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Steven Simoens - One of the best experts on this subject based on the ideXlab platform.

  • what is known about the cost effectiveness of Orphan Drugs evidence from cost utility analyses
    Journal of Clinical Pharmacy and Therapeutics, 2015
    Co-Authors: Eline Picavet, David Cassiman, Steven Simoens
    Abstract:

    Summary What is known and objective In times of financial and economic hardship, governments are looking to contain pharmaceutical expenditure by focusing on cost-effective Drugs. Because of their high prices and difficulties in demonstrating effectiveness in small patient populations, Orphan Drugs are often perceived as not able to meet traditional reimbursement threshold value for money. The aim of this study was to provide an overview of the available evidence on the cost-effectiveness of Orphan Drugs. Methods All Orphan Drugs listed as authorized on the website of the European Medicines Agency on 21 November 2013 were included in the analysis. Cost-utility analyses (CUAs) were identified by searching the Tufts Medical Center Cost-Effectiveness Analysis Registry and Embase. For each CUA, a number of variables were collected. Results and discussion The search identified 23 articles on the Tufts registry and 167 articles on Embase. The final analysis included 45 CUAs and 61 incremental cost-utility ratios (ICURs) for 19 Orphan Drugs. Of all ICURS, 16·3% were related to dominant Drugs (i.e. more effective and less expensive than the comparator), 70·5% were related to Drugs that are more effective, but at a higher cost, and 13·1% were related to dominated Drugs (i.e. less effective and more expensive than the comparator). The median overall ICUR was €40 242 per quality-adjusted life year (QALY) with a minimum ICUR of €6311/QALY and a maximum ICUR of €974 917/QALY. What is new and conclusion This study demonstrates that Orphan Drugs can meet traditional reimbursement thresholds. Considering a threshold of £30 000/QALY, in this study, ten (52·6%) of a total of 19 Orphan Drugs for which data were available meet the threshold. As much as fifteen Orphan Drugs (78·9%) are eligible for reimbursement if a threshold of €80 000/QALY is considered.

  • Reimbursement of Orphan Drugs in Belgium: what (else) matters?
    Orphanet Journal of Rare Diseases, 2014
    Co-Authors: Eline Picavet, David Cassiman, Steven Simoens
    Abstract:

    Background: Most Orphan Drugs do not meet traditional standards of cost-effectiveness. Yet, most Orphan Drugs are reimbursed, which implies that other factors are taken into account at the time of reimbursement. To increase accountability of decision-makers, there is a need for more transparency in the factors that play a role in reimbursement decisions of Orphan Drugs. Therefore, the aim of this study is to use a combination of qualitative research methods to examine which official and non-official factors influence reimbursement decisions for Orphan Drugs in Belgium. Methods: Six semi-structured interviews with past or present members of the Drug Reimbursement Committee (DRC) were performed with a view to obtaining an overview of the potential factors influencing reimbursement. Additionally, these presence of these factors was assessed in the reimbursement dossiers of all Orphan Drugs (n = 64) for which an application for reimbursement was submitted to the National Institute for Health and Disability Insurance in Belgium between January 2002 and July 2013. Results: Different official (i.e. therapeutic value, budget impact, price and impact in clinical practice) and non-official factors (i.e. pricing and reimbursement in other countries, interference by patient organisations and experts, arguments related to quality of branded drug versus compounding, media attention, innovative character, economic importance, ethical arguments and the political climate) may have influenced past reimbursement decisions for Orphan Drugs in Belgium. Discussion: The identification of factors influencing Orphan drug reimbursement is a crucial step in the development of a transparent and consistent framework which will guide future decision-making for reimbursement of Orphan Drugs.

