Reimbursement Of Orphan Drugs Research Articles

RESOURCE ALLOCATION FOR THE TREATMENT OF RARE DISEASES

IntroductionDrugs for rare diseases are often associated with extremely high costs which can be a barrier to patients' accessibility. Due to scarce healthcare resources, funding for expensive orphan therapies is...

Addressing the authorization of orphan drugs will not fix reimbursement problems

The October 3, 2012, orphan drug initiative announced by the Federal Ministry of Health aimed at improving availability of information about, and authorization of, orphan drugs may have the potential for accelerating Health Canada’s approval of treatments for many rare diseases. However, details about policy formulation or implementation are vague at best, and it is doubtful that it will address the more serious issue of patient affordability. Which begs the question, what is the ultimate result of accelerating authorization if the significant barrier of affordability still exists? Unfortunately, without provincial collaboration on approving reimbursement for orphan drugs, patients will still be left without long-term access.

PHP127 Reimbursement of Orphan Drugs: What is the Difference?

Orphan drugs are subject to regulatory and reimbursement regulations that differ with respect to application process and necessary documentation. An orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval. Reimbursement hurdles are also supposedly lower for orphan drugs in Europe than usually. Definition and assessment process of orphan drugs for reimbursement were reviewed and analyzed. Differences to other drugs are outlined and reimbursement decisions presented. The German law on health care reform (AMNOG) implemented in 2011 requires that with market access newly approved products demonstrate their innovation through a reimbursement dossier to avoid reference group pricing. For orphan drugs, manufacturers must also submit a dossier but the additional medical benefit is regarded as having been proofed by the market authorization itself. Thus proof of additional benefit does not need to be presented but information on relevant patient groups and on the extent of this additional benefit. However, if annual sales of an orphan drug within the statutory health insurance exceed 50 million EUR, a full assessment is made. For pirfenidone, the first orphan drug assessed under the new law, IQWiG (Institute for Quality and Efficiency in Health Care) declined an additional therapeutic benefit but the G-BA (Joint Federal Committee) did not follow this conclusion in accordance to the law. In Italy pirfenidone was grouped into the lowest reimbursement class. Unlike Germany, Italy has special funds set aside for orphan drugs, France has an early access program, and many countries are struggling with how to create a reimbursement process that reflects the different regulatory provisions for orphans. Although orphan drugs are often regarded as unquestioned reimburseable, differences in respective processes and assessments exists. Manufacturers are requested to build Market Access arguments carefully and expect challenges in orphan drug indications as well.

Orphan Drugs for Rare Diseases

Orphan drugs are intended for diseases with a very low prevalence, and many countries have implemented legislation to support market access of orphan drugs. We argue that it is time to revisit the special market access status of orphan drugs. Indeed, evidence suggests that there is no societal preference for treating rare diseases. Although society appears to assign a greater value to severity of disease, this criterion is equally relevant to many common diseases. Furthermore, the criterion of equity in access to treatment, which underpins orphan drug legislation, puts more value on health improvement in rare diseases than in common diseases and implies that population health is not maximized. Finally, incentives for the development, pricing and reimbursement of orphan drugs have created market failures, including monopolistic prices and the artificial creation of rare diseases. We argue that, instead of awarding special market access status to orphan drugs, there is scope to optimize research and development (R&D) of orphan drugs and to control prices of orphan drugs by means of, for example, patent auctions, advance purchase commitments, pay-as-you-go schemes and dose-modification studies. Governments should consider carefully the right incentive strategy for R&D of orphan drugs in rare diseases.

Pricing and reimbursement of orphan drugs: the need for more transparency.

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.

Zulassung und Erstattung von Orphan Drugs im internationalen Vergleich

This paper analyses schemes to promote the authorisation of and reimbursement for orphan drugs. 8 countries - Australia, Canada, Germany, Great Britain, France, Netherlands, Switzerland, USA - were studied to compare specific regulations for orphan drugs regarding drug admission, health technology assessment (HTA), decision-making for reimbursement, and off-label and compassionate use. Information was obtained by reviewing published and grey literature. Expert interviews were also conducted. The comparison of orphan drug legislation reveals that the EU and the USA offer the greatest incentives for the development of orphan drugs, whereas there is a tendency for Australia and Switzerland to profit from incentives in other countries. Although not explicitly stated, economic evaluation of orphan drugs takes the special circumstances for orphan drugs into account. In addition to common reimbursement practices, special schemes or programmes for the reimbursement of high-priced orphan drugs exist in all countries that were analysed. Therefore access to orphan drugs seems to be warranted. However, due to co-payments of 5%, the USA may form an exception. On the one hand, the use of special criteria for drug admission, HTA, and reimbursement promotes R&D for orphan drugs. On the other hand, high opportunity costs arise, because huge efforts are made for a minority of patients. A solution for this moral dilemma may be the application of "rule of rescue" or of "no cure, no pay" programmes.

A comparative study of European rare disease and orphan drug markets

This article aims to compare regulatory aspects of rare disease and orphan drug markets in Belgium, France, Italy, the Netherlands, Sweden and the United Kingdom. Information was derived from the international literature, analysis of legal texts, and a survey completed by national experts. These countries adopted varying approaches towards regulating rare disease and orphan drug markets and, hence, the availability, pricing and reimbursement of orphan drugs vary between countries. Strategies to keep down prices include public procurement in Sweden, profit controls in the United Kingdom, and price comparisons with other countries. To gain reimbursement, the cost-effectiveness and/or budget impact of orphan drugs is considered in some countries. Other societal considerations, such as whether the drug treats a life-threatening disease, are sometimes taken into account. Extensive government intervention exists in rare disease and orphan drug markets in the countries studied. Our recommendations are to define priorities for research on rare diseases and orphan drugs at the European level, to set up disease and patient registries with a view to investigating the long-term effectiveness and cost-effectiveness of orphan drugs, to assess the profitability of orphan drugs, and to take into account societal considerations when evaluating orphan drugs.

PHP55 RECOMMENDATIONS FOR ORPHAN DRUGS IN TWO EU MEMBER STATES—A FOCUS ON THE UNDERLYING PHARMACOECONOMIC EVALUATIONS

OBJECTIVES: In the Netherlands, orphan drug developers can be exempted from providing a full phwhereas in Scotland no such exceptions can be made. METHODS: All orphan drug reimbursement reports from the Dutch reimbursement institution (CFH) and guidance issued by the Scottish Medicines ConsoRESULTS: 38 orphan drugs were submitted to the CFH; 37 to the SMC. Only one CFH submission included a full pharmacoeconomic evaluation, while almost all SMC submissions did. The CFH gave positive recommendations for reimbursemen(95%). In contrast, of the 37 SMC submissions, 19 (51%) received a positive recom-mendation for use. Half of the SMC submissions reported an unfavourable cost-effectiveness outcome; almost all of these received a negative recommendation. CONCLUSIONS: Differences in policies and requirements for submissions for orphan drugs can explain discrepancies in reimbursement and guidance for use decisions between Scotland and the Netherlands.