of biosimilar adalimumab, only a few countries adopted measures specifically for adalimumab in addition to general policies regarding biosimilars. In some countries, a strategy was implemented even before loss of exclusivity (Denmark, Scotland), while others did not report specific measures. Conclusion: Even though originator adalimumab is the highest selling product in the world, few countries have implemented specific policies and practices for (biosimilar) adalimumab. Countries with biosimilars on the market seem to have competition lowering list or actual prices. Reported discounts varied widely between countries.

PMU44 Can Price-Prevalence Trends be Used to Guide the Pricing and Reimbursement Strategy for New Orphan Drugs? a Two-Country Case Study

Determinants of orphan drugs (ODs) pricing have been extensively investigated in several publications, with various studies identifying different relationships between the price of ODs and the prevalence of the indication. To summarise recent OD policy developments in England and Italy and analyse the correlation between annual treatment cost (ATC) and disease prevalence with a new dataset. HTA agency websites were reviewed to identify relevant OD policies. A five-step process was adopted to analyse the relationship between ATC and prevalence: (1) details of the regulatory approval (2006-2020) and reimbursement status of ODs in Italy and England were collected (2) ex-factory list prices for selected ODs were extracted from the IHS-Markit POLI database; (3) calculation of ATC; (4) extraction of disease prevalence from Orphanet, for multiple indications the most prevalent was reported; and (5) regression analysis of disease prevalence on ATC. The role of unmet need in oncology indications was investigated using 5-year overall survival (OS) data. AIFA introduced new mechanisms to balance between ensuring rapid access to OD for patients and improving the long-term sustainability of the system, whilst in England different routes to access reflect the historical differences in OD assessment and prioritisation. One hundred and sixteen ODs (89 indications) were identified. Regression analysis identified negative association between ATC and prevalence which was statistically significant consistently for Italy and England. Exploratory analyses identified weaker association between ATC and prevalence in oncology relative to non-oncology assets and non-significant association between OS and ATC for oncology assets. The weaker association for oncology ODs may be due to the limited duration of treatment and disease severity. A statistically significant negative association was established between ATC and prevalence for Italy and England. Further research is required into other factors such as disease severity, public pressure and available treatment alternatives.