  • do we need authorized Orphan Drugs when compounded medications are available
    Journal of Clinical Pharmacy and Therapeutics, 2013
    Co-Authors: Marc Dooms, Hilde Pince, Steven Simoens
    Abstract:

    Summary What is known and Objective:  Orphan Drugs are used to diagnose, prevent or treat a rare disease. This Commentary aims to present a number of case studies questioning the need for designating compounded medications with a long history of effective use, which is well-supported by published clinical evidence. Comment:  Prior to the market introduction of Orphan Drugs, medication compounding was done in our hospital pharmacy for several rare diseases. Examples include amifampridine for the treatment of Lambert–Eaton myasthenic syndrome (Firdapse®), ibuprofen for the treatment of neonatal patent ductus arteriosus (Pedea®) and zinc acetate for the treatment of Wilson’s disease (Wilzin®). Several ‘non-Orphan’ pharmaceutical products, used off-label for the treatment of rare diseases, that became Orphan medicinal products include Hydrea® for the treatment of sickle-cell syndrome (Siklos®) and Viagra® for the treatment of pulmonary arterial hypertension (Revatio®). What is new and Conclusion:  In our opinion, as indicated by our examples, a better balance should be struck between the development of Orphan Drugs along the recently established regulatory pathways and the pragmatic use of pharmacy-compounded products and evidence-based off-label use of already available commercial products. Societal needs would be best met by focusing Orphan drug development on rare diseases for which there is a high unmet medical need.

  • Ethical, legal and social implications of rare diseases and Orphan Drugs in Europe: meeting report of a Brocher symposium.
    Expert review of pharmacoeconomics & outcomes research, 2013
    Co-Authors: Eline Picavet, David Cassiman, Wim Pinxten, Steven Simoens
    Abstract:

    Ethical, legal and social implications of rare diseases and Orphan Drugs in Europe (Brocher symposium)Geneva, Switzerland 18–19 April 2013As part of the Scientific Program of the Fondation Brocher, a two-day symposium on Orphan Drugs and rare diseases was held on the shores of Lac Leman in Geneva. Specific focus was on the ethical, legal and social implications of rare diseases and Orphan Drugs in Europe. The symposium gathered about 30 international stakeholders and experts, representing different scientific disciplines, the pharmaceutical industry and patient representatives.

  • market uptake of Orphan Drugs a european analysis
    Journal of Clinical Pharmacy and Therapeutics, 2012
    Co-Authors: Eline Picavet, David Cassiman, Irina Cleemput, Lieven Annemans, Steven Simoens
    Abstract:

    Summary What is known and Objective:  Variations in market uptake of an Orphan drug have important implications with respect to access to care and inequality of treatment. Therefore, the aim of this study was to quantify both the sales and volume uptake of Orphan Drugs in Europe and to assess whether a country’s gross domestic product (GDP) and/or health technology assessment (HTA) influences the Orphan Drugs’ market uptake. Methods:  We analysed the numbers of Orphan Drugs launched and the sales and volume uptake for 17 Orphan Drugs in 23 European countries from 2001 until the beginning of 2010 using the IMS Health database. Countries were clustered based on GDP and the availability of a formal HTA-organization. Results and Discussion:  The uptake of Orphan Drugs varied across European countries. The highest volumes and contributions of Orphan Drugs in the first year occurred in countries with a high GDP (and implicitly, a higher budget for healthcare), independently of the existence of an HTA-organization. In contrast, in countries with a low GDP, Orphan Drugs were less available when there was a formal HTA-organization. There, budgetary restrictions can cause the exclusion of less cost-effective Orphan Drugs. What is new and Conclusion:  We observed substantial variation in the market uptake of Orphan Drugs. Such variation may have important implications with respect to access to care and inequality of treatment. The uptake of Orphan Drugs could be promoted through the clinical added value of Orphan Drugs (CAVOD) project and various conditional pricing and reimbursement mechanisms.

Erik Tambuyzer - One of the best experts on this subject based on the ideXlab platform.

  • rare diseases Orphan Drugs and their regulation questions and misconceptions
    Nature Reviews Drug Discovery, 2010
    Co-Authors: Erik Tambuyzer
    Abstract:

    Sustained advocacy efforts driven by patients' organizations to make rare diseases a health priority have led to regulatory and economic incentives for industry to develop Drugs for these diseases, known as Orphan Drugs. These incentives, enacted in regulations first introduced in the United States in 1983 and later in Japan, Europe and elsewhere, have resulted in substantial improvements in the treatment for patients with a range of rare diseases. However, the advent of Orphan drug development has also triggered several questions, from the definition of rarity to the pricing of Orphan Drugs and their impact on health-care systems. This article provides an industry perspective on some of the common questions and misconceptions related to Orphan drug development and its regulation, with the aim of facilitating future progress in the field.

