Gene Therapies: The Challenge of sUper-High-Cost Treatments and How to Pay for them

Carr, David; Bradshaw, S.

doi:10.2217/rme-2016-0010

Cited by 47 publications

(49 citation statements)

References 29 publications

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“…This increase in approvals, combined with high per patient prices, has led to worries amongst policy makers about the affordability of aggregate OMP expenditure [3, 5, 6]. More recently, the introduction of high-priced gene and cell therapies – many of which are also OMPs – has further focused attention on OMP expenditure [9–11].…”

Section: Introductionmentioning

confidence: 99%

An analysis of orphan medicine expenditure in Europe: is it sustainable?

Mestre-Ferrandiz¹,

Palaska

Kelly

et al. 2019

Orphanet J Rare Dis

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BackgroundOrphan medicinal product (OMP) prices are considered by some to be a challenge to the sustainability of healthcare expenditure. These concerns are compounded by the increasing number of OMPs receiving marketing authorisation (MA) annually. The aim of this study was to explore the sustainability of OMP expenditure within the context of total European pharmaceutical expenditure.MethodsUsing historical IQVIA data, an analysis was conducted on total pharmaceutical and OMP expenditure in eight countries (using values / volumes) in the branded, non-branded and overall pharmaceutical market. Country level and aggregated data was considered for EU5 countries, Austria, Belgium and Ireland.Three key analyses were conducted: The OMP share of total pharmaceutical expenditure was calculated from 2000 to 2017, to assess its evolution over time.The results of this analysis were compared with a 2011 forecast of OMP budget impact.The evolution of the total pharmaceutical market and its different segments (branded OMPs, non-OMP branded and unbranded) were assessed by estimating the compound annual growth rate (CAGR) and percentage of pharmaceutical expenditure for each market segment from 2010 to 2017.ResultsAcross countries, OMP share of total pharmaceutical expenditure has increased each year since 2000, rising to 7.2% of total pharmaceutical expenditure in 2017. OMP expenditure has increased at a CAGR of 16% since 2010. The number of OMPs receiving MA each year showed a CAGR of 11% since 2001, four percentage points greater than the CAGR for all medicines receiving MA over the same period. OMP share of total pharmaceutical expenditure is higher than forecasted in 2011 due to slower than expected growth in the non-OMP market. OMP growth has been offset by reduced expenditure in the general market and increased use of generics and biosimilars.ConclusionsRelative spending on OMPs has increased over the last 20 years, but this has been largely compensated for within the current allocation of total pharmaceutical spending by flat expenditure for non-OMPs and increased volumes of (lower-priced) generics/biosimilars, reflecting a shift towards expenditure in higher cost, lower volume patient populations and a shift in drug development towards more specialised targeting of diseases.

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Section: Introductionmentioning

confidence: 99%

An analysis of orphan medicine expenditure in Europe: is it sustainable?

Mestre-Ferrandiz¹,

Palaska

Kelly

et al. 2019

Orphanet J Rare Dis

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“…However, we maintain that some kinds of RMPs raise specific challenges, such as gene therapies when they are curative [37]. We have shown that risk-sharing specifically is far less discussed than reimbursement generally.…”

Section: ) Discussionmentioning

confidence: 85%

The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

Mahalatchimy

Faulkner

2017

Regen. Med.

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Aim: This paper aims to map the trends and analyze key institutional dynamics that constitute the policies for reimbursement of regenerative medicine (RM), especially in the UK. Materials & methods: Two quantitative publications studies using Google Scholar and a qualitative study based on a larger study of 43 semi-structured interviews. Results: Reimbursement has been a growing topic of publications specific to RM and independent from orphan drugs. Risk-sharing schemes receive attention among others for dealing with RM reimbursement. Trade organizations have been especially involved on RM reimbursement issues and have proposed solutions. Conclusion: The policy and institutional landscape of reimbursement studies in RM is a highly variegated and conflictual one and in its infancy.

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“…A major drawback of pay-for-outcome schemes is the added burden to both the patient and the healthcare system to monitor the performance of the therapy. These costs can become so high that they outweigh any savings made by such a scheme [3,5,28,49,54,59,64]. The enforcement of additional studies to resolve the uncertainties regarding long-term effects could sometimes also be achieved by implementing a form of coverage with evidence development where it is agreed that the price will drop iteratively by a certain percentage or amount after predefined periods when the manufacturer does not produce the necessary evidence [89].…”

Section: Methodsmentioning

confidence: 99%

Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis

Vreman

Broekhoff

Leufkens

et al. 2020

IJERPH

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The reimbursement of expensive, innovative therapies poses a challenge to healthcare systems. This study investigated the feasibility of managed entry agreements (MEAs) for innovative therapies in different settings and combinations. First, a systematic literature review included studies describing used or conceptual agreements between payers and manufacturers (i.e., MEAs). Identical and similar MEAs were clustered and data were extracted on their benefits and limitations. A feasibility assessment was performed for each individual MEA based on how it could be applied (financial/outcome-based), on what level (individual patients/target population), in which payment setting (centralized pricing and reimbursement authority yes/no), for what type of therapies (one-time/chronic), within what payment structures, and whether combinations with other MEAs were feasible. The literature search ultimately included 82 papers describing 117 MEAs. After clustering, 15 unique MEAs remained, each describing one or multiple similar agreements. Four of those entailed payment structures, while eleven entailed agreements between payers and manufacturers regarding price, usage, and/or evidence generation. The feasibility assessment indicated that most agreements could be applied throughout the different settings that were assessed and could be applied in different payment structures and in combination with multiple other agreements. The potential to combine multiple agreements leads to a multitude of different reimbursement mechanisms that may manage the price, usage, payment structure, and additional conditions for an innovative therapy. This overview of the feasibility of combinations of MEAs can help decision-makers construct a reimbursement mechanism most suited to their preferences, the type of therapy under evaluation, and the applicable healthcare system.

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Gene Therapies: The Challenge of sUper-High-Cost Treatments and How to Pay for them

Cited by 47 publications

References 29 publications

An analysis of orphan medicine expenditure in Europe: is it sustainable?

An analysis of orphan medicine expenditure in Europe: is it sustainable?

The Emerging Landscape of Reimbursement of Regenerative Medicine Products in the UK: Publications, Policies and Politics

Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis

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