Journal of Market Access & Health Policy

2 pages, 155 KiB

Open AccessLetter

Pricing and Reimbursement of Novel Oncology Drugs in Sweden

by Mattias Haglund and Eric Miller

J. Mark. Access Health Policy 2014, 2(1), 25925; https://doi.org/10.3402/jmahp.v2.25925 - 9 Dec 2014

Cited by 1 | Viewed by 114

We read with great interest the article by Cohen and Felix (1) concerning reimbursement restrictions on orphan drugs [...] Full article

4 pages, 185 KiB

Open AccessReview

Pharmaceutical Market Access in Emerging Markets: Concepts, Components, and Future

by Anuj Kumar, Karthaveerya Juluru, Phani Kishore Thimmaraju, Jayachandra Reddy and Anand Patil

J. Mark. Access Health Policy 2014, 2(1), 25302; https://doi.org/10.3402/jmahp.v2.25302 - 1 Dec 2014

Cited by 15 | Viewed by 320

Abstract

This article intends to consolidate the concepts of pharmaceutical market access and highlight its growing importance in emerging markets. Market access has gained considerable attentionworldwide as countries try to contain their escalating healthcare expenditures amidst the global economic slowdown. This has resulted in [...] Read more.

This article intends to consolidate the concepts of pharmaceutical market access and highlight its growing importance in emerging markets. Market access has gained considerable attentionworldwide as countries try to contain their escalating healthcare expenditures amidst the global economic slowdown. This has resulted in governments adopting stricter measures for new product approval. Thus, pharmaceutical companies are finding it increasingly difficult to successfully address the specific challenges posed by various government and regulatory agencies and stakeholders. There is an increasing need to establish market access functions, especially in emerging markets, where the complex, dynamic healthcare landscape confounds product approval and uptake. Moreover, emerging markets are the engines of growth today, and, thus, performing in these markets is critical for the majority of pharmaceutical companies. To address the challenges posed by regulatory agencies and diverse stakeholders, a customized market access strategy is the need of the hour. A market access framework with specific tools and tactics will help companies to plan, implement, and monitor stakeholder engagement activities. Full article

15 pages, 3611 KiB

Open AccessArticle

Novel Methodology for Pharmaceutical Expenditure Forecast

by Anne-Lise Vataire, Laurent Cetinsoy, Samuel Aballéa, Cécile Rémuzat, Duccio Urbinati, Åsa Kornfeld, Olfa Mzoughi and Mondher Toumi

J. Mark. Access Health Policy 2014, 2(1), 24082; https://doi.org/10.3402/jmahp.v2.24082 - 27 Nov 2014

Cited by 5 | Viewed by 128

Abstract

Background and objective: The value appreciation of new drugs across countries today features a disruption that is making the historical data that are used for forecasting pharmaceutical expenditure poorly reliable. Forecasting methods rarely addressed uncertainty. The objective of this project was to propose [...] Read more.

Background and objective: The value appreciation of new drugs across countries today features a disruption that is making the historical data that are used for forecasting pharmaceutical expenditure poorly reliable. Forecasting methods rarely addressed uncertainty. The objective of this project was to propose a methodology to perform pharmaceutical expenditure forecasting that integrates expected policy changes and uncertainty (developed for the European Commission as the ‘EU Pharmaceutical expenditure forecast’; see http://ec.europa.eu/health/healthcare/key_documents/index_en.htm). Methods: 1) Identification of all pharmaceuticals going off-patent and new branded medicinal products over a 5-year forecasting period in seven European Union (EU) Member States. 2) Development of a model to estimate direct and indirect impacts (based on health policies and clinical experts) on savings of generics and biosimilars. Inputs were originator sales value, patent expiry date, time to launch after marketing authorization, price discount, penetration rate, time to peak sales, and impact on brand price. 3) Development of a model for new drugs, which estimated sales progression in a competitive environment. Clinical expected benefits as well as commercial potential were assessed for each product by clinical experts. Inputs were development phase, marketing authorization dates, orphan condition, market size, and competitors. 4) Separate analysis of the budget impact of products going off-patent and new drugs according to several perspectives, distribution chains, and outcomes. 5) Addressing uncertainty surrounding estimations via deterministic and probabilistic sensitivity analysis. Results: This methodology has proven to be effective by 1) identifying the main parameters impacting the variations in pharmaceutical expenditure forecasting across countries: generics discounts and penetration, brand price after patent loss, reimbursement rate, the penetration of biosimilars and discount price, distribution chains, and the time to reach peak sales for new drugs; 2) estimating the statistical distribution of the budget impact; and 3) testing different pricing and reimbursement policy decisions on health expenditures. Conclusions: This methodology was independent of historical data and appeared to be highly flexible and adapted to test robustness and provide probabilistic analysis to support policy decision making. Full article

