Orphan drugs and the NHS: should we value rarity?
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ABSTRACT:
SUBMITTER: McCabe C
PROVIDER: S-EPMC1273462 | biostudies-literature | 2005 Oct
REPOSITORIES: biostudies-literature
Publications
Orphan drugs and the NHS: should we value rarity?
McCabe Christopher C Claxton Karl K Tsuchiya Aki A
BMJ (Clinical research ed.) 20051001 7523
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Project description: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.
| S-EPMC5405566 | biostudies-literature
Project description:BackgroundThe 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.ObjectivesThe 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.MethodsA 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.ResultsThe 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.ConclusionOur 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.
| S-EPMC4342524 | biostudies-literature
Project description:According to global annual estimates hunger/malnutrition is the major cause of death (36 of 62 million). Cardiovascular diseases and cancer (5.44 of 13.43 million) are the major causes of death in developed countries, while lower respiratory tract infections, human immunodeficiency virus infection/acquired immunodeficiency syndrome, diarrhoeal disease, malaria and tuberculosis (10.88 of 27.12 million) are the major causes of death in developing countries with more than 70% of deaths occurring in children. The majority of approximately 800 million people with other rare diseases, including 100000 children born with thalassaemia annually receive no treatment. There are major ethical dilemmas in dealing with global health issues such as poverty and the treatment of orphan and rare diseases. Of approximately 50000 drugs about 10% are orphan drugs, with annual sales of the latter approaching 100 billion USD. In comparison, the annual revenue in 2009 from the top 12 pharmaceutical companies in Western countries was 445 billion USD and the top drug, atorvastatin, reached 100 billion USD. In the same year, the total government expenditure for health in the developing countries was 410 billion USD with only 6%-7% having been received as aid from developed countries. Drugs cost the National Health Service in the United Kingdom more than 20 billion USD or 10% of the annual health budget. Uncontrollable drug prices and marketing policies affect global health budgets, clinical practice, patient safety and survival. Fines of 5.3 billion USD were imposed on two pharmaceutical companies in the United States, the regulatory authority in France was replaced and clinicians were charged with bribery in order to overcome recent illegal practises affecting patient care. High expenditure for drug development is mainly related to marketing costs. However, only 2 million USD was spent developing the drug deferiprone (L1) for thalassaemia up to the stage of multicentre clinical trials. The criteria for drug development, price levels and use needs to be readdressed to improve drug safety and minimise costs. New global health policies based on cheaper drugs can help the treatment of many categories of orphan and rare diseases and millions of orphan patients in developing and developed countries.
| S-EPMC4202455 | biostudies-literature
Project description:BackgroundOrphan drugs offer important therapeutic options to patients suffering from rare conditions, but are typically considerably more expensive than non-orphan drugs, leading to questions about their cost-effectiveness.ObjectiveTo compare the value of orphan and non-orphan drugs approved by the FDA from 1999 through 2015.DesignWe searched the PubMed database to identify estimates of incremental health gains (measured in quality-adjusted life-years, or QALYs) and incremental costs that were associated with orphan and non-orphan drugs compared with preexisting care. We excluded pharmaceutical industry-funded studies from the dataset. When a drug was approved for multiple indications, we considered each drug-indication pair separately. We then compared incremental QALY gains, incremental costs, and incremental cost-effectiveness ratios for orphan and non-orphan drugs using the Mann-Whitney U (MWU) test (to compare median values of the different distributions) and the Kolmogorov-Smirnov (KS) test (to compare the shape of different distributions).ResultsWe identified estimates for 49 orphan drug-indication pairs, and for 169 non-orphan drug-indication pairs. We found that orphan drug-indication pairs offered larger median incremental health gains than non-orphan drug-indication pairs (0.25 vs. 0.05 QALYs; MWU p = 0.0093, KS p = 0.02), but were associated with substantially higher costs ($47,652 vs. $2870; MWU p < 0.001, KS p < 0.001) and less favorable cost-effectiveness ($276,288 vs. $100,360 per QALY gained; MWU p = 0.0068, KS p = 0.009).ConclusionsOur study suggests that orphan drugs often offer larger health gains than non-orphan drugs, but due to their substantially higher costs they tend to be less cost-effective than non-orphan drugs. Our findings highlight the challenge faced by health care payers to provide patients appropriate access to orphan drugs while achieving value from drug spending.
