Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients. This study aims to conduct ...a literature review to provide insight into the drivers of orphan drug pricing and reimbursement. Although orphan drug pricing follows the same economic logic as drug pricing in general, the monopolistic power of orphan drugs results in high prices: a) orphan drugs benefit from a period of marketing exclusivity; b) few alternative health technologies are available; c) third-party payers and patients have limited negotiating power; d) manufacturers attempt to maximise orphan drug prices within the constraints of domestic pricing and reimbursement policies; and e) substantial R&D costs need to be recouped from a small number of patients. Although these conditions apply to some orphan drugs, they do not apply to all orphan drugs. Indeed, the small number of patients treated with an orphan drug and the limited economic viability of orphan drugs can be questioned in a number of cases. Additionally, manufacturers have an incentive to game the system by artificially creating monopolistic market conditions. Given their high price for an often modest effectiveness, orphan drugs are unlikely to provide value for money. However, additional criteria are used to inform reimbursement decisions in some countries. These criteria may include: the seriousness of the disease; the availability of other therapies to treat the disease; and the cost to the patient if the medicine is not reimbursed. Therefore, the maximum cost per unit of outcome that a health care payer is willing to pay for a drug could be set higher for orphan drugs to which society attaches a high social value. There is a need for a transparent and evidence-based approach towards orphan drug pricing and reimbursement. Such an approach should be targeted at demonstrating the relative effectiveness, cost-effectiveness and economic viability of orphan drugs with a view to informing pricing and reimbursement decisions.
Background
In an era of scarce resources, policy makers, neurologists and other stakeholders need to be aware of the economic burden of multiple sclerosis and the cost-effectiveness of ...disease-modifying therapies. The aim of this article is to provide a mini-review of these health economic facets of multiple sclerosis.
Methods
An umbrella review was conducted by searching PubMed and Google Scholar from 2002 until June 2022 for peer-reviewed systematic and narrative literature reviews.
Results
An extensive body of evidence corroborates that multiple sclerosis is associated with a substantial economic burden within and outside the health care sector, that costs of secondary progressive multiple sclerosis exceed those of relapsing-remitting multiple sclerosis, that costs increase with disease severity and are influenced by the occurrence of relapses and therapy adherence. However, cost estimates and their breakdown into various components vary between countries. Economic evaluations show that disease-modifying therapies for relapsing-remitting multiple sclerosis are generally not cost-effective, but these results depend on the local setting. Cost-effectiveness of disease-modifying therapies improves when a societal perspective is taken and efficacy does not wane over a lifetime horizon, when oral administration forms or dosing strategies requiring less maintenance are introduced, and when generic versions enter the market. Reimbursement recommendations related to disease-modifying therapies also differ between countries.
Conclusion
The local context matters when calculating the societal economic burden of multiple sclerosis and the cost-effectiveness of disease-modifying therapies.
Given that biosimilars are agents that are similar but not identical to the reference biopharmaceutical, this study aims to introduce and describe specific issues related to the economic evaluation ...of biosimilars by focusing on the relative costs, relative effectiveness, and cost-effectiveness of biosimilars. Economic evaluation assesses the cost-effectiveness of a medicine by comparing the costs and outcomes of a medicine with those of a relevant comparator. The assessment of cost-effectiveness of a biosimilar is complicated by the fact that evidence needed to obtain marketing authorization from a registration authority does not always correspond to the data requirements of a reimbursement authority. In particular, this relates to the availability of adequately powered equivalence or noninferiority studies, the need for comparative data about the effectiveness in a real-world setting rather than the efficacy in a structured setting, and the use of health outcome measures instead of surrogate endpoints. As a biosimilar is likely to be less expensive than the comparator (eg, the reference biopharmaceutical), the assessment of the cost-effectiveness of a biosimilar depends on the relative effectiveness. If appropriately designed and powered clinical studies demonstrate equivalent effectiveness between a biosimilar and the comparator, then a cost-minimization analysis identifies the least expensive medicine. If there are differences in the effectiveness of a biosimilar and the comparator, other techniques of economic evaluation need to be employed, such as cost-effectiveness analysis or cost-utility analysis. Given that there may be uncertainty surrounding the long-term safety (ie, risk of immunogenicity and rare adverse events) and effectiveness of a biosimilar, the cost-effectiveness of a biosimilar needs to be calculated at multiple time points throughout the life cycle of the product.
This paper aims to assess the methodological quality of economic evaluations included in Belgian reimbursement applications for Class 1 drugs.