12 pages, 495 KiB

Open AccessArticle

Using Multicriteria Decision Analysis during Drug Development to Predict Reimbursement Decisions

by Paul Williams, Josephine Mauskopf, Jake Lebiecki and Anne Kilburg

J. Mark. Access Health Policy 2014, 2(1), 25270; https://doi.org/10.3402/jmahp.v2.25270 - 31 Oct 2014

Cited by 11 | Viewed by 143

Abstract

Background: Pharmaceutical companies design clinical development programs to generate the data that they believe will support reimbursement for the experimental compound. Objective: The objective of the study was to present a process for using multicriteria decision analysis (MCDA) by a pharmaceutical company to [...] Read more.

Background: Pharmaceutical companies design clinical development programs to generate the data that they believe will support reimbursement for the experimental compound. Objective: The objective of the study was to present a process for using multicriteria decision analysis (MCDA) by a pharmaceutical company to estimate the probability of a positive recommendation for reimbursement for a new drug given drug and environmental attributes. Methods: The MCDA process included 1) selection of decisions makers who were representative of those making reimbursement decisions in a specific country; 2) two pre-workshop questionnaires to identify the most important attributes and their relative importance for a positive recommendation for a new drug; 3) a 1-day workshop during which participants undertook three tasks: i) they agreed on a final list of decision attributes and their importance weights, ii) they developed level descriptions for these attributes and mapped each attribute level to a value function, and iii) they developed profiles for hypothetical products ‘just likely to be reimbursed’; and 4) use of the data from the workshop to develop a prediction algorithm based on a logistic regression analysis. The MCDA process is illustrated using case studies for three countries, the United Kingdom, Germany, and Spain. The extent to which the prediction algorithms for each country captured the decision processes for the workshop participants in our case studies was tested using a post-meeting questionnaire that asked the participants to make recommendations for a set of hypothetical products. Results: The data collected in the case study workshops resulted in a prediction algorithm: 1) for the United Kingdom, the probability of a positive recommendation for different ranges of cost-effectiveness ratios; 2) for Spain, the probability of a positive recommendation at the national and regional levels; and 3) for Germany, the probability of a determination of clinical benefit. The results from the post-meeting questionnaire revealed a high predictive value for the algorithm developed using MCDA. Conclusions: Prediction algorithms developed using MCDA could be used by pharmaceutical companies when designing their clinical development programs to estimate the likelihood of a favourable reimbursement recommendation for different product profiles and for different positions in the treatment pathway. Full article

9 pages, 588 KiB

Open AccessArticle

EU Pharmaceutical Expenditure Forecast

by Duccio Urbinati, Cécile Rémuzat, Åsa Kornfeld, Anne-Lise Vataire, Laurent Cetinsoy, Samuel Aballéa, Olfa Mzoughi and Mondher Toumi

J. Mark. Access Health Policy 2014, 2(1), 23738; https://doi.org/10.3402/jmahp.v2.23738 - 30 Oct 2014

Cited by 8 | Viewed by 170

Abstract

Background and Objectives: With constant incentives for healthcare payers to contain their pharmaceutical budgets, forecasting has become critically important. Some countries have, for instance, developed pharmaceutical horizon scanning units. The objective of this project was to build a model to assess the net [...] Read more.

