Rare Diseases
The European Medicines Agency has recommended 16 drugs for EU-wide approval, including Vertex’s cystic fibrosis drug Alyftrek and five other orphans.
The European Medicines Agency is this week expected issue opinions on whether the European Commission should grant EU marketing authorization for 19 new products.
Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.
Industry feedback on a European Medicines Agency pilot that introduced a “stepwise” approach to pediatric investigation plans was positive, but the future of the new model will depend on the outcome of the pharmaceutical legislation negotiations, the agency says.
The hub’s goals of efficiency, centralization and coordination fit with the Trump Administration’s current focus for the FDA, Amy Comstock Rick, director of strategic coalitions, told the Biopharma Congress.
England’s health technology assessment institute, NICE, is looking to “reimagine” its evaluation process with the help of AI, rather than just using this technology to speed up its existing processes.
Not all companies will be able to access joint scientific consultations under the EU Health Technology Assessment Regulation, but success is still possible for those that engage with national agencies early on, says EUCOPE’s Alexander Natz.
Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.
Companies have until 17 March to respond to an EU consultation on a new strategy that will seek to simplify the regulatory framework and make it easier for innovative small and medium-sized enterprises to “access the capital they need” to scale up in the bloc.
Center for Biologics Evaluation and Research Director Peter Marks expects no fundamental changes in support for FDA initiatives and said the Rare Disease Innovation Hub, which promotes cross-center collaboration, is “consistent with what we're hearing in the current environment.”
Pharming has convinced NICE to reverse its rejection of its treatment for APDS by providing the health technology assessment institute with more data. It has also dropped the price it was asking for the drug, which has a list price of £352,000 per year per patient.
A new Innovative Health Initiative project will see the German health technology appraisal (HTA) body, IQWiG, work towards ensuring evidence generated in clinical studies meets the needs of HTA bodies and regulators.
Democrats are raising concerns that the continuing resolution intended to fund the government for the remainder of fiscal year 2025 gives Trump too much power, though it is unclear whether the FDA would be as impacted as other agencies.
Canada’s new guidance on rare disease registries is based on international guidelines, but in some areas there remains work to ensure that implementation of some recommendations is feasible in the Canadian context.
The EU, US and other countries with similar health care systems must “take responsibility” for the financial and health risks involved in getting innovative drugs, such as gene therapies, to market, says an academic expert who has worked in the advanced therapy field.
The European Medicines Agency has recommended in favor of approving four new products, and has also reaffirmed its positive opinion for the Alzheimer’s disease drug, Leqembi.
In the wake of cuts at the FDA, NIH and CMS, rare disease advocates are reminding Capitol Hill that some cuts can hinder progress, rather than eliminate waste.
The European Medicines Agency is looking for researchers to conduct scientific studies that address specific regulatory concerns, such as the development of innovative clinical trial designs or validation of novel manufacturing methods.
There has been “a lot of fear and anxiety” resulting from the Trump Administration’s early actions aimed at freezing grants and FDA communications, NORD’s Pamela Gavin says.
Pink Sheet editors discuss Center for Drug Evaluation and Research Director Patrizia Cavazzoni’s surprising retirement announcement, the importance of the large bolus of guidance documents that the FDA released 6 January, and the FDA’s decision to continue reviewing and granting rare pediatric disease designations even though the program lapsed.