A prominent rare disease researcher has praised the UK’s Department for Health and Social Care for its transparency and willingness to work with experts, but said that regulatory systems needed to speed up to facilitate patient access to innovative treatments.
The former CDER Director spoke to the Pink Sheet about why a new statutory standard is needed for certain rare disease drug approvals. We lay out some of her thinking in part one of a multi-part series on the topic.
With approval applications for new active substances expected to rise by 25% by 2026, the UK regulator says it wants to focus its national assessment procedure on new innovative therapies. It will also review progress with the new International Recognition Procedure.
The UK’s drug regulator, the MHRA, is exploring new opportunities to work closer with its counterparts from Australia, Canada, Singapore and Switzerland under the ACCESS Consortium group, with a focus on pre-submission scientific advice.
Regulators do not have the resources to “double check” that companies are using AI appropriately, meaning that manufacturers must ensure the AI tools they use meet relevant standards, says an EU regulatory expert.
As efforts to improve diversity in clinical trials gain momentum globally, the Pink Sheet asked regulators in the UK, the EU, Canada, Australia and Japan about their efforts to support representative enrolment.
The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.
In this third article of a series on new drug reimbursement recommendations by the HTA body NICE, the Pink Sheet finds that fewer innovative medicines are reimbursed in England than in eight other European nations.
Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.
Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.
The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.
The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.
Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.
CRO executive Horst Ruppach discusses the critical factors that drug manufacturers must consider to ensure compliance with the revised ICH Q5A(R2) guideline on biologics viral safety. Thorough documentation and proactive engagement with regulatory bodies are key to navigating the complexities of viral safety evaluation in biotechnology products, he says.
The rate of non-submissions for combination therapies in England has been on a downward trend in the last two years.
As Sandoz publishes a report on US drug shortages, including a call for policy and other improvements, the firm’s North America head and AAM Chair Keren Haruvi discussed potential solutions to ongoing supply-chain failures.
The ISRCTN clinical trials registry has launched an improved dashboard to provide metrics that reveal how many studies are complying with key transparency requirements. Badges are in place for individual studies meeting the transparency criteria.