Background and Objectives: With constant incentives for healthcare payers to contain their pharmaceutical budgets, forecasting has become critically important. Some countries have, for instance, developed pharmaceutical horizon scanning units. The objective of this project was to build a model to assess the net effect of the entrance of new patented medicinal products versus medicinal products going off-patent, with a defined forecast horizon, on selected European Union (EU) Member States’ pharmaceutical budgets. This model took into account population ageing, as well as current and future country-specific pricing, reimbursement, and market access policies (the project was performed for the European Commission; see http://ec.europa.eu/health/healthcare/key_documents/index_en.htm). Method: In order to have a representative heterogeneity of EU Member States, the following countries were selected for the analysis: France, Germany, Greece, Hungary, Poland, Portugal, and the United Kingdom. A forecasting period of 5 years (2012–2016) was chosen to assess the net pharmaceutical budget impact. A model for generics and biosimilars was developed for each country. The model estimated a separate and combined effect of the direct and indirect impacts of the patent cliff. A second model, estimating the sales development and the risk of development failure, was developed for new drugs. New drugs were reviewed individually to assess their clinical potential and translate it into commercial potential. The forecast was carried out according to three perspectives (healthcare public payer, society, and manufacturer), and several types of distribution chains (retail, hospital, and combined retail and hospital). Probabilistic and deterministic sensitivity analyses were carried out. Results: According to the model, all countries experienced drug budget reductions except Poland (+€41 million). Savings were expected to be the highest in the United Kingdom (−€9,367 million), France (−€5,589 million), and, far behind them, Germany (−€831 million), Greece (−€808 million), Portugal (−€243 million), and Hungary (−€84 million). The main source of savings came from the cardiovascular, central nervous system, and respiratory areas and from biosimilar entries. Oncology, immunology, and inflammation, in contrast, lead to additional expenditure. The model was particularly sensitive to the time to market of branded products, generic prices, generic penetration, and the distribution of biosimilars. Conclusions: The results of this forecast suggested a decrease in pharmaceutical expenditure in the studied period. The model was sensitive to pharmaceutical policy decisions. Full article

6 pages, 293 KiB

Open AccessArticle

Challenges in Generating Costs and Utilisation Rates Associated with Castration-Resistant Prostate Cancer

by Siobhan Bourke, Richéal Maria Burns and Caroline Gaynor

J. Mark. Access Health Policy 2014, 2(1), 24072; https://doi.org/10.3402/jmahp.v2.24072 - 4 Jul 2014

Cited by 5 | Viewed by 113

Abstract

Background: Prostate cancer (PCa), the most commonly diagnosed cancer among men in the United States and Europe, is an escalating resource allocation issue across healthcare systems in the Western world. The impact of skeletal-related events, associated with castration-resistant prostate cancer (CRPC), is considerable [...] Read more.

Background: Prostate cancer (PCa), the most commonly diagnosed cancer among men in the United States and Europe, is an escalating resource allocation issue across healthcare systems in the Western world. The impact of skeletal-related events, associated with castration-resistant prostate cancer (CRPC), is considerable with many new therapies being sought to treat these events in a cost-effective manner. Aims: The aim of this paper is to provide insight into the level of constraints associated with devising cost frameworks for economic analysis of CRPC in the Irish healthcare setting. Methods: An informal questionnaire was devised to obtain estimates of utilisation to populate a decision tree model; existing parameters from the literature were also employed. Cost parameters included Irish reference costs, and a costs literature review was undertaken; a healthcare payer perspective was adopted. Pharmacy dosages used for modelling costs were calculated for an average 75 kg male. Results: The estimated average cost of care associated with adverse events in CRPC was €23,264. Approximately 40% of the costs of CRPC are attributed to skeletal-related events; therefore, reducing the number of skeletal-related events could significantly reduce the cost of care. In attempting to generate accurate and reliable cost parameters, this study highlights the challenges of conducting economic analysis in the Irish healthcare setting. Conclusion: This study presents leading treatments and associated costs for CRPC patients in the Republic of Ireland (RoI), which are expected to steadily increase with demographic shifts. Further research is warranted in this area due to the limitations encountered in the study. Full article

7 pages, 247 KiB

Open AccessArticle

Health Economic Value of an Innovation: Delimiting the Scope and Framework of Future Market Entry Agreements

by Robert Launois, Lucia Fiestas Navarrete, Olivier Ethgen, Jean-Gabriel Le Moine and René Gatsinga

J. Mark. Access Health Policy 2014, 2(1), 24988; https://doi.org/10.3402/jmahp.v2.24988 - 23 Jun 2014

Cited by 5 | Viewed by 145

Abstract

Background and objectives: The objective of our paper is to offer a new, payer-friendly taxonomy of market entry agreements (MEAs) that aims to twin contracts with their methodological designs in an effort to clarify the distinction between contracts that are based on performance [...] Read more.

