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

Picavet, Eline; Cassiman, David; Simoens, Steven

doi:10.1186/s13023-014-0139-z

Cited by 21 publications

(25 citation statements)

References 22 publications

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“…Financial incentives utilised worldwide include: research grants, tax credits/corporate tax reductions, marketing exclusivity, and user fee waivers [ 1 , 12 , 17 , 31 , 41 , 42 , 49 ]. These provisions exist as a means to allow firms to recover research and development costs, which would not be possible with sales of orphan drugs given the small market sizes.…”

Section: Resultsmentioning

confidence: 99%

“…While orphan drugs often do not meet traditional cost-effectiveness criteria, they may be reimbursed by payers in some countries because other factors are taken into account in reimbursement decisions [ 17 ]. These include: therapeutic value, budget impact, impact on clinical practice, pricing and reimbursement practices globally, patient organisations, economic importance, ethical arguments and the political climate [ 17 ]. Standards of evidence required in reimbursement decisions across countries may explain these differences.…”

Section: Resultsmentioning

confidence: 99%

“…Managed entry agreements are an innovative and increasingly used approach to enable access to high cost orphan and non-orphan drugs in situations where there is a lack of sufficient evidence for coverage of promising technologies that may benefit patients [ 13 , 17 , 26 , 48 , 58 – 60 , 63 ].…”

Section: Discussionmentioning

confidence: 99%

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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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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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

2015

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“…Due to the increasing number of treatments for rare diseases that have been approved and are under development, and hence the rising costs for countries’ health systems, there is an ongoing debate about health policies for rare diseases and particularly about reimbursement of rare disease drugs. 4 , 10 , 11 Currently, when marketing authorization is granted for a medicine by EMA, the pricing and reimbursement is negotiated individually with pharmaceutical companies in each European country, where national health policies vary. The pricing of a drug can be set by the manufacturers (e.g.…”

mentioning

confidence: 99%

“…Decision-making about the availability, pricing and patient reimbursement of orphan drugs should be done according to clearly defined criteria. 11 , 14…”

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confidence: 99%

Negotiating prices of drugs for rare diseases

Henrard

Arickx

2016

Bull. World Health Organ.

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The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview

Contesse¹,

Valentine

Wall

et al. 2019

Adv Ther

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IntroductionThe traditional model of evaluating treatments based primarily on primary outcome measures has stumbled in its application to rare disease. Rare disease clinical trials face the methodological challenges of small, heterogeneous patient populations and relatively few validated, disease-specific outcome measures. Incorporating qualitative research into rare disease clinical trials may help sponsors, regulators, payers, and prescribers to better understand the real-world and patient-specific impact of a potential therapy. This paper provides a methodologic overview of the use of Patient and Caregiver Perception of Change (PPC and CPC) Assessments utilizing patient and caregiver video interviews to complement the data captured by traditional endpoints in rare disease clinical trials.MethodsIncorporating qualitative patient and caregiver video interviews into clinical trials allows for the rigorous capture of patient experiences and caregiver observations. Interview guides informed by input from key stakeholders provide the opportunity to solicit structured feedback on experiences before, during, and after the clinical trial. Patients and caregivers can complete their video interviews in a study mobile application, and interview transcripts are analyzed by independent coders. Themes are summarized by the treatment group and individual patient, which adds context to the clinical outcome measures of how patients feel and function, as well as elucidates the degree of change that is meaningful to patients and caregivers. The qualitative results can be compared to the data captured in clinical trials to assess data concordance.ConclusionCapturing patient experience data with sufficient rigor allows it to contribute to the body of evidence utilized in regulatory, payer, and prescriber decision-making. Adding PPC and CPC Assessments to rare disease clinical trials offers an innovative and powerful way to tap into the unique insights of patients and their families to develop a fuller picture of the patient experience in the clinical trial.FundingStealth BioTherapeutics Inc.

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

Cited by 21 publications

References 22 publications

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

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

Negotiating prices of drugs for rare diseases

The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview

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