For 19 reimbursement applications submitted during 2011 ...and Spring 2012, a descriptive analysis assessed the methodological quality of the economic evaluation, evaluated the assessment of that economic evaluation by the Drug Reimbursement Committee and the response to that assessment by the company. Compliance with methodological guidelines issued by the Belgian Healthcare Knowledge Centre was assessed using a detailed checklist of 23 methodological items. The rate of compliance was calculated based on the number of economic evaluations for which the item was applicable.
Economic evaluations tended to comply with guidelines regarding perspective, target population, subgroup analyses, comparator, use of comparative clinical data and final outcome measures, calculation of costs, incremental analysis, discounting and time horizon. However, more attention needs to be paid to the description of limitations of indirect comparisons, the choice of an appropriate analytic technique, the expression of unit costs in values for the current year, the estimation and valuation of outcomes, the presentation of results of sensitivity analyses, and testing the face validity of model inputs and outputs. Also, a large variation was observed in the scope and depth of the quality assessment by the Drug Reimbursement Committee.
Although general guidelines exist, pharmaceutical companies and the Drug Reimbursement Committee would benefit from the existence of a more detailed checklist of methodological items that need to be reported in an economic evaluation.
Celotno besedilo
Dostopno za:
DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
Drug shortages are a complex and global phenomenon. When a drug cannot be delivered at the moment of patient demand, every stakeholder in the health care system is affected. The aim of this study was ...to investigate the characteristics, clinical impact, financial impact and management of drug shortages in European hospital pharmacies and identify opportunities for prevention and mitigation of drug shortages in Europe. An online survey was designed based on a review of the literature and interviews and was sent to subscribers of Hospital Pharmacy Europe between June and September 2013. Forty-five percent of respondents (n = 161) indicated that life sustaining or life preserving drugs such as oncology drugs were affected by drug shortages. More than 30% of respondents indicated that drug shortages in Europe were always or often associated with increased costs for hospitals, increased personnel costs and more expensive alternative drugs (n = 161). On the question when information about a drug shortage was obtained, 42% of respondents answered that information from the pharmaceutical company was obtained at the time of no delivery, 50% indicated that information from the wholesaler was obtained at the time of no delivery, while 40% of respondents indicated that information was never or rarely received from the government (n = 161). Fifty seven percent of respondents strongly agreed that an obligation to the producer to notify further shortages could help to solve the problem (n = 161). These results showed that pharmaceutical companies and wholesalers are already involved in the management of drug shortages, while a role is still reserved for the government. Mandatory notification in advance and centralized information can help to reduce workload for hospital pharmacists, will allow early anticipation of drug shortages and will facilitate mitigation of the clinical impact on patients.
Celotno besedilo
Dostopno za:
DOBA, IZUM, KILJ, NUK, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK
Background:
Decision-makers have implemented a variety of value assessment frameworks (VAFs) for orphan drugs in European jurisdictions, which has contributed to variations in access for rare disease ...patients. This review provides an overview of the strengths and limitations of VAFs for the reimbursement of orphan drugs in Europe, and may serve as a guide for decision-makers.
Methods:
A narrative literature review was conducted using the databases Pubmed, Scopus and Web of Science. Only publications in English were included. Publications known to the authors were added, as well as conference or research papers, or information published on the website of reimbursement and health technology assessment (HTA) agencies. Additionally, publications were included through snowballing or focused searches.
Results:
Although a VAF that applies a standard economic evaluation treats both orphan drugs and non-orphan drugs equally, its focus on cost-effectiveness discards the impact of disease rarity on data uncertainty, which influences an accurate estimation of an orphan drug’s health benefit in terms of quality-adjusted life-years (QALYs). A VAF that weighs QALYs or applies a variable incremental cost-effectiveness (ICER) threshold, allows the inclusion of value factors beyond the QALY, although their methodologies are flawed. Multi-criteria decision analysis (MCDA) incorporates a flexible set of value factors and involves multiple stakeholders’ perspectives. Nevertheless, its successful implementation relies on decision-makers’ openness toward transparency and a pragmatic approach, while allowing the flexibility for continuous improvement.
Conclusion:
The frameworks listed above each have multiple strengths and weaknesses. We advocate that decision-makers apply the concept of accountability for reasonableness (A4R) to justify their choice for a specific VAF for orphan drugs and to strive for maximum transparency concerning the decision-making process. Also, in order to manage uncertainty and feasibility of funding, decision-makers may consider using managed-entry agreements rather than implementing a separate VAF for orphan drugs.
