Journal of Market Access & Health Policy

10 pages, 926 KiB

Open AccessArticle

Cost-Effectiveness of Evolocumab in Treatment of Heterozygous Familial Hypercholesterolaemia in Bulgaria: Measuring Health Benefit by Effectively Treated Patient-Years

by Borislav Borissov, Michael Urbich, Boryana Georgieva, Svetoslav Tsenov and Guillermo Villa

J. Mark. Access Health Policy 2017, 5(1), 1412753; https://doi.org/10.1080/20016689.2017.1412753 - 22 Dec 2017

Cited by 11 | Viewed by 252

Abstract

Background: An elevated level of low-density lipoprotein cholesterol (LDL-C) constitutes one of the most important modifiable risk factors for cardiovascular disease (CVD). Individuals with heterozygous familial hypercholesterolaemia (HeFH) are particularly vulnerable to CVD events. The addition of evolocumab to statins has shown marked [...] Read more.

Background: An elevated level of low-density lipoprotein cholesterol (LDL-C) constitutes one of the most important modifiable risk factors for cardiovascular disease (CVD). Individuals with heterozygous familial hypercholesterolaemia (HeFH) are particularly vulnerable to CVD events. The addition of evolocumab to statins has shown marked reductions in LDL-C levels. The objective of this analysis is to demonstrate the clinical and economic value of LDL-C lowering with evolocumab from the Bulgarian public health care perspective. Methods: A disease-specific measure of health benefit was devised: Effectively treated patient-years (ETPYs) combine length of life with the likelihood of attaining best-practice recommendations on LDL-C lowering. “Effective treatment” was defined as a reduction in LDL-C levels of ≥50%. A Markov cohort state-transition model was adapted, considering a life-long treatment duration. Demographics, baseline characteristics and efficacy data were taken from the RUTHERFORD-2 trial. The model uses the relationship between LDL-C lowering and reduced CVD event rates observed in the meta-analyses conducted by the Cholesterol Treatment Trialists’ Collaboration. Outcomes and costs (from year 2015) were discounted at an annual rate of 5%. Sensitivity analyses were conducted to assess uncertainty surrounding the results. Results: The total incremental costs of evolocumab added to statins versus statins alone are BGN 120,329 while adding 9.30 ETPYs over lifetime. These results imply an incremental cost per ETPY of BGN 12,937 (US$ 7,215; € 6,604). The use of evolocumab is associated with a relative reduction in the CVD event rate by 38% (18% per 1 mmol/L). Conclusions: Adding evolocumab to statins may be considered cost-effective in light of an additional expense per patient-year gained in which individuals with HeFH receive effective treatment under the terms of international prevention guidelines. ETPYs are an intuitive and clinically meaningful measure of patient benefit that, in relation to costs, can support health care decision-making that considers quality of care. Full article

8 pages, 842 KiB

Open AccessArticle

Cost-Effectiveness Analysis of Ibrutinib in Patients with Waldenström Macroglobulinemia in Italy

by Andrea Aiello, Anna D’Ausilio, Roberta Lo Muto, Francesca Randon and Luca Laurenti

J. Mark. Access Health Policy 2017, 5(1), 1393308; https://doi.org/10.1080/20016689.2017.1393308 - 7 Nov 2017

Cited by 1 | Viewed by 210

Abstract

Background and Objective: Ibrutinib has recently been approved in Europe for Waldenström Macroglobulinemia (WM) in symptomatic patients who have received at least one prior therapy, or in first-line treatment for patients unsuitable for chemo-immunotherapy. The aim of the study is to estimate the [...] Read more.

Background and Objective: Ibrutinib has recently been approved in Europe for Waldenström Macroglobulinemia (WM) in symptomatic patients who have received at least one prior therapy, or in first-line treatment for patients unsuitable for chemo-immunotherapy. The aim of the study is to estimate the incremental cost-effectiveness ratio (ICER) of ibrutinib in relapse/refractory WM, compared with the Italian current therapeutic pathways (CTP). Methods: A Markov model was adapted for Italy considering the National Health System perspective. Input data from literature as well as global trials were used. The percentage use of therapies, and healthcare resources consumption were estimated according to expert panel advice. Drugs ex-factory prices and national tariffs were used for estimating costs. The model had a 15-year time horizon, with a 3.0% discount rate for both clinical and economic data. Deterministic and probabilistic sensitivity analyses were performed to test the results strength. Results: Ibrutinib resulted in increased Life Years Gained (LYGs) and increased costs compared to CTP, with an ICER of €52,698/LYG. Sensitivity analyses confirmed the results of the BaseCase. Specifically, in the probabilistic analysis, at a willingness to pay threshold of €60,000/LYG ibrutinib was cost-effective in 84% of simulations. Conclusions: Ibrutinib has demonstrated a positive cost-effectiveness profile in Italy. Full article

10 pages, 935 KiB

Open AccessEssay

Utilization of Multiple-Criteria Decision Analysis (MCDA) to Support Healthcare Decision-Making FIFARMA, 2016

by Julia I. Drake, Juan Carlos Trujillo de Hart, Clara Monleón, Walter Toro and Joice Valentim

J. Mark. Access Health Policy 2017, 5(1), 1360545; https://doi.org/10.1080/20016689.2017.1360545 - 12 Oct 2017

Cited by 42 | Viewed by 308

Abstract

Background and objectives: MCDA is a decision-making tool with increasing use in the healthcare sector, including HTA (Health Technology Assessment). By applying multiple criteria, including innovation, in a comprehensive, structured and explicit manner, MCDA fosters a transparent, participative, consistent decision-making process taking into [...] Read more.

Background and objectives: MCDA is a decision-making tool with increasing use in the healthcare sector, including HTA (Health Technology Assessment). By applying multiple criteria, including innovation, in a comprehensive, structured and explicit manner, MCDA fosters a transparent, participative, consistent decision-making process taking into consideration values of all stakeholders. This paper by FIFARMA (Latin American Federation of Pharmaceutical Industry) proposes the deliberative (partial) MCDA as a more pragmatic, agile approach, especially when newly implemented. Methods: Literature review including real-world examples of effective MCDA implementation in healthcare decision making in both the public and private sector worldwide and in LA. Results and conclusion: It is the view of FIFARMA that MCDA should strongly be considered as a tool to support HTA and broader healthcare decision making such as the contracts and tenders process in order to foster transparency, fairness, and collaboration amongst stakeholders. Full article

11 pages, 917 KiB

Open AccessEssay

Proposed Changes to the Reimbursement of Pharmaceuticals and Medical Devices in Poland and Their Impact on Market Access and the Pharmaceutical Industry

by Karolina Badora, Aleksandra Caban, Cécile Rémuzat, Claude Dussart and Mondher Toumi

J. Mark. Access Health Policy 2017, 5(1), 1381544; https://doi.org/10.1080/20016689.2017.1381544 - 25 Sep 2017

Cited by 3 | Viewed by 273

Abstract

In Poland, two proposed amendments to the reimbursement act are currently in preparation; these are likely to substantially change the pricing and reimbursement landscape for both drugs and medical devices. Proposed changes include: alignment of medical device reimbursement with that of pharmaceuticals; relaxing [...] Read more.

