Delayed access to treatments for rare diseases: Who's to blame?

Feltmate, Karen; Janiszewski, Peter M.; Gingerich, Sheena; Cloutier, Michael

doi:10.1111/resp.12498

Cited by 21 publications

(15 citation statements)

References 12 publications

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“…Consistent with previous reports [25, 68, 69], our analysis revealed substantial differences in rare disease infrastructure across countries; however, it was limited in the scope of the countries considered and was not designed to assess the effect of specific policies and structures on patient outcomes. Subsequent analyses should be conducted to correlate policy with the presence of actual programs and, ultimately, their effects on patient care.…”

Section: Resultssupporting

confidence: 71%

Review of 11 national policies for rare diseases in the context of key patient needs

Dharssi

Wong‐Rieger²,

Harold

et al. 2017

Orphanet J Rare Dis

147

149

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Rare diseases collectively exert a global public health burden in the severity of their manifestations and the total number of people they afflict. For many patients, considerable barriers exist in terms of access to appropriate care, delayed diagnosis and limited or non-existing treatment options. Motivated by these challenges, the rare disease patient community has played a critical role, elevating the patient voice and mobilizing legislation to support the development of programs that address the needs of patients with rare diseases.The US Orphan Drug Act of 1983 served as a key milestone in this journey, providing a roadmap for other countries to introduce and implement similar orphan drug legislation; more recently, the European Union (EU) has gone further to encourage the widespread adoption and implementation of rare disease plans or strategies designed to more adequately address the comprehensive needs of patients with rare diseases. Despite these legislative efforts and the growing contributions of patient advocacy groups in moving forward implementation and adoption of rare disease programs, gaps still exist across the policy landscape for several countries. To gain deeper insights into the challenges and opportunities to address key needs of rare disease patients, it is critical to define the current status of rare disease legislation and policy across a geographically and economically diverse selection of countries. We analyzed the rare disease policy landscape across 11 countries: Germany, France, the United Kingdom, Canada, Bulgaria, Turkey, Argentina, Mexico, Brazil, China, and Taiwan. The status and implementation of policy was evaluated for each country in the context of key patient needs across 5 dimensions: improving coordination of care, diagnostic resources, access to treatments, patient awareness and support, and promoting innovative research. Our findings highlight the continuing role of the patient community in driving the establishment and adoption of legislation and programs to improve rare disease care. Further, we found that while national rare disease plans provide important guidance for improving care, implementation of plans is uneven across countries. More research is needed to demonstrate the effect of specific elements of rare disease plans on patient outcomes.

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Section: Resultssupporting

confidence: 71%

Review of 11 national policies for rare diseases in the context of key patient needs

Dharssi

Wong‐Rieger²,

Harold

et al. 2017

Orphanet J Rare Dis

147

149

View full text Add to dashboard Cite

show abstract

“…Availability and access to medicines are important to reduce morbidity and mortality of rare diseases. For instance, until the recent availability of pirfenidone, a lung transplant was the only treatment option for patients with idiopathic pulmonary fibrosis, a rare disease with a 50% chance of survival at 3 years [ 6 ]. Despite the need and importance of availability and access to orphan drugs, there is a paucity of available treatments for rare diseases.…”

Section: Introductionmentioning

confidence: 99%

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

2015

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ObjectiveTo review existing regulations and policies utilised by countries to enable patient access to orphan drugs.MethodsA review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country.ResultsFifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country’s legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing. The majority of countries (27/35) had in place orphan drug legislation. Access to orphan drugs depends on individual country’s pricing and reimbursement policies, which varied widely between countries. High prices and insufficient evidence often limit orphan drugs from meeting the traditional health technology assessment criteria, especially cost-effectiveness, which may influence access.ConclusionsOverall many countries have implemented a combination of legislations, regulations and policies for orphan drugs in the last two decades. While these may enable the availability and access to orphan drugs, there are critical differences between countries in terms of range and types of legislations, regulations and policies implemented. Importantly, China and India, two of the largest countries by population size, both lack national legislation for orphan medicines and rare diseases, which could have substantial negative impacts on their patient populations with rare diseases.

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“…The CDR’s approach to evaluating the value of drugs for rare disorders (DRDs) has long been criticized [1–5] as being heavily weighted towards negative reimbursement recommendations that have led to inequitable access to DRDs across Canada. Proposals for a separate, more broad-based process for these drugs have been rejected by CADTH [6].…”

Section: Introductionmentioning

confidence: 99%

Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost

Rawson

2017

Orphanet J Rare Dis

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BackgroundAuthors from the Canadian Agency for Drugs and Technologies in Health (CADTH) presented an analysis of submissions to the Common Drug Review (CDR) between 2004 and February 3, 2016 for drugs for rare disorders (disorders with a prevalence of <50 per 100,000).ObjectiveThe aim of this analysis was to examine the same CDR submissions to evaluate whether the negative reimbursement recommendation rate, clinical evidence of efficacy and statements concerning the drug’s cost in the CDR reports varied with the prevalence of the disorder treated by the drug grouped into three decreasing categories: <50 to >10, ≤10 to >1, and ≤1 per 100,000.ResultsAs the prevalence of the treated disorder decreased, the median daily cost of the drug, the negative recommendation rate and the proportion of submissions with statements in the CDR reports highlighting the cost of the drug increased, while the proportion of submissions with acceptable evidence of clinical efficacy decreased. Moreover, although the CADTH authors reported that only two submissions received a negative recommendation due to a “lack of cost-effectiveness/high cost,” high cost was mentioned in the CDR reports of 15 drugs with negative recommendations, all for disorders with a prevalence of ≤10 per 100,000.ConclusionsThe aggregated analysis of CDR submissions for drugs for disorders with wide ranging prevalence rates concealed information of concern to patients. The negative reimbursement recommendation rate and the significance of cost in the CDR assessments increased as the prevalence of the treated disorder decreased. Since 2012, the manner in which high cost drugs for rare disorders have been dealt with by the CDR has changed. Cost has ceased to be a factor in negative recommendations but is included in criteria accompanying positive recommendations. This trend is associated with the integration of the CDR process with the system for price negotiation between public drug plans and pharmaceutical companies.

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Delayed access to treatments for rare diseases: Who's to blame?

Cited by 21 publications

References 12 publications

Review of 11 national policies for rare diseases in the context of key patient needs

Review of 11 national policies for rare diseases in the context of key patient needs

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost

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