Background and objectives: The objective of our paper is to offer a new, payer-friendly taxonomy of market entry agreements (MEAs) that aims to twin contracts with their methodological designs in an effort to clarify the distinction between contracts that are based on performance and those that are based on demonstrated effect. Methods: Our analysis proceeds in two stages: First, we delimit the scope and framework of pay for performance (P4P) and pay for demonstrated effect (P4E) agreements. Second, we distinguish the methodological designs supporting the implementation of each of these contracts. Results: We elucidate why P4P contracts prevent the payer from funding the true effectiveness of an innovation by expanding on their limitations. These include: 1) the normative nature of comparisons, 2) the impossibility of true effect imputability for each individual, and 3) the use of intermediary outcome measures. We then explore three main criticisms that payers must take into account when reasoning in terms of performance rather than in terms of the product effectiveness. Conclusion: The potential effect that performance-based reimbursements may have on dissociating the components of the cost-effectiveness ratio constitutes an obstacle to a true health economic reasoning. Full article

4 pages, 196 KiB

Open AccessCommunication

And If the Discovery of New Drugs for the Treatment of Brain Diseases Depends on Asian Countries?

by Jean-Louis Kraus

J. Mark. Access Health Policy 2014, 2(1), 24379; https://doi.org/10.3402/jmahp.v2.24379 - 11 Jun 2014

Viewed by 108

Abstract

At the present time, developed countries are making a huge financial effort to support neuroscience research programs, particularly in the fields of advanced research and treatment of brain diseases and mental disorders. A part of this financial support is devoted to drug discovery [...] Read more.

At the present time, developed countries are making a huge financial effort to support neuroscience research programs, particularly in the fields of advanced research and treatment of brain diseases and mental disorders. A part of this financial support is devoted to drug discovery programs. The purpose of this communication is to focus on the different parameters (economic, social, and scientific) allowing for the prominent belief that the discovery of new efficient drugs to treat brain disease to an increasing extent is likely to emanate from the Asian countries. A special focus on drug research and discovery in France reveals that, due to the current social context, the lack of small pharmaceutical ventures, the Mediator drug scandal, and the economic situation, the potential for discovering and develo** new drugs is dramatically declining. Full article

8 pages, 263 KiB

Open AccessArticle

Pharmaceutical Expenditure Forecast Model to Support Health Policy Decision Making

by Cécile Rémuzat, Duccio Urbinati, Åsa Kornfeld, Anne-Lise Vataire, Laurent Cetinsoy, Samuel Aballéa, Olfa Mzoughi and Mondher Toumi

J. Mark. Access Health Policy 2014, 2(1), 23740; https://doi.org/10.3402/jmahp.v2.23740 - 4 Jun 2014

Cited by 5 | Viewed by 134

Abstract

Background and objective: With constant incentives for healthcare payers to contain their pharmaceutical budgets, modelling policy decision impact became critical. The objective of this project was to test the impact of various policy decisions on pharmaceutical budget (developed for the European Commission for [...] Read more.

Background and objective: With constant incentives for healthcare payers to contain their pharmaceutical budgets, modelling policy decision impact became critical. The objective of this project was to test the impact of various policy decisions on pharmaceutical budget (developed for the European Commission for the project ‘European Union (EU) Pharmaceutical expenditure forecast’ – http://ec.europa.eu/health/healthcare/key_documents/index_en.htm). Methods: A model was built to assess policy scenarios’ impact on the pharmaceutical budgets of seven member states of the EU, namely France, Germany, Greece, Hungary, Poland, Portugal, and the United Kingdom. The following scenarios were tested: expanding the UK policies to EU, changing time to market access, modifying generic price and penetration, shifting the distribution chain of biosimilars (retail/hospital). Results: Applying the UK policy resulted in dramatic savings for Germany (10 times the base case forecast) and substantial additional savings for France and Portugal (2 and 4 times the base case forecast, respectively). Delaying time to market was found be to a very powerful tool to reduce pharmaceutical expenditure. Applying the EU transparency directive (6-month process for pricing and reimbursement) increased pharmaceutical expenditure for all countries (from 1.1 to 4 times the base case forecast), except in Germany (additional savings). Decreasing the price of generics and boosting the penetration rate, as well as shifting distribution of biosimilars through hospital chain were also key methods to reduce pharmaceutical expenditure. Change in the level of reimbursement rate to 100% in all countries led to an important increase in the pharmaceutical budget. Conclusions: Forecasting pharmaceutical expenditure is a critical exercise to inform policy decision makers. The most important leverages identified by the model on pharmaceutical budget were driven by generic and biosimilar prices, penetration rate, and distribution. Reducing, even slightly, the prices of generics had a major impact on savings. However, very aggressive pricing of generic and biosimilar products might make this market unattractive and can be counterproductive. Worth noting, delaying time to access innovative products was also identified as an effective leverage to increase savings but might not be a desirable policy for breakthrough products. Increasing patient financial contributions, either directly or indirectly via their private insurances, is a more likely scenario rather than expanding the national pharmaceutical expenditure coverage. Full article