In Poland, two proposed amendments to the reimbursement act are currently in preparation; these are likely to substantially change the pricing and reimbursement landscape for both drugs and medical devices. Proposed changes include: alignment of medical device reimbursement with that of pharmaceuticals; relaxing the strict reimbursement criteria for ultra-orphan drugs; establishment of an additional funding category for vaccines; introduction of compassionate use, and a simplified reimbursement pathway for well-established off-label indications; appreciation of manufacturers’ innovation and research and development efforts by creating a dedicated innovation budget; introduction of a mechanism preventing excessive parallel import; prolonged duration of reimbursement decisions and reimbursement lists; and increased flexibility in defining drug programmes. Both amendments are still at a draft stage and many aspects of the new regulations remain unclear. Nonetheless, the overall direction of some of the changes is already evident and warrants discussion due to their high expected impact on pharmaceutical and device manufacturers. Here we evaluate the main changes proposed to the reimbursement of drugs, vaccines, and medical devices, and examine the impact they are likely to have on market access and pharmaceutical industry in Poland. Full article

10 pages, 1070 KiB

Open AccessArticle

A Tolerability Burden Index in Schizophrenia: Incorporating Patient Perspective in Clinical Trial Adverse Event Reporting

by Clément François, Alice Guiraud-Diawara, Christophe Lançon, Pierre Michel Llorca, Ann Hartry, Lene Hammer-Helmich, Djamel A. Zighed, Adrian Tanasescu and Mondher Toumi

J. Mark. Access Health Policy 2017, 5(1), 1372026; https://doi.org/10.1080/20016689.2017.1372026 - 13 Sep 2017

Cited by 2 | Viewed by 278

Abstract

Background: Adverse event (AE) reporting in clinical trials does not fully capture the patient-level perspective and comparing tolerability across treatments or among studies is difficult. Objective: This study was designed to develop a treatment tolerability index algorithm that combines AE reporting with physician- [...] Read more.

Background: Adverse event (AE) reporting in clinical trials does not fully capture the patient-level perspective and comparing tolerability across treatments or among studies is difficult. Objective: This study was designed to develop a treatment tolerability index algorithm that combines AE reporting with physician- and patient-level AE information into a global burden score to allow comparison of the overall tolerability of antipsychotic medications used in treating schizophrenia. Study design: Data from a 4-arm, placebo-controlled clinical trial were used in the proposed tolerability index algorithm. For each patient, AEs were adjusted by frequency, severity, duration, and patient-experienced importance, and average tolerability-related burden scores were calculated. Setting: Algorithm development analyses. Patients: This study analyzed data from a previously completed clinical trial that evaluated a potential antipsychotic medication; no patients were involved in the current study. Intervention: No interventions were administered in this study; the analyses described used data derived from a previously completed clinical trial in which patients received bifeprunox, risperidone, or placebo. Main outcome measure: Burden scores and tolerability index scores were compared for patients who did or did not discontinue treatment because of AEs. Results: The number of AEs varied widely among patients. Burden scores were significantly worse for patients who discontinued treatment because of AEs. Mean tolerability index scores, adjusted based on AE frequency, severity-adjusted duration, and patient-experienced impact, were poorer for active medications than placebo, and increased with dose. Conclusion: The treatment tolerability index will allow comparison of AE burden and tolerability between treatments using existing clinical trial information. This suggests that scoring is possible, is clinically relevant, and allows standardized comparison of antipsychotic tolerability. Full article

11 pages, 1276 KiB

Open AccessArticle

Creating an Index to Measure Health State of Depressed Patients in Automated Healthcare Databases: The Methodology

by Clément François, Adrian Tanasescu, François-Xavier Lamy, Nicolas Despiegel, Bruno Falissard, Ylana Chalem, Christophe Lançon, Pierre-Michel Llorca, Delphine Saragoussi, Patrice Verpillat, Alan G. Wade and Djamel A. Zighed

J. Mark. Access Health Policy 2017, 5(1), 1372025; https://doi.org/10.1080/20016689.2017.1372025 - 13 Sep 2017

Cited by 3 | Viewed by 207

Abstract

Background and objective: Automated healthcare databases (AHDB) are an important data source for real life drug and healthcare use. In the filed of depression, lack of detailed clinical data requires the use of binary proxies with important limitations. The study objective was to [...] Read more.

Background and objective: Automated healthcare databases (AHDB) are an important data source for real life drug and healthcare use. In the filed of depression, lack of detailed clinical data requires the use of binary proxies with important limitations. The study objective was to create a Depressive Health State Index (DHSI) as a continuous health state measure for depressed patients using available data in an AHDB. Methods: The study was based on historical cohort design using the UK Clinical Practice Research Datalink (CPRD). Depressive episodes (depression diagnosis with an antidepressant prescription) were used to create the DHSI through 6 successive steps: (1) Defining study design; (2) Identifying constituent parameters; (3) Assigning relative weights to the parameters; (4) Ranking based on the presence of parameters; (5) Standardizing the rank of the DHSI; (6) Developing a regression model to derive the DHSI in any other sample. Results: The DHSI ranged from 0 (worst) to 100 (best health state) comprising 29 parameters. The proportion of depressive episodes with a remission proxy increased with DHSI quartiles. Conclusion: A continuous outcome for depressed patients treated by antidepressants was created in an AHDB using several different variables and allowed more granularity than currently used proxies. Full article

9 pages, 1029 KiB

Open AccessArticle

Healthcare Resource Use in Schizophrenia, EuroSC Findings

by A. Millier, M. Horváth, F. Ma, K. Kóczián, A. Götze and M. Toumi

J. Mark. Access Health Policy 2017, 5(1), 1372027; https://doi.org/10.1080/20016689.2017.1372027 - 5 Sep 2017

Cited by 8 | Viewed by 253

Abstract

Background: It is unclear if the burden associated with schizophrenia is affected by the type and severity of patient’s symptoms. Objective: This study aims to quantify healthcare resource use associated with different profiles of schizophrenia symptoms. Study design: Post-hoc analysis of data from [...] Read more.

Background: It is unclear if the burden associated with schizophrenia is affected by the type and severity of patient’s symptoms. Objective: This study aims to quantify healthcare resource use associated with different profiles of schizophrenia symptoms. Study design: Post-hoc analysis of data from a naturalistic follow-up study. Setting: Secondary psychiatric services in France, Germany and the UK. Patients: EuroSC cohort:, representative sample of 1,208 schizophrenia patients. Main outcome measure: We classified patients into eight health states, according to the Lenert classification (HS1–HS8), and estimated 6-month healthcare resource use (outpatient and day clinic visits, and hospitalisations) across the health states. Results: Approximately half of the patients were classed as having mild symptoms (HS1), with around 20% experiencing moderate, predominantly negative symptoms (HS2). The remaining health states were represented by <10% of patients each. Very few patients experienced extremely severe symptoms (HS8). No health state was associated with excess utilisation across all resource types. In terms of outpatient visits, patients were estimated to see a psychiatrist most often (3.01–4.15 visits over 6 months). Hospital admission was needed in 11%(HS1) – 35%(HS8) of patients and inpatient stays were generally prolonged for all health states (39–57 days). The average number of inpatient days was highest for patients in HS8 (18.17 days), followed by patients with severe negative symptoms (HS4; 13.37 days). In other health states characterised by severe symptoms (HS5–HS7), the average number of inpatient days was approximately half of those seen for HS4 (6.09–7.66). Conclusion: While none of the symptom profiles was associated with excess resource usage, hospitalization days were highest for HS with severe, predominantly negative or extremely severe symptoms. Patients with predominantly negative, moderate or severe symptoms appeared to have a high number of psychologist visits – an interesting finding that may reflect a specific therapeutic approach to the treatment of these patients. Full article

