Blogs
IQWiG recommends downgrading Gilead’s Yescarta to ‘no added benefit’ – a sign of things to come for orphan drugs in Germany?
Yescarta, Gilead’s CAR-T cell therapy recently underwent a full HTA assessment in Germany after breaching the annual sales threshold for orphan drugs. Upon reassessment, Germany’s lower HTA body IQWiG recommended downgrading Yescarta’s rating in one indication to ‘no added benefit’, which is likely to negatively impact pricing negotiations.
Swissmedic and FOPH are joining forces next year to provide a simultaneous regulatory and reimbursement review process.
From 2024, the Swiss regulatory body (Swissmedic) and health technology assessment (HTA) organization (Federal office of public health; FOPH) are introducing a simultaneous reimbursement and regulatory review process, to promote earlier access of medicines. This approach will enable manufacturers to submit both applications at the same time, making it possible to achieve marketing authorization and reimbursement in situ (1). This has been evidenced under a successful pilot with Roche’s Lunsumio (completed in March 2023).
Calls for reform in the assessment of orphan drugs in Germany, upon the implementation of EU Joint Evaluation.
OD (orphan drug) privilege status under the AMNOG procedure means that ODs are currently exempt from providing comparative data for their assessment. They are assumed to have a positive added benefit (with the lowest rating possible being a positive but unquantifiable added benefit), even when the data is insufficient to support this. If sales over 12 months surpass the €30 million mark, this privilege is revoked, and a full assessment is performed based on comparative data.
Advancing Rare Disease Care: The National Rare Disease Plan 2023-2026
Written by Andrea Bernardini, Associate Consultant & Marco Sancandi, Analyst Contacts: abernardini@partners4access; msancandi@partners4access In a significant step forward for healthcare in Italy, the National Rare
Is the Chinese market for pharmaceuticals going to overtake a stagnant Europe?
Written by: James Lee, Analyst Contact: jlee@partners4access.com China, with 1.4 billion people, holds promise as a market for pharmaceutical companies due to its major overhaul of
Hospital Exemption; still a big risk for pharma
Written by: Angeliki Meletsi; Analyst Contact: ameletsi@partners4access.com The European Commission proposals on the EU pharmaceutical legislation revision, announced in April 2023, have not included any
Accelerated approval and accelerated withdrawal – will the new clinical trial guidance bring US closer to Europe?
Written by: Srishti Gupta, PhD; Senior Consultant Contact: sgupta@partners4access.com In March 2023, the Food and Drug Administration (FDA) published, a first of its kind, draft
Orphan Drug market, how is Italy doing? The latest OSSFOR Booklet paints a favourable picture for the Italian landscape.
By Marco Sancandi, PhD; Analyst On the 31st of January 2023, the 6th joint report from the Orphan Drugs Observatory and the Italian Society of
New UK Regulation to Spark Major Changes in the CAR-T Landscape
By Fisentzos Stylianou, PhD; Senior Analyst The UK will be the first country to introduce a regulatory framework for innovative medicines manufactured at the point
