Assessing the economic challenges posed by orphan drugs

2007 ◽  
Vol 23 (1) ◽  
pp. 36-42 ◽  
Author(s):  
Michael F. Drummond ◽  
David A. Wilson ◽  
Panos Kanavos ◽  
Peter Ubel ◽  
Joan Rovira
Keyword(s):  
Rare Diseases ◽  
Research Agenda ◽  
Cost Effective ◽  
Health Technology ◽  
Orphan Drugs ◽  
Patient Access ◽  
Standard Methods ◽  
Societal Value ◽  
Small Market ◽  
Societal Preferences

Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions, specific legislation has been enacted to encourage the development of drugs for rare diseases (orphan drugs), which would otherwise not be commercially viable. However, because of the small market, these drugs are often very expensive. Under the standard methods of health technology assessment (HTA) incorporating economic evaluation, orphan drugs do not usually prove to be cost-effective and this, coupled with their high cost, means that funding and patient access may be limited. However, these restrictions may not be in line with societal preferences. Therefore, this study discusses whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs and outlines a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.

Assessing the economic challenges posed by orphan drugs: A comment on Drummond et al.

2007 ◽  
Vol 23 (3) ◽  
pp. 397-401 ◽  
Keyword(s):  
Research Agenda ◽  
Orphan Drugs ◽  
Patient Access ◽  
Standard Methods ◽  
Societal Value

To the Editor:In a recent study in this Journal, Drummond and colleagues (5) attempt to address the question “Whether the standard methods of HTA are adequate for assisting decisions on patient access to and funding of orphan drugs” before proposing “a research agenda to help understand the societal value of orphan drugs and issues surrounding their development, funding, and use.”

scholarly journals Building a trusted framework for uncertainty assessment in rare diseases: suggestions for improvement (Response to “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases”)

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Sabine E. Grimm ◽  
Xavier Pouwels ◽  
Bram L. T. Ramaekers ◽  
Ben Wijnen ◽  
Saskia Knies ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Research Agenda ◽  
Health Technology ◽  
Orphan Drugs ◽  
Uncertainty Assessment ◽  
Letter To The Editor ◽  
Research Prioritisation ◽  
Evidence Generation

AbstractThe aim of this letter to the editor is to provide a comprehensive summary of uncertainty assessment in Health Technology Assessment, with a focus on transferability to the setting of rare diseases. The authors of “TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases” presented recommendations for reducing uncertainty in rare diseases. Their article is of great importance but unfortunately suffers from a lack of references to the wider uncertainty in Health Technology Assessment and research prioritisation literature and consequently fails to provide a trusted framework for decision-making in rare diseases. In this letter to the editor we critique the authors’ tool and provide pointers as to how their proposal can be strengthened. We present references to the literature, including our own tool for uncertainty assessment (TRUST; unrelated to the authors’ research), apply TRUST to two assessments of orphan drugs in rare diseases and provide a broader perspective on uncertainty and risk management in rare diseases, including a detailed research agenda.

PP452 Impact Of Patient Access Schemes On Health Technology Assessment Agency Guidance For Rare Diseases In England and Scotland

2021 ◽  
Vol 37 (S1) ◽  
pp. 38-38
Author(s):  
Karen Mark ◽  
Prachi Manchanda ◽  
Judith Rubinstein ◽  
Riza Veronica Inumerable
Keyword(s):  
Cost Effectiveness ◽  
Health Technology Assessment ◽  
Incremental Cost ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Incremental Cost Effectiveness Ratio ◽  
Base Case ◽  
Patient Access ◽  
Cost Effectiveness Ratio

IntroductionPatient access schemes (PAS) are agreements that may enable patients to access drugs or other treatments that may not be cost effective under normal circumstances. The aim of this study was to determine whether the use of PAS by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for recommended drugs can lead to greater access to medications for rare diseases.MethodsReimbursement data for rare diseases between 2004 and 2021 from health technology assessment (HTA) agencies, namely the SMC (Scotland) and NICE (England), were included. The reviews with positive HTA decisions were considered, while those with negative decisions were excluded. Several observations were made from these data and reported.ResultsAmong the total positive reviews (n = 81), 43 included PAS. The inclusion of PAS in manufacturer submissions was more frequent for NICE than for the SMC (79% and 40% percent, respectively). Most of the drugs with PAS were included in the HTA guidance from both agencies. The positive NICE reviews contingent on PAS consisted of 20 drugs. For the same set of drugs, the SMC recommended 14 with PAS and one without PAS; five drugs were not assessed. Adalimumab was recommended by NICE with a PAS (base-case incremental cost-effectiveness ratio of GBP12,336 [EUR14,256]; GBP13,676 [EUR15,804]) and by the SMC without a PAS (base-case incremental cost-effectiveness ratio of GBP22,519 [EUR26,023]). Hence, without a PAS, the drug was costlier per quality-adjusted life-year for the National Health Service (NHS) Scotland.ConclusionsPAS submissions for rare diseases are more frequent for NICE than for the SMC. With the PAS discounts, the overall cost of the drugs is reduced, resulting in cost effectiveness. The SMC approved some drugs for which NICE required a PAS to improve the economic argument. Hence, the use of PAS for these drugs could lead to potential cost-savings to the NHS Scotland.