11 pages, 1319 KiB

Open AccessArticle

The Perverse Impact of External Reference Pricing (ERP): A Comparison of Orphan Drugs Affordability in 12 European Countries. A Call for Policy Change

by K. E. Young, I. Soussi and M. Toumi

J. Mark. Access Health Policy 2017, 5(1), 1369817; https://doi.org/10.1080/20016689.2017.1369817 - 3 Sep 2017

Cited by 23 | Viewed by 290

Abstract

Objective: The study compared the relative cost differences of similar orphan drugs among high and low GDP countries in Europe: Bulgaria, France, Germany, Greece, Hungary, Italy, Norway, Poland, Romania, Spain, Sweden, UK. Methods: Annual treatment costs per patient were calculated. Relative costs were [...] Read more.

Objective: The study compared the relative cost differences of similar orphan drugs among high and low GDP countries in Europe: Bulgaria, France, Germany, Greece, Hungary, Italy, Norway, Poland, Romania, Spain, Sweden, UK. Methods: Annual treatment costs per patient were calculated. Relative costs were computed by dividing the costs by each economic parameter: nominal GDP per capita, GDP in PPP per capita, % GDP contributed by the government, government budget per inhabitant, % GDP spent on healthcare, % GDP spent on pharmaceuticals, and average annual salary. An international comparison of the relative costs was done using UK as the reference country and results were analysed descriptively. Results: 120 orphan drugs were included. The median annual costs of orphan drugs in all countries varied minimally (cost ratios: 0.87 to 1.08). When the costs were adjusted using GDP per capita, the EU-5 and Nordic countries maintained minimal difference in median cost. However, the lower GDP countries showed three to six times higher relative costs. The same pattern was evident when costs were adjusted using the other economic parameters. Conclusion: When the country’s ability to pay is taken into consideration, lower GDP countries pay relatively higher costs for similarly available orphan drugs in Europe. Full article

16 pages, 1720 KiB

Open AccessReview

3. How Comprehensive Can We Be in the Economic Assessment of Vaccines?

by Baudouin Standaert and Rino Rappuoli

J. Mark. Access Health Policy 2017, 5(1), 1336044; https://doi.org/10.1080/20016689.2017.1336044 - 31 Aug 2017

Cited by 11 | Viewed by 271

Abstract

In two previous papers we argued on current vaccines economic assessment not fully comprehensive when using the incremental cost-utility analysis normally applied for treatments. Many differences exist between vaccines and drug treatments making vaccines economic evaluation more cumbersome. Four challenges overwhelmingly present in [...] Read more.

In two previous papers we argued on current vaccines economic assessment not fully comprehensive when using the incremental cost-utility analysis normally applied for treatments. Many differences exist between vaccines and drug treatments making vaccines economic evaluation more cumbersome. Four challenges overwhelmingly present in vaccines assessment are less important for treatments: requirements for population, societal perspectives, budget impact evaluation, and time focused objectives (control or elimination). Based on this, economic analysis of vaccines may need to be presented to many different stakeholders with various evaluation preferences, in addition to the current stakeholders involved for drugs treatment assessment. Then, we may need a tool making the inventory of the different vaccines health economic assessment programmes more comprehensive. The cauliflower value toolbox has been developed with that aim, and its use is illustrated here with rotavirus vaccine. Given the broader perspectives for vaccine assessment, it provides better value and cost evaluations. Cost-benefit analysis may be the preferred economic assessment method when considering substitution from treatment to active medical prevention. Other economic evaluation methods can be selected (i.e. optimisation modelling, return on investment) when project prioritisation is the main focus considered and when stakeholders would like to influence the development of the healthcare programme. Full article

14 pages, 1487 KiB

Open AccessReview

2. How Is the Economic Assessment of Vaccines Performed Today?

by Baudouin Standaert and Rino Rappuoli

J. Mark. Access Health Policy 2017, 5(1), 1335163; https://doi.org/10.1080/20016689.2017.1335163 - 31 Aug 2017

Cited by 11 | Viewed by 219

Abstract

This paper describes how the economic assessment of vaccines is performed today. It discusses why it may be incomplete and explores potential approaches to adjust the analysis to be more comprehensive. Besides helping protect against serious disease, vaccines also help avoid mild disease [...] Read more.

This paper describes how the economic assessment of vaccines is performed today. It discusses why it may be incomplete and explores potential approaches to adjust the analysis to be more comprehensive. Besides helping protect against serious disease, vaccines also help avoid mild disease episodes that may not receive medical attention but which have important societal consequences. They also benefit unvaccinated individuals by reducing disease transmission. Wider societal benefits may extend beyond a decrease in disease incidence, as lower transmission rates reduce the risk of epidemics, which in turn reduces the pressure on healthcare providers, and may improve the quality of care for patients with unrelated diseases. Vaccines also lower the use of antibiotics leading to less pressure on anti-microbial resistance. Conventional ICUA focuses on individual health benefits, like increased survival. Therefore, this approach may not adequately capture the wider vaccination benefits. We discuss differences between treatment and vaccine prevention in the economic assessment, and how ICUA has been adapted to cope with the inconsistencies. Although such adaptations may fulfil the demand of one specific stakeholder, they may not meet the needs of other stakeholders who operate at the societal level, such as ministries other than healthcare, employers, caregivers, and insurers. Full article

15 pages, 1530 KiB

Open AccessArticle

Towards a More Comprehensive Approach for a Total Economic Assessment of Vaccines?

by Baudouin Standaert and Rino Rappuoli

J. Mark. Access Health Policy 2017, 5(1), 1335162; https://doi.org/10.1080/20016689.2017.1335162 - 31 Aug 2017

Cited by 19 | Viewed by 268

Abstract

Since we were born, we all take preventative actions to avoid unpredictable adverse conditions. Some actions are done automatically. Others require a conscious choice , either for personal or social benefit. A distinction can therefore be drawn between non-active and active prevention, and [...] Read more.

Since we were born, we all take preventative actions to avoid unpredictable adverse conditions. Some actions are done automatically. Others require a conscious choice , either for personal or social benefit. A distinction can therefore be drawn between non-active and active prevention, and between individual and social prevention. Active prevention requires making a choice in time, effort, and cost. We call it an economic choice. Vaccines belong to the group of active and social prevention. Because a vaccination program is an economic social choice, how should it be valued, and what cost should we pay for? To date, the economic evaluations developed for treatment have been applied to vaccines. However, over 25 different characteristics differentiate vaccines from treatment. For example, the benefit of vaccination is measured at the population level not at the individual level, the main effect of prevention is societal and not an individual-based gain only, and the biggest hurdle to implement a new vaccine is the initial budget investment and not so much its estimated ‘value for money’. This makes the current application of incremental cost-utility analysis difficult for vaccines for a comprehensive evaluation. New approaches may be needed to capture the full economic benefit of vaccines. Full article

9 pages, 972 KiB

Open AccessArticle

Development of an Instrument Evaluating the Impact of Surgeon-Patient Relationship in Patients on Sick Leave

by Thierry Dubert, Cedric Girault, Alexandre Kilink, Marc Rozenblat, Yves Lebellec, Anne-Lise Vataire, Myriam Vilasco and Gregory Katz

J. Mark. Access Health Policy 2017, 5(1), 1345586; https://doi.org/10.1080/20016689.2017.1345586 - 11 Aug 2017

Cited by 3 | Viewed by 211

Abstract

Background: To date, no specific instruments exist to measure the quality of the patient-surgeon relationship despite its potential to influence clinical and economic outcomes in patients undergoing surgery for musculoskeletal disorders (MSDs). Objective: The objective was to develop and validate an instrument to [...] Read more.