9 pages, 226 KiB

Open AccessReview

Is the Gap Between Micro- and Macroeconomic Assessments in Health Care Well Understood? The Case of Vaccination and Potential Remedies

by Nikolaos Kotsopoulos and Mark P. Connolly

J. Mark. Access Health Policy 2014, 2(1), 23897; https://doi.org/10.3402/jmahp.v2.23897 - 10 Apr 2014

Cited by 14 | Viewed by 246

Abstract

Vaccination is an established intervention that reduces the burden and prevents the spread of infectious diseases. Investing in vaccination is known to offer a wide range of economic and intangible benefits that can potentiate gains for the individual and for society. The discipline [...] Read more.

Vaccination is an established intervention that reduces the burden and prevents the spread of infectious diseases. Investing in vaccination is known to offer a wide range of economic and intangible benefits that can potentiate gains for the individual and for society. The discipline of economics provides us with microeconomic and macroeconomic methods for evaluating the economic gains attributed to health status changes. However, the observed gap between micro and macro estimates attributed to health presents challenges to our understanding of health-related productivity changes and, consequently, economic benefits. The gap suggests that the manner in which health-related productive output is quantified in microeconomic models might not adequately reflect the broader economic benefit. We propose that there is a transitional domain that links the micro- and macroeconomic improvement attributed to health status changes. Currently available economic evaluation methods typically omit these consequences, however; they may be adjusted to integrate these transitional consequences. In practical terms, this may give rise to multipliers to apply toward indirect costs to account for the broader macroeconomic benefits linked to changes in health status. In addition, it is possible to consider that different medical conditions and health care interventions may pose different multiplying effects, suggesting that the manner in which resources are allocated within health services gives rise to variation in the amount of the micro–macro gap. An interesting way to move forward in integrating the micro- and macro-level assessment might be by integrating computable general equilibrium (CGE) models as part of the evaluation framework, as was recently performed for pandemic flu and malaria vaccination. Full article

4 pages, 181 KiB

Open AccessCommunication

The Value of Quantitative Patient Preferences in Regulatory Benefit-Risk Assessment

by Mart oude Egbrink and Maarten IJzerman

J. Mark. Access Health Policy 2014, 2(1), 22761; https://doi.org/10.3402/jmahp.v2.22761 - 1 Apr 2014

Cited by 34 | Viewed by 174

Abstract

Quantitative patient preferences are a method to involve patients in regulatory benefit-risk assessment. Assuming preferences can be elicited, there might be multiple advantages to their use. Legal, methodological and procedural issues do however imply that preferences are currently at most part of the [...] Read more.

Quantitative patient preferences are a method to involve patients in regulatory benefit-risk assessment. Assuming preferences can be elicited, there might be multiple advantages to their use. Legal, methodological and procedural issues do however imply that preferences are currently at most part of the solution on how to best involve patients in regulatory decision making. Progress is recently made on these issues. Full article

10 pages, 1394 KiB

Open AccessReview

Health Technology Assessment in Middle-Income Countries: Recommendations for a Balanced Assessment System

by Dávid Dankó

J. Mark. Access Health Policy 2014, 2(1), 23181; https://doi.org/10.3402/jmahp.v2.23181 - 11 Mar 2014

Cited by 31 | Viewed by 148

Abstract

Because of significant differences in institutional contexts, health technology assessment (HTA) systems that are in place in core pharmaceutical markets may not be suitable, fully or in part, for middle-income countries (MICs) and for other noncore markets. Particular challenges may arise when systems [...] Read more.