  • Rare diseases, Orphan Drugs and their regulation: questions and misconceptions
    Nature Reviews Drug Discovery, 2010
    Co-Authors: Erik Tambuyzer
    Abstract:

    Regulatory and economic incentives to develop Drugs for rare diseases, known as Orphan Drugs, have resulted in substantial improvements in the treatment for patients with some such diseases. However, the advent of Orphan drug development has also raised several questions, from the definition of rarity, to the pricing of Orphan Drugs and their impact on health-care systems. Tambuyzer considers such questions and related misconceptions with the aim of aiding future progress in the field. Sustained advocacy efforts driven by patients' organizations to make rare diseases a health priority have led to regulatory and economic incentives for industry to develop Drugs for these diseases, known as Orphan Drugs. These incentives, enacted in regulations first introduced in the United States in 1983 and later in Japan, Europe and elsewhere, have resulted in substantial improvements in the treatment for patients with a range of rare diseases. However, the advent of Orphan drug development has also triggered several questions, from the definition of rarity to the pricing of Orphan Drugs and their impact on health-care systems. This article provides an industry perspective on some of the common questions and misconceptions related to Orphan drug development and its regulation, with the aim of facilitating future progress in the field.

Enrique Seoanevazquez - One of the best experts on this subject based on the ideXlab platform.

  • ethical imperatives of timely access to Orphan Drugs is possible to reconcile economic incentives and patients health needs
    Orphanet Journal of Rare Diseases, 2017
    Co-Authors: Rosa Rodriguezmonguio, T. Spargo, Enrique Seoanevazquez
    Abstract:

    More than 6,800 rare diseases and conditions have been identified in the US, which affect 25–30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of Drugs to treat rare diseases and conditions. This study analyzed all Orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to Orphan Drugs. Study data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of Orphan Drugs Development. A search was conducted to assess literature on the ethical principles and economic incentives for the development of Orphan Drugs. In the period 1983–2015, the FDA granted 3,647 Orphan drug designations and 554 Orphan drug approvals. The Orphan drug approvals corresponded to 438 different brand names. Cancer was the therapeutic area with the highest number of approvals. The increased number of patients with rare diseases and the growth in the cost of Orphan Drugs pose a significant economic burden for patients, public programs and private third party payers. Regulatory differences to qualify for Orphan designation and various population thresholds employed by the FDA and the European Medicines Agency lead to further unmet health needs for patients with rare diseases and aggravate health inequities. There is no societal consensus on the population and economic thresholds, the drug effectiveness indicator(s), or the societal value to be placed for the approval and reimbursement of Orphan Drugs. Orphan drug development and marketing in the US concentrate in few therapeutic areas. Despite the increase in the number of FDA approved Orphan Drugs, the unmet needs of patients with rare diseases evidence that the current incentives are not efficiently stimulating Orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of Orphan Drugs without jeopardizing patients’ access to treatment. Thus, aligning pharmaceutical companies’ incentives with societal budgetary constraints is necessary and the ethical imperatives of timely access to Orphan Drugs need to be agreed upon.

L. Hakkaart - One of the best experts on this subject based on the ideXlab platform.

  • International differences in patient access to ultra-Orphan Drugs
    Health policy and technology, 2017
    Co-Authors: Tim A. Kanters, W. Ken Redekop, L. Hakkaart
    Abstract:

    Abstract Objectives Reimbursement recommendations on (Orphan) Drugs are usually made at a national level and this can lead to variation in patient access to the same drug in different countries. We compared differences in patient access to ultra-Orphan Drugs between countries. Furthermore, we describe how reimbursed and non-reimbursed Orphan Drugs differ with respect to pharmacoeconomic properties. Methods We studied patient access to eight high-priced inpatient ultra-Orphan Drugs in nine countries. In addition, we determined whether differences with respect to cost per patient, budget impact and cost-effectiveness existed between Orphan Drugs with a positive and negative reimbursement status. Results Reimbursement status was available for 78 Orphan Drugs, of which 56 (72%) were positive. Large differences were observed between countries; while two countries had a positive status for two out of nine ultra-Orphan Drugs, four countries had positive status for all Drugs it assessed. A number of Drugs were reimbursed only after price negotiations and/or through specific Orphan drug policies. The average cost per patient, budget impact and incremental cost-effectiveness ratios were lower for ultra-Orphan Drugs with a positive reimbursement status than for those with a negative status, although only cost-effectiveness ratios were statistically significant. Conclusions Large differences in patient access to ultra-Orphan Drugs were observed between countries. Future research should examine if similar findings can be seen in other countries and with other Orphan Drugs, and it should also determine which other factors play a role in reimbursement status of Orphan Drugs.