Background: To date, no specific instruments exist to measure the quality of the patient-surgeon relationship despite its potential to influence clinical and economic outcomes in patients undergoing surgery for musculoskeletal disorders (MSDs). Objective: The objective was to develop and validate an instrument to assess the quality of the patient-surgeon relationship, taking into account the return to work after functional restoration surgery. Methods: The instrument development was based on literature review, cognitive interviews and expert examinations. The instrument’s psychometric properties were explored in a sample of 50 French patients on sick leave with musculoskeletal disorders or hand injuries. Face validity, internal consistency and test-retest reliability were evaluated. The dimensionality of the instrument was studied using an exploratory principal component analysis. Results: The 11-item instrument showed good psychometric properties. The cognitive interviews allowed enhancing the validity of the instrument content by capturing patients’ point of view. The exploratory principal component analysis demonstrated the uni-dimensionality of the instrument with the first factor accounting for 83% of the total explained variance. Conclusion:This study has developed the first instrument capable of the specific assessment of the impact of the surgeon-patient relationship on recovery, in patients with hand traumas and MSDs. Full article

12 pages, 930 KiB

Open AccessReview

A Comparative Review of Haute Autorité de Santé and National Institute for Health and Care Excellence Health Technology Assessments of Ikervis^® to Treat Severe Keratitis in Adult Patients with Dry Eye Disease Which Has Not Improved despite Treatment with Tear Substitutes

by Yasmina Iffet Eroglu

J. Mark. Access Health Policy 2017, 5(1), 1336043; https://doi.org/10.1080/20016689.2017.1336043 - 3 Aug 2017

Cited by 18 | Viewed by 342

Abstract

Background: In 2015, Ikervis® became the only EMA-approved cyclosporine A (CsA) eye-drop for the treatment of severe keratitis in adult patients with dry eye disease, which has not improved despite treatment with tear substitutes. Since the 1980s, CsA has been used empirically [...] Read more.

Background: In 2015, Ikervis® became the only EMA-approved cyclosporine A (CsA) eye-drop for the treatment of severe keratitis in adult patients with dry eye disease, which has not improved despite treatment with tear substitutes. Since the 1980s, CsA has been used empirically for ocular conditions in veterinary medicine then in humans. However, its extremely low aqueous solubility led to its administration in vegetable oils, which is characterized by low ocular availability, poor intraocular penetration, poor tolerability and short shelf-life. Concentrations from 0.05% to 2% are compounded on an industrial scale and reimbursed throughout Europe. In France, Ikervis® has been granted an ASMR score of 5 by HAS, whereas in UK NICE endorsed its use. Objective: To review the dry eye disease environment, its challenges and available treatment options, and compare the NICE and HAS assessments to question HAS’ decision to maintain full reimbursement of compounded CsA formulations in the absence of evidence, while reimbursing the EMA-approved drug at 15%. Method: extensive search on PubMED. Results: Comparator selection, composite score assessment and use of CE model are key differentiators. Conclusion: In topical formulations, improvements to the vehicle are key innovations that can bring significant benefits. After the USA, a Compounding Act is needed in Europe. Full article

10 pages, 996 KiB

Open AccessArticle

Annuity Payments Can Increase Patient Access to Innovative Cell and Gene Therapies under England’s Net Budget Impact Test

by Jesper Jørgensen and Panos Kefalas

J. Mark. Access Health Policy 2017, 5(1), 1355203; https://doi.org/10.1080/20016689.2017.1355203 - 31 Jul 2017

Cited by 40 | Viewed by 285

Abstract

Background: Cell and gene therapies have the potential to provide therapeutic breakthroughs, but the high costs of researching, developing, manufacturing and delivering them translate into prices that may challenge healthcare budgets. Various measures exist that aim to address the affordability challenge, including reducing [...] Read more.

Background: Cell and gene therapies have the potential to provide therapeutic breakthroughs, but the high costs of researching, developing, manufacturing and delivering them translate into prices that may challenge healthcare budgets. Various measures exist that aim to address the affordability challenge, including reducing price, limiting patient numbers and/or linking remuneration to product performance. Objective: To explore how the net budget impact test recently introduced in England can affect patient access to high-value, one-off cell and gene therapies, and how managed entry agreements can improve access. Methods: We use a hypothetical example where a new high-value, one-off therapy launches in an indication where it displaces a relatively low cost chronic treatment. We calculate the number of patients that can be treated without exceeding the £20 million net budget impact threshold, and compare results for scenarios where a full upfront payment is used, and where annuity-based payments are used. Results: Charging a full upfront payment at the time of treatment can lead to suboptimal patient access. Conclusion: Annuity-based payments in combination with an outcomes-based remuneration scheme reduce consequences of decision uncertainty and can increase patient access, without exceeding the net budget impact test. Full article

8 pages, 780 KiB

Open AccessArticle

Towards a More Transparent HTA Process in Poland: New Polish HTA Methodological Guidelines

by Krzysztof Lach, Michal Dziwisz, Cécile Rémuzat and Mondher Toumi

J. Mark. Access Health Policy 2017, 5(1), 1355202; https://doi.org/10.1080/20016689.2017.1355202 - 26 Jul 2017

Cited by 3 | Viewed by 236

Abstract

Introduction: Health technology assessment (HTA) in Poland supports reimbursement decisions via the Polish HTA Agency (AOTMiT), whose guidelines were updated in 2016. Methods: We identified key changes introduced by the update and, before guideline publication, analysed discrepancies between AOTMiT assessments and the submitting [...] Read more.

Introduction: Health technology assessment (HTA) in Poland supports reimbursement decisions via the Polish HTA Agency (AOTMiT), whose guidelines were updated in 2016. Methods: We identified key changes introduced by the update and, before guideline publication, analysed discrepancies between AOTMiT assessments and the submitting marketing authorisation holders (MAHs) to elucidate the context of the update. We compared the clarity and detail of the new guidelines versus those of the UK’s National Institute for Health and Care Excellence (NICE). Results: The update specified more precise requirements for items such as indirect comparison or input data for economic modelling. Agency–MAH discrepancies relating to the subjects of the HTA update were found in 14.6% of published documents. The new Polish HTA guidelines were as clear and detailed as NICE’s on topics such as assessing quality of evidence and economic modelling, but were less informative when describing (for example) pairwise meta-analysis. Conclusions: The Polish HTA guidelines update demonstrates lessons learned from internal and external experiences. The new guidelines adhere more closely to UK HTA standards, being clearer and more informative. While the update is expected to reduce Agency–MAH discrepancies, there remain areas for development, such as providing templates to aid HTA submissions. Full article

8 pages, 862 KiB

Open AccessArticle

Balanced Assessment Systems Revisited

by Dávid Dankó and Márk Péter Molnár

J. Mark. Access Health Policy 2017, 5(1), 1355190; https://doi.org/10.1080/20016689.2017.1355190 - 26 Jul 2017

Cited by 10 | Viewed by 232

Abstract

In 2014, balanced assessment systems (BAS) were proposed as a resource-conscious, ‘fit-for-purpose’ form of health technology assessment for middle-income countries which lack resources and competences necessary for resource-intensive health technology assessment models. BAS has undergone extensive policy debate in the period since its [...] Read more.