| S-EPMC7458970 | biostudies-literature
Project description:BackgroundThe National Institute for Health and Care Excellence (NICE) is responsible for ensuring that patients in England and Wales can access clinically and cost-effective treatments. However, NICE's processes pose significant reimbursement challenges for treatments for rare diseases. While some orphan medicines have been appraised via the highly specialised technology route, most are appraised via the single technology appraisal programme, a route that is expected to be increasingly used given new more restrictive highly specialised technology criteria. This often results in delays to access owing to differences in applicable thresholds and the single technology appraisal approach being ill-equipped to deal with the inevitable decision uncertainty. NICE recently published their updated methods and process manual, which includes a new severity-of-disease modifier and an instruction to be more flexible when considering uncertainty in rare diseases. However, as the threshold gap between the single technology appraisal and highly specialised technology programmes remains, it is unlikely that these changes alone will address the problem.ObjectiveWe explored the potential impact of quality-adjusted life-year weights in decision making.MethodsWe explored the impact of NICE's new severity-of-disease modifier weighting and two alternative methods (the use of alternative quality-adjusted life-year weights and the fair rate of return), using three recent single technology appraisals of orphan medicines (caplacizumab, teduglutide and pirfenidone for mild idiopathic pulmonary fibrosis).ResultsOur results suggest NICE's severity-of-disease modifier would not have affected the recommendations. Using alternative methods, based upon achievement of an incremental cost-effectiveness ratio below standard thresholds, patients could have received access to caplacizumab approximately 5 months earlier, and the appraisals for teduglutide and pirfenidone would have resulted in a positive recommendation following appraisal consultation meeting 1 when neither of these products was available over 5 years from the initial submission.ConclusionUltimately, moving from a restrictive end-of-life modifier to one based on disease severity is a more equitable approach likely to benefit many therapies, including orphan products. However, NICE's single technology appraisal updates are unlikely to result in faster reimbursement of orphan medicines, nor will they address concerns around market access for orphan medicines in the UK.
| S-EPMC10043140 | biostudies-literature
Project description:Background and aimsOrphan diseases, or rare diseases, are defined in Europe as diseases that affect less than 5 out of every 10,000 citizens. Given the small number of cases and the lack of profit potential, pharmaceutical companies have not invested much in the development of possible treatments. However, over the last few years, new therapies for rare diseases have emerged, giving physicians a chance to offer personalized treatment. With this paper, we aim to present some of the orphan neurological diseases for which new drugs have been developed lately.MethodsWe have conducted a literature review of the papers concerning rare diseases and their treatment, and we have analyzed the existing studies for each orphan drug. For this purpose, we have used the Google Scholar search engine and the Orphanet. We have selected the studies published in the last 15 years.ResultsSince the formation of the National Organization for Rare Diseases, the Orphan Drug Act, and the National Institutes of Health Office of Rare Diseases, pharmacological companies have made a lot of progress concerning the development of new drugs. Therefore, diseases that until recently were without therapeutic solutions benefit today from personalized treatment. We have detailed in our study over 15 neurological and systemic diseases with neurological implications, for which the last 10-15 years have brought important innovations regarding their treatment.ConclusionsMany steps have been taken towards the treatment of these patients, and the humanity and professionalism of the pharmaceutical companies, along with the constant support of the patient's associations for rare diseases, have led to the discovery of new treatments and useful future findings.
| S-EPMC10058745 | biostudies-literature
Project description:BackgroundHigh orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse.MethodsUsing publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.ResultsWe found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug.ConclusionsMore discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.
