Negotiating prices of drugs for rare diseases

Henrard, Séverine; Arickx, Francis

doi:10.2471/blt.15.163519

Cited by 27 publications

(26 citation statements)

References 10 publications

(14 reference statements)

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“…Over the last three years, Member States have also developed regional cooperations, e.g. BeNeLuxAIr, the Valletta Declaration, FINOSE and Visegrad projects, to undertake joint activities such as HTA,price negotiations especially for new medicines for orphan diseases, horizon scanning and information sharing [15][16][17][18].…”

Section: Historical Background On Initiatives For Htamentioning

confidence: 99%

“…Ideally Member States should agree to cooperate between them in a framework which is set and managed by them. In the meantime, Member States could build on current cross border collaborations and other arrangements with existing co-operation around key activities such as horizon scanning as well as pricing and reimbursement considerations [15][16][17][18]97,98].…”

Section: Opinions Of the Authors On The Model For Collaboration On Htmentioning

confidence: 99%

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Proposal for a regulation on health technology assessment in Europe – opinions of policy makers, payers and academics from the field of HTA

Bonanno

Bucsics²,

Simoens

et al. 2019

Expert Review of Pharmacoeconomics & Outcomes Research

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Section: Historical Background On Initiatives For Htamentioning

confidence: 99%

Section: Opinions Of the Authors On The Model For Collaboration On Htmentioning

confidence: 99%

Proposal for a regulation on health technology assessment in Europe – opinions of policy makers, payers and academics from the field of HTA

Bonanno

Bucsics²,

Simoens

et al. 2019

Expert Review of Pharmacoeconomics & Outcomes Research

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“…Generally, low patient volumes make it unattractive for pharmaceutical companies to invest in the development of orphan drugs. [ 148 – 150 ] Fabry disease, for example, has a prevalence of approximately 1 per 100,000 persons, thus making it unattractive for companies to develop drugs for these patients without further incentives. [ 149 ] That is why the orphan drug regulation was designed.…”

Section: Drug Innovation Regulation and Pricing Interventionsmentioning

confidence: 99%

“…European regulatory bodies offer exclusive licensing for ten years, lower regulatory fees and scientific advice. [ 148 ; 150 ] Orphan drug legislation has worked, yielding at least 73 drugs for orphan indications in the European Union since the law passed in 2000[ 151 ] and 335 in the US since the FDA set regulations in 1983 and 2002[ 152 ].…”

Section: Drug Innovation Regulation and Pricing Interventionsmentioning

confidence: 99%

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Addressing the challenge of high-priced prescription drugs in the era of precision medicine: A systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks

2017

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ContextRecent public outcry has highlighted the rising cost of prescription drugs worldwide, which in several disease areas outpaces other health care expenditures and results in a suboptimal global availability of essential medicines.MethodA systematic review of Pubmed, the Financial Times, the New York Times, the Wall Street Journal and the Guardian was performed to identify articles related to the pricing of medicines.FindingsChanges in drug life cycles have dramatically affected patent medicine markets, which have long been considered a self-evident and self-sustainable source of income for highly profitable drug companies. Market failure in combination with high merger and acquisition activity in the sector have allowed price increases for even off-patent drugs. With market interventions and the introduction of QALY measures in health care, governments have tried to influence drug prices, but often encounter unintended consequences. Patent reform legislation, reference pricing, outcome-based pricing and incentivizing physicians and pharmacists to prescribe low-cost drugs are among the most promising short-term policy options. Due to the lack of systematic research on the effectiveness of policy measures, an increasing number of ad hoc decisions have been made with counterproductive effects on the availability of essential drugs. Future challenges demand new policies, for which recommendations are offered.ConclusionA fertile ground for high-priced drugs has been created by changes in drug life-cycle dynamics, the unintended effects of patent legislation, government policy measures and orphan drug programs. There is an urgent need for regulatory reform to curtail prices and safeguard equitable access to innovative medicines.

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Sustainable Flow‐Synthesis of (Bulky) Nucleoside Drugs by a Novel and Highly Stable Nucleoside Phosphorylase Immobilized on Reusable Supports

Benítez‐Mateos

Paradisi

2021

ChemSusChem

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The continuous synthesis of valuable nucleoside drugs was achieved in up to 99 % conversion by using a novel halotolerant purine nucleoside phosphorylase from Halomonas elongata (HePNP). HePNP showed an unprecedented tolerance to DMSO, usually required for substrate solubility, and could be immobilized on agarose microbeads through disulfide bonds, via a genetically fused Cystag. This covalent yet reversible binding chemistry showcased the reusability of agarose microbeads in a second round of enzyme immobilization with high reproducibility, reducing waste and increasing the sustainability of the process. Finally, the flow synthesis of a Nelarabine analogue (6‐O‐methyl guanosine) was optimized to full conversion on a 10 mm scale within 2 min residence time, obtaining the highest space‐time yield (89 g L−1 h−1) reported to date. The cost‐efficiency of the system was further enhanced by a catch‐and‐release strategy that allowed to recover and recirculate the excess of sugar donor from the downstream water waste.

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Negotiating prices of drugs for rare diseases

Cited by 27 publications

References 10 publications

Proposal for a regulation on health technology assessment in Europe – opinions of policy makers, payers and academics from the field of HTA

Proposal for a regulation on health technology assessment in Europe – opinions of policy makers, payers and academics from the field of HTA

Addressing the challenge of high-priced prescription drugs in the era of precision medicine: A systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks

Sustainable Flow‐Synthesis of (Bulky) Nucleoside Drugs by a Novel and Highly Stable Nucleoside Phosphorylase Immobilized on Reusable Supports

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