In 2014, balanced assessment systems (BAS) were proposed as a resource-conscious, ‘fit-for-purpose’ form of health technology assessment for middle-income countries which lack resources and competences necessary for resource-intensive health technology assessment models. BAS has undergone extensive policy debate in the period since its publication but it has not been critically assessed in a structured form yet. This article aims to describe both the contributions and the weak spots of the original framework and to reflect on them with the intention of further developing the model. Full article

10 pages, 1336 KiB

Open AccessArticle

Policy Implementation for Methicillin-Resistant Staphylococcus aureus in Seven European Countries: A Comparative Analysis from 1999 to 2015

by Takuya Kinoshita, Hironobu Tokumasu, Shiro Tanaka, Axel Kramer and Koji Kawakami

J. Mark. Access Health Policy 2017, 5(1), 1351293; https://doi.org/10.1080/20016689.2017.1351293 - 26 Jul 2017

Cited by 7 | Viewed by 204

Abstract

Background: Policies to reduce methicillin-resistant Staphylococcus aureus (MRSA) infections, both healthcare-acquired (HA-MRSA) and livestock-associated (LA-MRSA) are implemented Europe-wide, but evaluations are difficult for countries yet to implement such policies. A descriptive study was conducted, describing multinational MRSA rates and policy implementation, focusing on [...] Read more.

Background: Policies to reduce methicillin-resistant Staphylococcus aureus (MRSA) infections, both healthcare-acquired (HA-MRSA) and livestock-associated (LA-MRSA) are implemented Europe-wide, but evaluations are difficult for countries yet to implement such policies. A descriptive study was conducted, describing multinational MRSA rates and policy implementation, focusing on MRSA mandatory surveillance. We also investigated antibiotic use and MRSA rates and the use of veterinary antibiotics. Methods: This study used Europe-wide surveillance data on infectious diseases (EARS-Net), antibiotic consumption (ESAC-Net), and veterinary medicine (ESVAC). We visualized LA- and HA-MRSA related policies and MRSA rates from 1999 to 2015 in seven European countries. Changes in MRSA rates after implementation of an MRSA mandatory surveillance policy were investigated by setting each country as rate of 1.0 and compared countries with and without such policy. Correlations between antibiotic use and MRSA rates from 1999 to 2012 were investigated using defined daily dose. Sales data were used to investigate veterinary antibiotic use. Results: MRSA rates were 1–45.4% across the seven countries between 1999 and 2015. MRSA rates changed between 0.61 and 0.24 after the implementation of mandatory surveillance policies within a 6–12 year span. The rate of decrease rate in implemented and non-implemented countries ranged from 10% in Spain to 76% in the UK. The correlation between MRSA rate and cephalosporin consumption was r = 0.419, and for fluoroquinolones r = 0.305. Mean annual sales of veterinary cephalosporin and quinolone antibiotics were lowest in the UK (0.8 mg/PCU) and highest in Spain (9.7 mg/PCU) between 2009 and 2014. Conclusions: There were similar but different health policy implications in the seven countries regarding LA- and HA-MRSA. Although causation could not be defined, some policies such as mandatory surveillance may be helpful for countries that have yet to implement an MRSA policy. Further investigations are needed to evaluate each policies. Full article

11 pages, 1282 KiB

Open AccessArticle

Analysis of Health Economics Assessment Reports for Pharmaceuticals in France—Understanding the Underlying Philosophy of CEESP Assessment

by Mondher Toumi, Anastasiia Motrunich, Aurélie Millier, Cécile Rémuzat, Christos Chouaid, Bruno Falissard and Samuel Aballéa

J. Mark. Access Health Policy 2017, 5(1), 1344088; https://doi.org/10.1080/20016689.2017.1344088 - 16 Jul 2017

Cited by 20 | Viewed by 250

Abstract

Background: Despite the guidelines for Economic and Public Health Assessment Committee (CEESP) submission having been available for nearly six years, the dossiers submitted continue to deviate from them, potentially impacting product prices. Objective: to review the reports published by CEESP, analyse deviations from [...] Read more.

Background: Despite the guidelines for Economic and Public Health Assessment Committee (CEESP) submission having been available for nearly six years, the dossiers submitted continue to deviate from them, potentially impacting product prices. Objective: to review the reports published by CEESP, analyse deviations from the guidelines, and discuss their implications for the pricing and reimbursement process. Study design: CEESP reports published until January 2017 were reviewed, and deviations from the guidelines were extracted. The frequency of deviations was described by type of methodological concern (minor, important or major). Results: In 19 reports, we identified 243 methodological concerns, most often concerning modelling, measurement and valuation of health states and results presentation and sensitivity analyses; nearly 63% were minor, 33% were important and 4.5% were major. All reports included minor methodological concerns, and 17 (89%) included at least one important and/or major methodological concern. Global major methodological concerns completely invalidated the analysis in seven dossiers (37%). Conclusion: The CEESP submission dossiers fail to adhere to the guidelines, potentially invalidating the health economics analysis and resulting in pricing negotiations. As these negotiations tend to be unfavourable for the manufacturer, the industry should strive to improve the quality of the analyses submitted to CEESP. Full article

8 pages, 1077 KiB

Open AccessArticle

Patient Access to Reimbursed Biological Disease-Modifying Antirheumatic Drugs in the European Region

by Zoltán Kaló, Zoltán Vokó, Andrew Östör, Emma Clifton-Brown, Radu Vasilescu, Alysia Battersby and Edward Gibson

J. Mark. Access Health Policy 2017, 5(1), 1345580; https://doi.org/10.1080/20016689.2017.1345580 - 5 Jul 2017

Cited by 37 | Viewed by 297

Abstract

Background & Objectives: Biological disease-modifying antirheumatic drugs (bDMARDs) for the treatment of rheumatoid arthritis (RA) are not always accessible to all patients in accordance with international guidelines, partly owing to their high direct costs against a background of restricted healthcare budgets. This study [...] Read more.