scholarly journals Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making

2017 ◽  
Vol 37 (5) ◽  
pp. 567-576 ◽  
Author(s):  
Johanna Wiss ◽  
Lars-Ake Levin ◽  
David Andersson ◽  
Gustav Tinghög
Keyword(s):  
Rare Disease ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
Postal Survey ◽  
Psychological Effects ◽  
Medical Decision ◽  
Common Disease ◽  
Common Diseases ◽  
General Preference ◽  
Societal Preferences

Background. Measuring societal preferences for rarity has been proposed to determine whether paying premium prices for orphan drugs is acceptable. Objective. To investigate societal preferences for rarity and how psychological factors affect such preferences. Method. A postal survey containing resource allocation dilemmas involving patients with a rare disease and patients with a common disease, equal in severity, was sent out to a randomly selected sample of the population in Sweden (return rate 42.3%, n = 1270). Results. Overall, we found no evidence of a general preference for prioritizing treatment of patients with rare disease patients over those with common diseases. When treatment costs were equal, most respondents (42.7%) were indifferent between the choice options. Preferences for prioritizing patients with common diseases over those with rare diseases were more frequently displayed (33.3% v. 23.9%). This tendency was, as expected, amplified when the rare disease was costlier to treat. The share of respondents choosing to treat patients with rare diseases increased when presenting the patients in need of treatment in relative rather than absolute terms (proportion dominance). Surprisingly, identifiability did not increase preferences for rarity. Instead, identifying the patient with a rare disease made respondents more willing to prioritize the patients with common diseases. Respondents’ levels of education were significantly associated with choice—the lower the level of education, the more likely they were to choose the rare option. Conclusions. We find no support for the existence of a general preference for rarity when setting health care priorities. Psychological effects, especially proportion dominance, are likely to play an important role when preferences for rarity are expressed.

Do Drugs for Special Populations Warrant Higher Prices?

2021 ◽  
pp. 151-172
Author(s):  
Neumann Peter J. ◽  
Cohen Joshua T. ◽  
Ollendorf Daniel A
Keyword(s):  
Cost Effectiveness ◽  
Rare Diseases ◽  
Health Technology ◽  
Orphan Drugs ◽  
Special Populations ◽  
Value Assessment ◽  
Manufacturing Costs ◽  
Gene Therapies ◽  
Run Up ◽  
Additional Value

Despite its widespread acceptance, cost-effectiveness analysis, which examines an intervention’s incremental cost per unit of health gained, can run up against intuition in the context of drug coverage for conditions affecting special populations. In particular, the “rule of rescue” (i.e., spending more for populations perceived to be in immediate and great peril) may be justified for certain conditions. Examples include rare diseases and the “orphan drugs” used to treat them; cancer, with its attendant societal dread; and pediatric conditions, which are often inherited and severe. Cell and gene therapies may also challenge traditional value assessment, given their high manufacturing costs and potential for one-time, curative treatment. Health technology assessment (HTA) bodies worldwide have made exceptions for these populations by comparing cost-effectiveness ratios to higher (less stringent) benchmarks and by incorporating additional value considerations. Still, evaluating these higher-priced therapies remains a challenge for HTA and society.

OP100 How Health Technology Assessment Is Adapting To Orphan Drugs In Canada – Not!

2017 ◽  
Vol 33 (S1) ◽  
pp. 45-46
Author(s):  
Durhane Wong-Rieger ◽  
Ferg Mills
Keyword(s):  
Health Technology Assessment ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Unmet Need ◽  
Health Technology ◽  
Orphan Drugs ◽  
Evaluation Framework ◽  
Lymphocytic Leukemia ◽  
Primary Analysis ◽  
Small Patient Population