  • Orphan Drugs expenditure in the Netherlands in the period 2006–2012
    Orphanet Journal of Rare Diseases, 2014
    Co-Authors: Tim A. Kanters, Adri Steenhoek, L. Hakkaart
    Abstract:

    Background: The relatively low budget impact of Orphan Drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of Orphan Drugs can still be substantial. In this study, we assess the uptake and budget impact of Orphan Drugs in the Netherlands. Methods: We examined the number of Orphan Drugs, the number of patients and budget impact of Orphan Drugs in the Netherlands in the period 2006 to 2012, both for inpatient and outpatient Orphan Drugs. Budget impact was provided in absolute numbers and relative to total pharmaceutical spending. Results: The number of Orphan Drugs and patients treated increased substantially over the period studied. Overall, budget impact increased substantially over a period of six years, both in absolute terms (326% increase) as well as relative to total pharmaceutical spending (278% increase). Growth rates decreased over time. In 2012, 17% of available Drugs had an individual budget impact of more than €10 million per year. Conclusions: Individual budget impact of Orphan Drugs is often limited, although exceptions exist. However, in total, the budget impact of Orphan Drugs is considerable and has grown substantially over the years. This could potentially influence reimbursement decisions for Orphan Drugs in the future.

  • Orphan Drugs expenditure in the netherlands in the period 2006 2012
    Orphanet Journal of Rare Diseases, 2014
    Co-Authors: Tim A. Kanters, Adri Steenhoek, L. Hakkaart
    Abstract:

    Background: The relatively low budget impact of Orphan Drugs is often used as an argument in reimbursement decisions. However, overall, the budget impact of Orphan Drugs can still be substantial. In this study, we assess the uptake and budget impact of Orphan Drugs in the Netherlands. Methods: We examined the number of Orphan Drugs, the number of patients and budget impact of Orphan Drugs in the Netherlands in the period 2006 to 2012, both for inpatient and outpatient Orphan Drugs. Budget impact was provided in absolute numbers and relative to total pharmaceutical spending. Results: The number of Orphan Drugs and patients treated increased substantially over the period studied. Overall, budget impact increased substantially over a period of six years, both in absolute terms (326% increase) as well as relative to total pharmaceutical spending (278% increase). Growth rates decreased over time. In 2012, 17% of available Drugs had an individual budget impact of more than €10 million per year. Conclusions: Individual budget impact of Orphan Drugs is often limited, although exceptions exist. However, in total, the budget impact of Orphan Drugs is considerable and has grown substantially over the years. This could potentially influence reimbursement decisions for Orphan Drugs in the future.

  • systematic review of available evidence on 11 high priced inpatient Orphan Drugs
    Orphanet Journal of Rare Diseases, 2013
    Co-Authors: Tim A. Kanters, Caroline De Sonnevillekoedoot, Ken W Redekop, L. Hakkaart
    Abstract:

    Background: Attention for Evidence Based Medicine (EBM) is growing, but evidence for Orphan Drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget impact for Orphan Drugs. Methods: A systematic review was performed in PubMed, Embase, NHS EED and HTA databases for 11 inpatient Orphan Drugs listed on the Dutch policy rule on Orphan Drugs. For included studies, we determined the type of study and various study characteristics. Results: A total of 338 studies met all inclusion criteria. Almost all studies (96%) focused on clinical effectiveness of the drug. Of these studies, most studies were case studies (41%) or observational studies (39%). However, for all Orphan diseases at least one experimental or quasi-experimental study was found, and a randomized clinical trial was available for 60% of the Orphan Drugs. Eight studies described the cost-effectiveness of an Orphan drug; an equal number described an Orphan drug’s budget impact. Conclusions: Despite the often heard claim that RCTs are not feasible for Orphan Drugs, we found that an RCT was available in 60% of Orphan Drugs investigated. Cost-effectiveness and budget impact analyses for Orphan Drugs are seldom published.

