Clinical Trials
A US FDA Advisory Committee responded enthusiastically to the potential use of challenge models to enable pivotal trials of novel pertussis vaccines, but most members suggested more refinements are needed before they are ready to use.
Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.
Slow adoption of alternatives to animal testing in the current decentralized regulatory framework shows the need for a ‘one-stop shop’ at FDA that can provide advice, precedents and qualification programs.
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
It is time for a much greater focus on pull incentives to ensure viable market conditions for medicines for neglected diseases that are traditionally regarded as unprofitable.
The US FDA is finalizing a guidance intended to encourage the use of decentralized clinical trial techniques. After the COVID-19 pandemic necessitated adoption of the more patient-friendly approaches, the agency is clearly eager to see them become routine.
Having its own priority review vouchers could serve as an important pull incentive for neglected disease R&D in Europe, although the unpredictable nature of the vouchers may deter some investors.
Contract research organizations in India must gear up to comply with new registration requirements coming into effect in April 2025 that aim to enhance the quality and integrity of clinical trials, as well as of any bioavailability and bioequivalence studies conducted by them.
Draft guidance recommendations on setting enrollment goals lead PhRMA and BIO to seek assurances of FDA flexibility absent reliable disease prevalence data.
Industry organizations argued feedback should be expected and object to draft guidance language suggesting it may be optional. The agency also should apply waiver criteria flexibly and address potential consequences if enrollment goals are not met, stakeholders say.
New guidance from the European Medicines Agency explains how in vitro and in vivo models may be used instead of clinical data for the purpose of establishing therapeutic equivalence in a stepwise approach.
Companies dealing with commercial costings or contracts with the National Health Service for early phase clinical trials or trials of advanced therapy medicinal products should soon be able to conclude their negotiations more quickly.
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.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
Japan plans to offer wider support to foreign firms and ventures with innovative candidates to start clinical trials in the country, as part of key measures from current prime minister Fumio Kishida.
Patient centricity, diversity, innovation and transparency are key topics in the World Health Organization’s new guidance that recommends best practices for clinical trials and complements existing international guidance in this area.
New guidance from the European Commission on clinical trial validity for forthcoming joint clinical assessments has eased some concerns about the generation of evidence for rare disease treatments and advanced therapies.
Clinical trial sponsors could face an “extremely burdensome” experience if patients were allowed to opt out of their data being shared anonymously under the European Health Data Space, warns industry federation EFPIA.
The EU’s public-private Trials@Home Consortium examined what it would take to develop the fully integrated, unified digital platforms needed to enhance the implementation of decentralized clinical trials.
Draft recommendation for investigators to maintain a task log of activities that local health care providers perform was removed from final guidance on decentralized clinical trials. Other changes include more detail on US FDA oversight and what HCPs can and cannot do in such studies.