EMA Officials: Regulatory, HTA Advice Sped Access to Bluebird’s Gene Therapy

Regulatory NewsRegulatory News | 12 November 2019 |  By 

Officials from the European Medicines Agency (EMA) say that advice from the agency and health technology assessment (HTA) bodies played a “key role” in speeding access to Bluebird Bio’s gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene).
“Groundbreaking therapies such as Zynteglo present specific challenges for those who assess their benefits and risks for the initial authorisation and those who assess their value as a basis for pricing and reimbursement decisions,” EMA writes, noting that continuous dialogue with regulators and HTA bodies can help to avert those challenges.
In a commentary in Clinical Pharmacology & Therapeutics, three EMA officials, including Committee for Advanced Therapies (CAT) Chair Martina Schussler-Lenz, Committee for Medical Products for Human Use (CHMP) Chair Harald Enzmann and EMA’s Head of Scientific Advice Spiros Vamvakas, detail how those interactions helped address clinical and manufacturing issues early on and contributed to a more robust application.
In June, the European Commission conditionally authorized Zynteglo to treat patients 12 years and older with beta thalassemia, a rare inherited blood disorder, who require blood transfusions and do not having a matching donor for stem cell transplants.
The gene therapy, priced at €1.575 million over five years, was part of EMA’s adaptive pathways pilot and received an accelerated assessment under EMA’s PRIME (PRIority MEdicines) scheme that was completed in just 150 days.
According to the officials, these two programs gave Bluebird more frequent opportunities to interact with regulators during the review process.
“In the case of Zynteglo, changes in the manufacturing site and processes like improvement of transduction efficiency in the commercial scale process were discussed with regard to their impact on the interpretation of clinical data,” the officials write.
Additionally, the officials say that clinical issues were discussed with both patients and HTA bodies, including the suitability of the clinical data set, the primary endpoint used to support approval and the design of confirmatory trials and postmarket real-world data collection.
The officials attribute these discussions and EMA’s accelerated assessment with reducing the agency’s review and opinion for Zynteglo “by several months.”
However, the officials also note that the additional interaction and speedy assessment was resource-intensive and “should be reserved for products that are highly likely to offer true advancements for patients,” such as first-in-class medicines for rare diseases or that treat a condition with high unmet medical need.
EMA, Perspective


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