Background & Objectives: Biological disease-modifying antirheumatic drugs (bDMARDs) for the treatment of rheumatoid arthritis (RA) are not always accessible to all patients in accordance with international guidelines, partly owing to their high direct costs against a background of restricted healthcare budgets. This study compares the size of RA patient populations with access to reimbursed bDMARDs across 37 European countries, Russia, and Turkey, according to their treatment eligibility defined by European League Against Rheumatism (EULAR) recommendations and national reimbursement criteria. Methods: The size of the RA patient population eligible for bDMARD treatment was estimated in a population model using published RA epidemiological data and clinical criteria defined by 2013 EULAR recommendations along with national reimbursement criteria defined in a survey of the 39 countries in November 2015. Results: According to EULAR recommendations, 32% of the total RA population in the European region is eligible for bDMARD treatment. However, only an average 59% of this EULAR-eligible population remains eligible after applying national reimbursement criteria (from 86% in ‘high access’ to 13% in ‘low-access’ countries). Conclusion: Access to reimbursed bDMARDs remains unequal in the European region. As biosimilars of bDMARDs are introduced, changes in reimbursement criteria may increase access to bDMARDs and reduce this inequality. Full article

7 pages, 821 KiB

Open AccessArticle

Drug Pricing and Reimbursement Information Management: Processes and Decision Making in the Global Economy

by Dimitrios Tsourougiannis

J. Mark. Access Health Policy 2017, 5(1), 1340747; https://doi.org/10.1080/20016689.2017.1340747 - 30 Jun 2017

Viewed by 479

Abstract

Background: Cost-containment initiatives are re-shaping the pharmaceutical business environment and affecting market access as well as pricing and reimbursement decisions. Effective price management procedures are too complex to accomplish manually. Prior to February 2013, price management within Astellas Pharma Europe Ltd was done [...] Read more.

Background: Cost-containment initiatives are re-shaping the pharmaceutical business environment and affecting market access as well as pricing and reimbursement decisions. Effective price management procedures are too complex to accomplish manually. Prior to February 2013, price management within Astellas Pharma Europe Ltd was done manually using an Excel database. The system was labour intensive, slow to update, and prone to error. An innovative web-based pricing information management system was developed to address the shortcomings of the previous system. Development: A secure web-based system for submitting, reviewing and approving pricing requests was designed to: Track all pricing applications and approval status; update approved pricing information automatically; provide fixed and customizable reports of pricing information; collect pricing and reimbursement rules from each country; validate pricing and reimbursement rules monthly. Several sequential phases of development emphasized planning, time schedules, target dates, budgets and implementation of the entire system. A test system was used to pilot the electronic (e)-pricing system with three affiliates (four users) in February 2013. Outcomes: The web-based system was introduced in March 2013, currently has about 227 active users globally and comprises more than 1000 presentations of 150 products. The overall benefits of switching from a manual to an e-pricing system were immediate and highly visible in terms of efficiency, transparency, reliability and compliance. Conclusions: The e-pricing system has improved the efficiency, reliability, compliance, transparency and ease of access to multinational drug pricing and approval information. Full article

5 pages, 640 KiB

Open AccessArticle

Direct Cost Analysis of the Second Year Post-allogeneic Hematopoietic Stem Cell Transplantation in the Bone Marrow Transplant Centre of Tunisia

by Myriam Razgallah Khrouf, Leila Achour, Asma Thabti, Mohamed Ali Soussi, Nour Abdejelil, Olfa Lazreg, Chema Drira, Aida Zahaf, Saloua Ladab and Tarek Ben Othman

J. Mark. Access Health Policy 2017, 5(1), 1335161; https://doi.org/10.1080/20016689.2017.1335161 - 15 Jun 2017

Cited by 4 | Viewed by 247

Abstract

Background: Hematopoietic stem cell transplantation (HSCT) is a medically complicated therapy with a long recovery time. In Tunisia, the National Health Insurance Fund (CNAM) covers only the first year post-transplantation, after which the costs are borne by the hospital. Objective: Describe [...] Read more.

Background: Hematopoietic stem cell transplantation (HSCT) is a medically complicated therapy with a long recovery time. In Tunisia, the National Health Insurance Fund (CNAM) covers only the first year post-transplantation, after which the costs are borne by the hospital. Objective: Describe complications that can occur during the second year post-allogeneic HSCT and calculate direct costs in different groups of patients. Methods: In this pharmacoeconomic study, medical records of the second year post-allogeneic HSCT were collected. Studied variables included frequent observed complications and medical and non-medical direct costs. Results: The average total direct cost in the population during the second year post-transplantation was $11,571, 97% of which represents direct medical costs Drugs accounted for the largest share (80%) of total direct costs, dominated by the cost of antifungals (52%) and antivirals (26%) drug . Cytomegalovirus status was seen in 9.3% of patients and was associated with a seven-fold increase in direct costs (p < 0.001). In patients who developed chronic GVHD, the average direct cost was three times higher than for those who did not (p = 0.032). Conclusion: Given the importance of direct costs in the post-transplantation period a review of the hospital financing mechanism and a new convention with the CNAM is crucial. Full article

16 pages, 1469 KiB

Open AccessArticle

Use and Cost Comparison of Clobazam to Other Antiepileptic Drugs for Treatment of Lennox-Gastaut Syndrome

by Clément François, John M. Stern, Augustina Ogbonnaya, Tasneem Lokhandwala, Pamela Landsman-Blumberg, Amy Duhig, Vivienne Shen and Robin Tan

J. Mark. Access Health Policy 2017, 5(1), 1318691; https://doi.org/10.1080/20016689.2017.1318691 - 19 May 2017

Cited by 8 | Viewed by 325

Abstract

Background: Lennox-Gastaut syndrome (LGS) is a severe form of childhood-onset epilepsy associated with serious injuries due to frequent and severe seizures. Of the antiepileptic drugs (AEDs) approved for LGS, clobazam is a more recent market entrant, having been approved in October 2011. [...] Read more.

Background: Lennox-Gastaut syndrome (LGS) is a severe form of childhood-onset epilepsy associated with serious injuries due to frequent and severe seizures. Of the antiepileptic drugs (AEDs) approved for LGS, clobazam is a more recent market entrant, having been approved in October 2011. Recent AED budget impact and cost-effectiveness analyses for LGS suggest that adding clobazam to a health plan formulary may result in decreased medical costs; however, research on clinical and economic outcomes and treatment patterns with these AED treatments in LGS is limited. Objectives: To compare the baseline characteristics and treatment patterns of new initiators of clobazam and other AEDs among LGS patients and compare healthcare utilization and costs before and after clobazam initiation among LGS patients. Methods: A retrospective study of probable LGS patients was conducted using the MarketScan® Commercial, Medicare Supplemental, and Medicaid databases (10/1/2010-3/31/2014). Results: In the Commercial/Medicare Supplemental population, clobazam users were younger, had fewer comorbidities, and more prior AED use than non-clobazam users. In the 12 months pre-treatment initiation, clobazam users had significantly more seizure-related inpatient stays and outpatient visits and higher total seizure-related (p < 0.001) and all-cause (p < 0.001) costs than non-clobazam users. Among clobazam users, when compared to the 12 months pre-clobazam initiation, seizure-related medical utilization and costs were lower in the 12 months post-clobazam initiation (p = 0.004). Total all-cause (p < 0.001) and seizure-related (p = 0.029) costs increased post-clobazam initiation mainly due to the increase in outpatient pharmacy costs. Similar results were observed in the Medicaid population. Conclusions: Baseline results suggest a prescribing preference for clobazam in severe LGS patients. Clobazam users had a reduction in seizure-related medical utilization and costs after clobazam initiation. The improvement in medical costs mostly offset the higher prescription costs following clobazam initiation. Full article

13 pages, 1379 KiB

Open AccessArticle

Ethical Issues Associated with Medical Tourism in Africa

by John J. O. Mogaka, Lucia Mupara and Joyce M Tsoka-Gwegweni

J. Mark. Access Health Policy 2017, 5(1), 1309770; https://doi.org/10.1080/20016689.2017.1309770 - 5 May 2017

Cited by 8 | Viewed by 351

Abstract

Global disparities in medical technologies, laws, economic inequities, and social–cultural differences drive medical tourism (MT), the practice of travelling to consume healthcare that is either too delayed, unavailable, unaffordable or legally proscribed at home. Africa is simultaneously a source and destination for MT. [...] Read more.