Given that antibiotic use is associated with externalities, standard economic evaluation which considers costs and health gains accruing to patients under-values antibiotics. Informed by a scoping ...review, this discussion paper aims to identify the societal value elements of antibiotics and to provide guidance on how these value elements can be incorporated in economic evaluation. With a view to appropriately quantify the societal value of antibiotics, there is a need for good practice guidelines on the methodology of economic evaluation for such products. We argue that it is important to assess antibiotics at population level to account for their transmission, diversity, insurance, spectrum, novel action and enablement values. In addition to the value of antibiotics to infected patients, economic evaluations need to use modeling approaches to explore the impact of different modes of employing new and existing antibiotics (for example, as last resort treatment) on disease transmission and resistance development in current and future patients. Hence, assessing the value of antibiotics also involves an ethical dimension. Further work is required about how the multiple value elements of antibiotics are linked to each other and how they can be aggregated.
Background
Despite the benefits offered by biosimilars in terms of cost savings and improved patient access to biological therapies, and an established regulatory pathway in Europe, biosimilar ...adoption is challenged by a lack of knowledge and understanding among stakeholders such as healthcare professionals and patients about biosimilars, impacting their trust and willingness to use them. In addition, stakeholders are faced with questions about clinical implementation aspects such as switching.
Objective
This study aims to provide recommendations on how to improve biosimilar understanding and adoption among stakeholders based on insights of healthcare professionals (physicians, hospital pharmacists, nurses), patient(s) (representatives) and regulators across Europe.
Method
The study consists of a structured literature review gathering original research data on stakeholder knowledge about biosimilars, followed by semi-structured interviews across five stakeholder groups including physicians, hospital pharmacists, nurses, patient(s) (representatives) and regulators across Europe.
Results
Although improvement in knowledge was observed over time, generally low to moderate levels of awareness, knowledge and trust towards biosimilars among healthcare professionals and patients are identified in literature (
N
studies = 106). Based on the provided insights from interviews with European experts (
N
= 44), a number of challenges regarding biosimilar stakeholder understanding are identified, including a lack of practical information about biosimilars and their use, a lack of understanding about biosimilar concepts and a lack of knowledge about biologicals in general. Misinformation by originator industry is also believed to have impacted stakeholder trust. In terms of possible solutions and actions to improve stakeholder understanding, broad support exists to (1) organize initiatives focussed on explaining the rationale behind biosimilar concepts and the approval pathway, (2) invest in education about biologicals in general, (3) develop clear and one-voice regulatory guidance about biosimilar interchangeability and switching across Europe, (4) disseminate real-world clinical biosimilar (switch) data, (5) share biosimilar experiences by key opinion leaders and among peers, (6) provide practical biosimilar product information, (7) provide guidance about biosimilar use, (8) actively counterbalance misinformation and organize information initiatives by neutral entities, (9) organize multi-stakeholder informational and educational efforts, aligning information between involved stakeholder groups and (10) design initiatives in a way that ensures active information uptake. Furthermore, interviewees argue that governments should be proactive in these regards.
Conclusions
This study argues in favour of a structural, multi-stakeholder framework at both European and national level to improve stakeholder biosimilar understanding and acceptance. It proposes a number of actionable recommendations that can inform policy making and guide stakeholders, which can contribute to realizing healthcare system benefits offered by biosimilar competition.
...the diseases, not the drugs, are the orphans because all drugs are very expensive,3 having marrying this success story (table). ...in some cases, several OMPs are available for the same disease; ...for example, three drugs are licensed for treatment of Gaucher's disease (imiglucerase, velaglucerase alfa, and taliglucerase alfa).15 No evidence favours any one product over the other, and each drug costs about US$200 000 per patient per year. In a landmark departure from previous practice in 2014, the US Senate requested information on developmental costs and numerous other details for sofosbuvir, a drug for radical treatment of hepatitis C virus infection.19 Individual EU member state governments have since increasingly been requesting that industry disclose information about costs incurred during drug development that justify drug prices, but without legal obligations, these requests have largely been evaded. First synthesised in 1869, hydroxyurea has been used for decades in patients with myeloproliferative disorders and is now also indicated for sickle-cell disease.22,23 In the 2017 issue of the British National Formulary, one type of hydroxyurea for myeloproliferative disorders is listed at £0·24 per g, and another type of hydroxyurea for sickle-cell disease is listed at £16·7 per g. Common sense suggests that something must be wrong here.
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