Clinical evidence for orphan medicinal products-a cause for concern?

Picavet, Eline; Cassiman, David; Hollak, Carla E. M.; Maertens, Johan; Simoens, Steven

doi:10.1186/1750-1172-8-164

Cited by 31 publications

(39 citation statements)

References 28 publications

(44 reference statements)

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“…However, for payers, the risks associated with coverage decisions on DRDs are considerable. Clinical evidence is typically limited to small short-term trials relying on surrogate rather than hard clinical outcomes (Picavet et al 2013). The annual treatment costs, most of which are life-long, can exceed $300,000 CDN per patient (Hollis 2005).…”

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confidence: 99%

Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

Menon

Clark

Stafinski

2015

hcpol

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Introduction: Over the past 20 years, the number of therapies developed for rare diseases has rapidly increased. Often, these therapies represent the only active treatment for debilitating and/or life-threatening conditions. However, they create significant challenges for public and private payers. Because they target small patient populations, clinical evidence of efficacy/ effectiveness is typically limited, while the cost per patient is high. In Canada, each province/territory establishes its own mechanisms for determining which drugs for rare diseases (DRDs) to provide. Objectives: To compare current mechanisms across provinces and territories, and explore their impact on access. Methods: A systematic review of relevant published and unpublished documents was performed. Electronic bibliographic databases, the internet, and government websites were scanned using structured search strategies. Information was extracted independently by two researchers, and included aspects such as program type, condition/patient/therapy eligibility criteria, role of health technology assessment (HTA), decision options, ethical assumptions, and stakeholder input. It was validated through member-checking with provincial/territorial policy experts and tabulated to facilitate qualitative analyses. Impact on access was assessed through a cross-province/territory comparison of the coverage status of all non-cancer therapies reviewed by the Common Drug Review for indications affecting <1/2,000 Canadians using the Kappa statistic. Reasons for variations were explored using qualitative techniques. Results: Each province/territory has formal and informal mechanisms through which such therapies may be accessed. In most cases, formal mechanisms constitute the centralized HTA processes that also apply to common therapies. While several provinces have established dedicated processes/programs, whether they have affected access is not clear. Despite broadly comparable approaches, there is less than perfect agreement on publicly funded DRDs across jurisdictions. Conclusions: Individual jurisdictions have developed different approaches to providing access to these therapies. However, as the number increases, a more systematic approach to decisionmaking may be needed.

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confidence: 99%

Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

Menon

Clark

Stafinski

2015

hcpol

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“…Demonstrating therapeutic value for orphan drugs is often complicated, for instance due to the use of surrogate endpoints, the small study population and the heterogeneous presentation of rare diseases [23]. Furthermore, it is often unclear how improvement in a hard or surrogate endpoint translates into clinical benefit for patients thereby complicating reimbursement decisions.

“There are also a few [orphan drugs] for which the evidence is far less convincing, I’m thinking of some enzyme replacement therapies”.

…”

Section: Resultsmentioning

confidence: 99%

Reimbursement of orphan drugs in Belgium: what (else) matters?

Picavet

Cassiman

Simoens

2014

Orphanet J Rare Dis

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BackgroundMost 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.MethodsSix 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.ResultsDifferent 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.DiscussionThe 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.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-014-0139-z) contains supplementary material, which is available to authorized users.

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“…There was an increasing tendency to evaluate the quality of life of the patients in the clinical trials (62.4%), in contrast to other series studied [28]. This trend reflects the growing intention to provide evidence in this area, which is so important for rare diseases, where an increase in quality of life is often as important as achieving high treatment efficacy [29].…”

Section: Discussionmentioning

confidence: 99%