Global disparities in medical technologies, laws, economic inequities, and social–cultural differences drive medical tourism (MT), the practice of travelling to consume healthcare that is either too delayed, unavailable, unaffordable or legally proscribed at home. Africa is simultaneously a source and destination for MT. MT however, presents a new and challenging health ethics frontier, being largely unregulated and characterized by policy contradictions, minority discrimination and conflict of interest among role-players. This article assesses the level of knowledge of MT and its associated ethical issues in Africa; it also identifies critical research gaps on the subject in the region. Exploratory design guided by Arksey and O’Malley’s (2005) framework was used. Key search terms and prior determined exclusion/inclusion criteria were used to identify relevant literature sources. Fifty-seven articles met the inclusion criteria. Distributive justice, healthcare resource allocation, experimental treatments and organ transplant were the most common ethical issues of medical tourism in Africa. The dearth of robust engagement of MT and healthcare ethics, as identified through this review, calls for more rigorous research on this subject. Although the bulk of the medical tourism industry is driven by global legal disparities based on ethical considerations, little attention has been given to this subject. Full article

10 pages, 808 KiB

Open AccessReview

Should New Health Technology Be Available Only for Patients Able and Willing to Pay?

by Piet Calcoen, Albert Boer and Wynand P.M.M. van de Ven

J. Mark. Access Health Policy 2017, 5(1), 1315294; https://doi.org/10.1080/20016689.2017.1315294 - 4 May 2017

Cited by 6 | Viewed by 240

Abstract

New health technology comes on the market at a rapid pace and—sometimes—at a huge cost. Providing access to new health technology is a serious challenge for many countries with mandatory health insurance. This article analyses access to new health technology in Belgium and [...] Read more.

New health technology comes on the market at a rapid pace and—sometimes—at a huge cost. Providing access to new health technology is a serious challenge for many countries with mandatory health insurance. This article analyses access to new health technology in Belgium and the Netherlands, using eight concrete examples as a starting point for comparing the two – neighbouring – countries. Contrary to the Netherlands, out-of-pocket payments for new health technology are widely accepted and practiced in Belgium. This difference is largely the result of different regulatory environments. A major difference is the way that entitlements to care are described: closed and explicit in Belgium versus open and non-explicit in the Netherlands. The characteristics of in-kind policies versus reimbursement policies also play a role. Allowing out-of-pocket payments for new health technology has consequences for the patients. It leads to greater access to new health technology (for those who are able and willing to pay), but has a negative effect on equal access to care. Choice and transparency are enhanced by allowing out-of-pocket payments for new health technology. It could be argued that lack of coverage by mandatory health insurance should not render private access to new health technology impossible. Full article

16 pages, 1351 KiB

Open AccessArticle

Supply-Side and Demand-Side Policies for Biosimilars: An Overview in 10 European Member States

by Cécile Rémuzat, Anna Kapuśniak, Aleksandra Caban, Dan Ionescu, Guerric Radière, Cyril Mendoza and Mondher Toumi

J. Mark. Access Health Policy 2017, 5(1), 1307315; https://doi.org/10.1080/20016689.2017.1307315 - 28 Apr 2017

Cited by 46 | Viewed by 585

Abstract

Objective: This study aimed to provide an overview of biosimilar policies in 10 EU MSs. Methods: Ten EU MS pharmaceutical markets (Belgium, France, Germany, Greece, Hungary, Italy, Poland, Spain, Sweden, and the UK) were selected. A comprehensive literature review was performed [...] Read more.

Objective: This study aimed to provide an overview of biosimilar policies in 10 EU MSs. Methods: Ten EU MS pharmaceutical markets (Belgium, France, Germany, Greece, Hungary, Italy, Poland, Spain, Sweden, and the UK) were selected. A comprehensive literature review was performed to identify supply-side and demand-side policies in place in the selected countries. Results: Supply-side policies for biosimilars commonly include price linkage, price re-evaluation, and tendering; the use of internal or external reference pricing varies between countries; health technology assessment is conducted in six countries. Regarding demand-side policies, pharmaceutical prescription budgets or quotas and monitoring of prescriptions (with potential financial incentives or penalties) are in place in eight and in seven countries respectively. Switching is generally allowed, but is solely the physician’s responsibility. Automatic substitution is not recommended, or even forbidden, in most EU MSs. Prescription conditions or guidelines that apply to biosimilars are established in nearly all surveyed EU MSs. Conclusions: Important heterogeneity in policies on biosimilars was seen between (and even within) selected countries, which may partly explain variations in biosimilar uptake. Supply-side policies targeting price have been reported to limit biosimilar penetration in the long term, despite short-term savings, while demand-side policies are considered to positively impact uptake. Full article

7 pages, 1077 KiB

Open AccessArticle

Filling the Gap in CNS Drug Development: Evaluation of the Role of Drug Repurposing

by A. Caban, K. Pisarczyk, K. Kopacz, A. Kapuśniak, M. Toumi, C. Rémuzat and A. Kornfeld

J. Mark. Access Health Policy 2017, 5(1), 1299833; https://doi.org/10.1080/20016689.2017.1299833 (registering DOI) - 10 Apr 2017

Cited by 34 | Viewed by 309

Abstract

Background and objective: Drug repurposing has been considered a cost-effective and reduced-risk strategy for developing new drugs. Little is known and documented regarding the efficiency of repurposing strategies in drug development. The objective of this article is to assess the extent and [...] Read more.

Background and objective: Drug repurposing has been considered a cost-effective and reduced-risk strategy for developing new drugs. Little is known and documented regarding the efficiency of repurposing strategies in drug development. The objective of this article is to assess the extent and meaning of this process in the CNS area. Methods: In order to identify repurposed drugs that target the CNS, an extensive search was performed. For each identified case, its initial and target indication, development status and the type of repurposing strategy (repositioning, reformulation or both) was recorded. Results: One hundred and eighteen source products were identified. They were repurposed (mainly repositioned) 203 times with 81 products repurposed once and 38 products repurposed twice or more. The highest number of source drugs originated from the CNS area. Alzheimer’s disease was targeted most often. Half of the new indications were approved. Regarding repurposing within the CNS area, epilepsy, schizophrenia and depression were the richest sources of repurposed drugs. Conclusions: Repurposing drugs into CNS is an efficient and very active drug development method, exemplified by the considerable number of new indications that have been found via this strategy, with approximately half of the target indications currently under development. Full article

16 pages, 2251 KiB

Open AccessArticle

Do Payers Value Rarity? An Analysis of the Relationship between Disease Rarity and Orphan Drug Prices in Europe

by Goran Medic, Daria Korchagina, Katherine Eve Young, Mondher Toumi, Maarten Jacobus Postma, Micheline Wille and Michiel Hemels

J. Mark. Access Health Policy 2017, 5(1), 1299665; https://doi.org/10.1080/20016689.2017.1299665 - 10 Apr 2017

Cited by 34 | Viewed by 361

Abstract

Background and Objective: Orphan drugs have been a highlight of discussions due to their higher prices than non-orphan drugs. There is currently no European consensus on the method of value assessment for orphan drugs. This study assessed the relationship between the prevalence [...] Read more.