Because of significant differences in institutional contexts, health technology assessment (HTA) systems that are in place in core pharmaceutical markets may not be suitable, fully or in part, for middle-income countries (MICs) and for other noncore markets. Particular challenges may arise when systems based on the economic evaluation paradigm are conceptualized and implemented in MICs, sometimes with an insufficient level of awareness of the local institutional factors that influence pricing and reimbursement decision making. Focusing on pharmaceuticals, this article investigates possible development directions for HTA systems in MICs and noncore markets bearing similar institutional characteristics, and it provides recommendations for a balanced assessment system (BAS). For this, the main paradigms of HTA have also been reviewed briefly and factors influencing HTA and pricing and reimbursement decisions in MICs and in similar noncore countries have been summarized. The proposed BAS framework takes into account available resources and capabilities and is supposed to facilitate access to new pharmaceuticals while ensuring the transparency of decision-making processes and the stability of the pharmaceutical budget. Full article

8 pages, 302 KiB

Open AccessPerspective

Economic Impact of New Active Substance Status on EU Payers’ Budgets: Example of Dimethyl Fumarate (Tecfidera^®) for Multiple Sclerosis

by Mondher Toumi and Guy Jadot

J. Mark. Access Health Policy 2014, 2(1), 23932; https://doi.org/10.3402/jmahp.v2.23932 - 4 Mar 2014

Cited by 4 | Viewed by 131

Abstract

Background: Recently, collaboration between regulators and payers was set up and was mainly focused on evidence generation along product clinical development. However, neither the regulatory path nor the new active substance status (NASs) was considered. Granting NASs will provide the product with 8 [...] Read more.

Background: Recently, collaboration between regulators and payers was set up and was mainly focused on evidence generation along product clinical development. However, neither the regulatory path nor the new active substance status (NASs) was considered. Granting NASs will provide the product with 8 years of data protection and 2 years of market exclusivity during which no generic could enter the market. Objective: To review the economic impact (for payers) of NASs granted by the European Medicines Agency (EMA) for dimethyl fumarate (DMF), developed by Biogen and approved for multiple sclerosis (MS) as Tecfidera^® on 3 February 2014. Method: We reviewed the available DMF-containing products and identified their indication and price through relevant databases and official Web sites. The economic impact of Tecfidera^® on payers’ budgets was calculated assuming NASs was or was not granted. The forecast was identified in Datamonitor. Results: Results identified four products already containing DMF as the main or unique active substance. This would have potentially prevented Tecfidera^® from being granted NASs. The EMA Committee for Medicinal Products for Human Use (CHMP) denied Tecfidera^® NASs and, following a company appeal, reversed its position opening as polemic. The impact of that decision has been evaluated at €7 to €10 billion over a 10-year period. Conclusion: NASs is a critical decision because it does have a major budget impact for payers, and it prevents generic competition. Current European Union (EU) regulations on that topic are unclear and open up too many interpretations thus distorting fair trade and affecting payers’ bills. Greater clarity and more stringent rules are required to prevent mistrust of this EMA decision. Full article

13 pages, 162 KiB

Open AccessArticle

Drug Repurposing in Pharmaceutical Industry and Its Impact on Market Access: Market Access Implications

by Susana Murteira, Aurélie Millier and Mondher Toumi

J. Mark. Access Health Policy 2014, 2(1), 22814; https://doi.org/10.3402/jmahp.v2.22814 - 25 Feb 2014

Cited by 16 | Viewed by 213

Abstract

Background: Drug repurposing is a group of development strategies employed in order to overcome some of the hurdles innate to drug research and development. Drug repurposing includes drug repositioning, reformulation and combination. Objective: This study aimed to identify the determinants of successful market [...] Read more.

Background: Drug repurposing is a group of development strategies employed in order to overcome some of the hurdles innate to drug research and development. Drug repurposing includes drug repositioning, reformulation and combination. Objective: This study aimed to identify the determinants of successful market access outcome for drug repurposing in the United States of America (USA) and in Europe. Methods: The case studies of repurposing strategies were identified through a systematic review of the literature. Price information and reimbursement conditions for all the case studies were collected mainly through access of public datasources. A list of attributes that could be associated with market access outcome (price level and reimbursement conditions) was developed, discussed, and validated by an external expert group. Detailed information for all attributes was researched and collected for each case study. Bivariate regression models were conducted to identify factors associated with price change for all repurposing cases. A similar analysis was performed for reformulation and repositioning cases, in the USA and in Europe, separately. A significance level of 5% was used for all analyses. Results: A total of 144 repurposing case studies were included in the statistical analysis for evaluation of mean price change. Combination cases (the combination of two or more individual drug components) were excluded from the statistical analysis due to the low number of cases retrieved. The main attributes associated with a significant price increase for overall repurposing cases were ‘change in administration setting to hospital’ (374%, p < 0.0001), ‘addressing unmet needs’ (69%, p < 0.05), ‘reformulations belonging to Group 3’—that is, change in administration route (117%, p < 0.001), and being a repurposed product with the ‘same brand name’ as the original product (65%, p < 0.05). Conclusion: We found that the ability of the repurposed product to address unmet needs, a reformulation where the target product had a different administration route than the source product, and having a similar brand name for repurposed and original products, were variables that impacted a positive price change for repurposed drugs overall. Our research results also suggested that orphan designation could have a positive impact for repositioning in the USA, in particular. Although a change of administration from ambulatory to hospital setting seemed to be significantly correlated with a price increase for the target product, only one case was retrieved for this parameter; as such, it was not possible to infer a firm correlation between this parameter and a change in price. Full article

