Written by Thea Collier- Lawlor, Analyst & Josh McLuckie, Analsyt The well-known challenges faced by developers of advanced therapies for rare diseases have been under
Vertex and CRISPR Therapeutics’ Casgevy became the first CRISPR gene therapy to be approved in the world after the UK’s MHRA granted conditional approval. Casgevy is indicated for the treatment of sickle cell disease and transfusion-dependent beta thalassemia, with the hope of being a potentially curative therapy for patients
Written by Marco Sancandi, PhD, Analyst. Email: [email protected] Rare diseases, individually infrequent but collectively prevalent, are in the spotlight as scientists strive to develop safer
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.
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).
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.
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
Written by: Srishti Gupta, PhD; Senior Consultant Contact: [email protected] In March 2023, the Food and Drug Administration (FDA) published, a first of its kind, draft
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