| S-EPMC6327525 | biostudies-literature
Project description:The Orphan Drug Act of 1983 provides benefits to promote the development of treatments for rare diseases that have limited sales potential. Policy makers have questioned whether this purpose is furthered in the case of "partial orphan drugs" approved to treat both rare and common diseases, as many of these drugs are top sellers. In this study we used national commercial claims data to estimate the proportion of spending in the US on fifteen top-selling partial orphan drugs that was assigned to orphan indications in 2018. Of this spending, 21.4 percent was assigned to orphan indications, 70.7 percent to nonorphan indications, and 7.9 percent to neither orphan nor nonorphan indications (for example, off-label use). These findings support growing concerns regarding the costs of granting orphan drug benefits to the sponsors of top-selling partial orphan drugs.
| S-EPMC8011952 | biostudies-literature
Project description:BackgroundGlobal Engagement works with health partnerships to establish workforce and educational translation on a global scale to support the National Health Service (NHS). There is growing evidence on how international experiences (through volunteering, exchanges and placements) benefit the NHS through an innovative workforce that develops international best practice and promotes lifelong learning. Most of this evidence has been captured though surveys to returned international volunteers. However, there is limited evidence about how to quantify the value that returned international healthcare volunteers bring back to their country of residence.MethodsThis paper identifies the various benefits to the NHS from returned international healthcare volunteers. The outcomes from returned international volunteers, which have been identified as relevant form a NHS perspective, are linked to three key areas in a multisector analytical framework used by the World Bank to evaluate labour market programmes: (1) Investment climate and Infrastructure, (2) Labor market regulations and institutions, and (3) Education and skills development. The monetary value of these outcomes is quantified through productivity indices which capture the economic value that the achievement of these outcomes have on the quality of the NHS labor force. This model is applied to a dataset of international volunteers provided by the Global Engagement health partnerships.ResultsThe results suggest that international volunteering generates average productivity gains of up to 37% for doctors and up to 62% for nurses. Average productivity gains estimated from health partnerships data vary depending on duration of volunteering periods and occupational category mix.ConclusionsOur analysis offers a value for money rationale for international volunteering programmes purely from a domestic and NHS perspective. The valuation method considers only one of the aims of international volunteering programmes: the development of the existing and future NHS workforce. Broader benefits for health system strengthening at a global level are acknowledged but not accounted for. Overall, we conclude that if the acquisition of volunteering outcomes is realised, the NHS can accrue a productivity increase of between 24 and 41% per volunteer, with a value ranging from £13,215 to £25,934 per volunteer.
| S-EPMC6480499 | biostudies-literature
Project description:ObjectiveWe evaluated the uptake of medicines licensed as orphan drugs by the United States Food and Drug Administration (FDA) or European Medicines Agency (EMA) into the WHO Model list of essential medicines and the WHO Model list of essential medicines for children from 1977 to 2021.MethodsWe collated and analysed data on drug characteristics, reasons for adding or rejecting medicines, and time between regulatory approval and inclusion in the lists. We compared trends in listing orphan drugs before and after revisions to the inclusion criteria of the essential medicines lists in 2001, as well as differences in trends for listing orphan and non-orphan drugs, respectively.FindingsThe proportion of orphan drugs in the essential medicines lists increased from 1.9% (4/208) in 1977 to 14.6% (70/478) in 2021. While orphan drugs for communicable diseases have remained stable over time, we observed a considerable shift towards more orphan drugs for noncommunicable diseases, particularly for cancer. The median period for inclusion in the essential medicines lists after either FDA or EMA first approval was 13.5 years (range: 1-28 years). Limited clinical evidence base and uncertainty about the magnitude of net benefit were the most frequent reasons to reject proposals to add new orphan drugs to the essential medicines lists.ConclusionDespite lack of a global definition of rare diseases, the essential medicines lists have broadened their scope to include medicines for rare conditions. However, the high costs of many listed orphan drugs pose accessibility and reimbursement challenges in resource-constrained settings.
| S-EPMC10753278 | biostudies-literature