Christopher Mccabe - One of the best experts on this subject based on the ideXlab platform.

  • value based reimbursement decisions for Orphan Drugs a scoping review and decision framework
    PharmacoEconomics, 2015
    Co-Authors: Mike Paulden, Tania Stafinski, Devidas Menon, Christopher Mccabe
    Abstract:

    Background The rate of development of new Orphan Drugs continues to grow. As a result, reimbursing Orphan Drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing Orphan Drugs at the expense of other health technologies is now recognised as an important input to policy debates.

  • Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework
    PharmacoEconomics, 2015
    Co-Authors: Mike Paulden, Tania Stafinski, Devidas Menon, Christopher Mccabe
    Abstract:

    Background The rate of development of new Orphan Drugs continues to grow. As a result, reimbursing Orphan Drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing Orphan Drugs at the expense of other health technologies is now recognised as an important input to policy debates. Objectives The aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of Orphan Drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources. Methods A scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making. Results The scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or ‘opportunity cost’-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework. Conclusion Our framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for Orphan Drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.

  • Orphan Drugs revisited
    QJM: An International Journal of Medicine, 2006
    Co-Authors: Christopher Mccabe, Aki Tsuchiya, Karl Claxton, James Raftery
    Abstract:

    Hughes et al .1 recently discussed arguments for and against giving special funding status to Orphan Drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive. The aims of this paper are to correct some inaccuracies in the original paper, develop some of the key issues, and to draw some conclusions regarding the question ‘Do Drugs for exceptionally rare disease deserve special status for funding?’ For ease, our paper adopts the same structure as the original. Hughes et al . state that a key issue is ‘whether the rarity and gravity of the condition represents a rational basis for applying a different value to health gain …’1 The defining characteristic of an Orphan drug is that it treats a rare disease. However, the justification for special funding frequently rests upon the ‘gravity’ of the condition. To examine whether Orphan drug legislation accurately represents societal preferences, it would be necessary to ask whether society was willing to pay more for treatments for rare severe disorders than for more prevalent severe disorders. No study has done this. Hughes et al . recount another frequently cited argument for special treatment: ‘ensuring access to treatment where no other treatment exists.’ Like ‘gravity’, this is not a defining characteristic of an Orphan drug, but it is a frequently cited argument for their special status in licensing and reimbursement.1 Not being unique to Orphan Drugs, it cannot be a justification for their special status. Further, this argument contains an implicit preference for biological disease modification over health gain. In the developed world, ‘no other treatment’ is a substantial misrepresentation of reality; patients are not simply left with no medical treatment at …

  • Orphan Drugs and the nhs should we value rarity
    BMJ, 2005
    Co-Authors: Christopher Mccabe, Karl Claxton, Aki Tsuchiya
    Abstract:

    The growing number and costs of Drugs for rare diseases are straining healthcare budgets. Decisions on funding these treatments need to be made on a sound basis Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (Orphan Drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients. Several countries have put measures in place to safeguard research and development of Orphan Drugs, but few get close to meeting the cost effectiveness criteria for funding by healthcare providers. We examine the justifications for special status for rare diseases and ask whether the cost effectiveness of Drugs for rare or very rare diseases should be treated differently from that of other Drugs and interventions. The citizen's council of the National Institute for Health and Clinical Excellence (NICE) was recently asked to consider whether the NHS should be prepared to pay premium prices for Drugs to treat patients with very rare diseases.1 It recommended that the NHS should consider paying premium prices based on three criteria: the severity of the disease, evidence of health gain, and whether the disease is life threatening.1 The decision by the Department of Health to ring fence funding for enzyme replacement therapy for lysosomal disorders, with expected annual costs above £100 000 ($180 000, €150 000) per patient for life, suggests that central government also currently believes that premium prices should be paid.2 NICE has conducted a feasibility study to explore whether its current processes and methods of technology appraisal can be applied to the appraisal of ultra-Orphan Drugs (those for diseases with a prevalence of 0.18/10 000 or less).3 It has not yet stated whether it will recommend that treatments for very rare diseases should have special …