Background and Objective: Orphan drugs have been a highlight of discussions due to their higher prices than non-orphan drugs. There is currently no European consensus on the method of value assessment for orphan drugs. This study assessed the relationship between the prevalence of rare diseases and the annual treatment cost of orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Approved orphan drugs and prevalence data were extracted from the European Medicines Agency website. Annual treatment costs were calculated using ex-factory price. Simple regression was used to analyse the relationship between costs and prevalence. A specific bivariate analysis was performed for the rarest diseases (≤1 per 10,000). Results: 120 drugs were analysed. Prevalence ranged from 0.001 to 5 per 10,000 (mean 1.24, median 1). Annual treatment costs per patient ranged from €755 to €1,051,956 (mean €100,000, median €39,303). Results show a statistically significant inverse correlation between annual treatment cost and disease prevalence in all countries (France: r = −0.370, p = 0.002; Germany: r = −0.365, p = 0.002; Italy: r = −0.340, p = 0.002; Spain: r = −0.316, p = 0.041; UK: r = −0.358, p = 0.0004; Sweden: r = −0.414, p = 0.014; Norway: r = −0.367, p = 0.002). When analysis was focused on the rarest diseases, a stronger correlation exists in all countries (France: r = −0.525, Germany: r = −0.482, Italy: r = −0.497, Spain: r = −0.531, UK: r = −0.436, Sweden: r = −0.455, Norway: r = −0.466; all p < 0.05 except Sweden p = 0.077). Conclusions: This study shows an inverse correlation between annual treatment cost and prevalence with high statistical significance in the studied countries. Although pricing is a complex process where different attributes are assessed, this study supports the idea that payers value rarity in pricing decisions. Full article

13 pages, 1827 KiB

Open AccessArticle

A Comparative Study of Orphan Drug Prices in Europe

by Katherine Eve Young, Imen Soussi, Michiel Hemels and Mondher Toumi

J. Mark. Access Health Policy 2017, 5(1), 1297886; https://doi.org/10.1080/20016689.2017.1297886 - 29 Mar 2017

Cited by 23 | Viewed by 340

Abstract

Background and Objective: This study assessed price differences by comparing annual treatment costs of similarly available orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Annual treatment costs per drug were calculated using ex-factory prices from IHS POLI [...] Read more.

Background and Objective: This study assessed price differences by comparing annual treatment costs of similarly available orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Annual treatment costs per drug were calculated using ex-factory prices from IHS POLI and country price databases. The treatment cost in the comparator country was compared to the UK and ratios were analysed. Subanalyses were done on disease areas and UK cost quartiles. Results: 120 orphan drugs were included. Compared to the UK, the average costs were more expensive in France (1.13), Germany (1.11), Italy (1.08), Spain (1.07), and were cheaper in Sweden (0.99) and Norway (0.88). The average ratios offered a restrictive view as ratios were greatly heterogeneous (0.26 to 1.92) which was also seen in the different disease areas. The averaged ratios varied minimally among the cost quartiles which shows that cost differences were similar for the most expensive and least expensive orphan drugs in the UK. Conclusions: Individual orphan drug prices can vary widely across European countries, although on average these differences are relatively minor. This study suggests that in Europe, we may not be able predict which country may have higher or lower prices for orphan drugs. Full article

15 pages, 1443 KiB

Open AccessArticle

Unlocking the Potential of Established Products: Toward New Incentives Rewarding Innovation in Europe

by Gabrielle Nayroles, Sandrine Frybourg, Sylvie Gabriel, Åsa Kornfeld, Fernando Antoñanzas-Villar, Jaime Espín, Claudio Jommi, Nello Martini, Gérard de Pouvourville, Keith Tolley, Jürgen Wasem and Mondher Toumi

J. Mark. Access Health Policy 2017, 5(1), 1298190; https://doi.org/10.1080/20016689.2017.1298190 - 12 Mar 2017

Cited by 14 | Viewed by 327

Abstract

Background: Many established products (EPs – marketed for eight years or more) are widely used off-label despite little evidence on benefit–risk ratio. This exposes patients to risks related to safety and lack of efficacy, and healthcare providers to liability. Introducing new indications [...] Read more.

Background: Many established products (EPs – marketed for eight years or more) are widely used off-label despite little evidence on benefit–risk ratio. This exposes patients to risks related to safety and lack of efficacy, and healthcare providers to liability. Introducing new indications for EPs may represent a high societal value; however, manufacturers rarely invest in R&D for EPs. The objective of this research was to describe incentives and disincentives for developing new indications for EPs in Europe and to investigate consequences of current policies. Methods: Targeted literature search and expert panel meetings. Results: Within the current European-level and national-level regulatory framework there are limited incentives for development of new indications with EPs. Extension of indication normally does not allow the price to be increased or maintained, the market protection period to be extended, or exclusion from a reference price system. New indication frequently triggers re-evaluation, resulting in price erosion, regardless of the level of added value with the new indication. In consequence, manufacturers are more prone to undertake R&D efforts at early to mid-stage of product life cycle rather than with EPs, or to invest in new chemical entities, even in therapeutic areas with broad off-label use. This represents a potentially missed opportunity as developing new indications for EPs offers an alternative to off-label use or lengthy and expensive R&D for new therapies, opens new opportunities for potentially cost-effective treatment alternatives, as well as greater equity in patients’ access to treatment options. Conclusion: There are potential benefits from the development of new indications for EPs that are currently not being realized due to a lack of regulatory and pricing incentives in Europe. Incentives for orphan or paediatric drugs have proven to be effective in promoting R&D. Similarly, incentives to promote R&D in EPs should be developed, for the benefit of patients and healthcare systems. Full article

6 pages, 925 KiB

Open AccessCommunication

Role of Preliminary Registry Data in Development of a Clinical Trial for an Innovative Device: A Small but Integral Piece of a Health Policy Initiative

by Donald R. Ricci, Joost de Vries and Raphael Blanc

J. Mark. Access Health Policy 2017, 5(1), 1283106; https://doi.org/10.1080/20016689.2017.1283106 - 2 Mar 2017

Cited by 3 | Viewed by 176

Abstract

Establishing a national health policy at a macro level involves the integration of a series of health initiatives across a spectrum of activities, including clinical care. Evaluation of the safety and efficacy of a new medical device ultimately evolves to testing in humans. [...] Read more.

Establishing a national health policy at a macro level involves the integration of a series of health initiatives across a spectrum of activities, including clinical care. Evaluation of the safety and efficacy of a new medical device ultimately evolves to testing in humans. The pathway to a formal prospective clinical trial includes a stepwise appreciation of pre-clinical data and detailed analysis of data obtained from preliminary registries, where information about appropriate patient selection and use of the device is obtained. Evaluation of procedural and follow-up efficacy and safety data in a preliminary series of cases, chosen to simulate published data, allows the design and conduct of clinical trials that are required to verify preliminary observations, closing the loop on one aspect of modifying health policy decisions. Full article

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J. Mark. Access Health Policy, Volume 5, Issue 1 (January 2017) – 35 articles

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