INTRODUCTION:Some countries have distinct pathways for drugs for rare diseases (DRDs) (1). In May 2014, the Canadian Agency for Technologies in Health (CADTH) rejected the option of a separate review pathway for DRDs, reiterating that “pharmacoeconomic analyses are critical for all types of drugs”. While the gap between positive recommendations for common and rare drugs may have narrowed, the rejection for DRDs is still proportionally much higher (2). The default has been to provincially negotiate drug access, for patient populations, subgroups or individuals. Still not wishing to create a separate pathway, in March 2016, CADTH produced a revised evaluation framework for “uncertain clinical and pharmacoeconomic evidence” and other considerations representing “significant unmet need” including rarity and difficulty to study because of small patient population”(3). This study analyzes recommendations for DRDs following the two CADTH revisions.METHODS:Methods used were: synthesis of previously conducted analyses of CADTH recommendations for rare and non-rare drugs, primary comparative analysis of CADTH recommendations for DRDs from 2004 to 2016, and qualitative analysis of two drugs submitted for both rare and non-rare conditions: everolimus (breast cancer, pancreatic neuroendocrine tumours, and tuberous sclerosis complex) and ibrutinib (chronic lymphocytic leukemia, small lymphocytic lymphoma, and Waldenström's Macroglobulinemia).RESULTS:Previous analyses found that DRDs received more negative recommendations than did non-rare drugs; both clinical and economic evidence were differentiating factors. The primary analysis provided an additional understanding of reasons for negative recommendations. There is low consistency across assessments and across the two CADTH review committees. The case studies illustrated the challenges for DRDs to overcome barriers of cost-effectiveness and certainty of clinical evidence, even with the revised framework.CONCLUSIONS:This research challenges the premise that Health Technology Assessment for all drugs can result in fair and equitable recommendations for DRDs. Moreover, assessments based on “significant unmet need” do not appear to provide consistent or equitable guidelines for addressing the issues specific to rare diseases.

scholarly journals The application of multi-criteria decision analysis to inform in resource allocation

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 445
Author(s):  
Carina Schey ◽  
Maarten Postma ◽  
Paul Krabbe ◽  
Goran Medic ◽  
Mark Connolly
Keyword(s):  
Decision Analysis ◽  
Health Technology Assessment ◽  
Disease Severity ◽  
Rare Diseases ◽  
Technology Assessment ◽  
Health Technology ◽  
Orphan Drugs ◽  
Analysis Framework ◽  
Multi Criteria Decision Analysis ◽  
Annual Treatment

Background: There is a perception held by payers that orphan products are expensive. As a result, the current health technology assessment systems might be too restrictive for orphan drugs, therefore potentially denying patients access to life-saving medicines. While price is important, it should be considered in relation to a broader range of disease-related product attributes that are not necessarily considered by many health technology assessment agencies. To overcome these challenges, multi-criteria decision analysis has been proposed as an alternative to evaluate technologies. Methods: A targeted literature review was conducted to identify the most frequently cited attributes in multi-criteria decision analysis (MCDA) in rare diseases. From the leading attributes identified, we developed a multi-criteria decision analysis framework with which to aggregate the orphan drug values. We subsequently reviewed and plotted the relationship between single attributes and the average annual treatment costs for 8 drugs used in the treatment of rare endocrine diseases. The annual treatment costs were based on UK list prices for the average daily dose per patient. Results: The five most frequently mentioned attributes in the literature were as follows: Disease severity, Unmet need (or availability of therapeutic alternatives), Comparative effectiveness or efficacy, Quality of evidence and Safety & tolerability. Results from the multi-criteria decision analysis framework indicate a wide range of average annual per-patients costs for drugs intended for the same diseases, and likewise for diseases with a similar level of Disease severity. Conclusions: Multi-criteria decision analysis may offer a viable alternative to support discussion in reimbursement decisions for orphan drugs. The analyses can be used to inform investigations on the application of MCDAs in rare diseases.

Comparison and analysis of the fda approved orphane drugs registerand the vital and essential drugs list of the Russian Federation

2020 ◽  
pp. 4-16
Author(s):  
D.S. Yurochkin ◽  
◽  
A.A. Leshkevich ◽  
Z.M. Golant ◽  
I.A. NarkevichSaint ◽  
...  
Keyword(s):  
United States ◽  
Russian Federation ◽  
Rare Diseases ◽  
Orphan Drugs ◽  
The United States ◽  
Orphan Diseases ◽  
Essential Drugs ◽  
The Russian Federation ◽  
Comparison And Analysis ◽  
The Government

The article presents the results of a comparison of the Orphan Drugs Register approved for use in the United States and the 2020 Vital and Essential Drugs List approved on October 12, 2019 by Order of the Government of the Russian Federation No. 2406-r. The comparison identified 305 international non-proprietary names relating to the main and/or auxiliary therapy for rare diseases. The analysis of the market of drugs included in the Vital and Essential Drugs List, which can be used to treat rare (orphan) diseases in Russia was conducted.

scholarly journals Pricing of orphan drugs in oncology and rare diseases

2020 ◽  
Vol 8 (1) ◽  
pp. 1838191
Author(s):  
Mark Nuijten ◽  
Stefano Capri
Keyword(s):  
Rare Diseases ◽  
Orphan Drugs
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