16 pages, 684 KiB

Open AccessArticle

Drug Reformulations and Repositioning in the Pharmaceutical Industry and Their Impact on Market Access: Regulatory Implications

by Susana Murteira, Aurélie Millier, Zied Ghezaiel and Michel Lamure

J. Mark. Access Health Policy 2014, 2(1), 22813; https://doi.org/10.3402/jmahp.v2.22813 - 29 Jan 2014

Cited by 38 | Viewed by 271

Abstract

Background: Repurposing has become a mainstream strategy in drug development, but it faces multiple challenges, amongst them the increasing and ever changing regulatory framework. This is the second study of a series of three-part publication project with the ultimate goal of understanding the [...] Read more.

Background: Repurposing has become a mainstream strategy in drug development, but it faces multiple challenges, amongst them the increasing and ever changing regulatory framework. This is the second study of a series of three-part publication project with the ultimate goal of understanding the market access rationale and conditions attributed to drug repurposing in the United States and in Europe. The aim of the current study to evaluate the regulatory path associated with each type of repurposing strategy according to the previously proposed nomenclature in the first article of this series. Methods: From the cases identified, a selection process retrieved a total of 141 case studies in all countries, harmonized for data availability and common approval in the United States and in Europe. Regulatory information for each original and repurposed drug product was extracted, and several related regulatory attributes were also extracted such as, designation change and filing before or after patent expiry, among others. Descriptive analyses were conducted to determine trends and to investigate potential associations between the different regulatory paths and attributes of interest, for reformulation and repositioning cases separately. Results: Within the studied European countries, most of the applications for reformulated products were filed through national applications. In contrast, for repositioned products, the centralized procedure was the most frequent regulatory pathway. Most of the repurposing cases were approved before patent expiry, and those cases have followed more complex regulatory pathways in the United States and in Europe. For new molecular entities filed in the United States, a similar number of cases were developed by serendipity and by a hypothesis-driven approach. However, for the new indication's regulatory pathway in the United States, most of the cases were developed through a hypothesis-driven approach. Conclusion: The regulations in the United States and in Europe for drug repositionings and reformulations allowed confirming that repositioning strategies were usually filed under a more complex regulatory process than reformulations. Also, it seems that parameters such as patent expiry and type of repositioning approach or reformulation affect the regulatory pathways chosen for each case. Full article

5 pages, 109 KiB

Open AccessArticle

Are Payers Treating Orphan Drugs Differently?

by Joshua P. Cohen and Abigail Felix

J. Mark. Access Health Policy 2014, 2(1), 23513; https://doi.org/10.3402/jmahp.v2.23513 - 15 Jan 2014

Cited by 39 | Viewed by 161

Abstract

Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years. In this article, we [...] Read more.

Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years. In this article, we examine payer coverage in the United States, England and Wales, and the Netherlands of outpatient orphan drugs approved between 1983 and 2012, as well as the 11 most expensive orphan drugs. Methods: We collected data from drug regulatory agencies as well as payers and drug evaluation authorities. Results: We found that orphan drugs have more coverage restrictions than non-orphan drugs in all three jurisdictions. From an economic perspective, the fact that a drug is an orphan product or has a high per-unit price per se should not imply a special kind of evaluation by payers, or necessarily the imposition of more coverage restrictions. Conclusion: Payers should consider the same set of decision criteria that they do with respect to non-orphan drugs: disease severity, availability of treatment alternatives, level of unmet medical need, and cost-effectiveness, criteria that justifiably may be taken into account and traded off against one another in prescribing and reimbursement decisions for orphan drugs. Full article

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J. Mark. Access Health Policy, Volume 2, Issue 1 (January 2014) – 16 articles

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