Search Results for: FDA approva un nuovo trattamento per l’emicrania

The 1976 Medical Device Amendments granted the US Food and Drug Administration (FDA) regulatory authority over medical devices. The FDA’s regulatory review requirements are intended to correspond to the amount of information needed to provide “reasonable assurance of device safety and effectiveness” and establish a 3-tiered system for classifying devices based on their risk profile (low, moderate, and high) with a corresponding degree of evidence required for review. Low-risk devices are typically only subject to general controls (eg, good manufacturing practices) and exempt from premarket review. Nearly 90% of medical devices that undergo regulatory review are classified as moderate risk and receive authorization through the 510(k) process, which additionally requires manufacturers to demonstrate “substantial equivalence” to a previously authorized (predicate) device, usually without being required to show evidence of device safety and effectiveness from clinical studies. Devices in the third category, high risk, are fewer in number, frequently implanted and/or life-sustaining, and undergo the most rigorous regulatory review through the premarket approval pathway, which requires evidence from clinical studies demonstrating safety and effectiveness.

Introduction
The megalencephaly capillary malformation polymicrogyria (MCAP syndrome) results from mosaic gain-of-function PIK3CA variants. The main clinical features are macrocephaly, somatic overgrowth, neurodevelopmental delay and brain anomalies. Alpelisib (Vijoice) is a recently FDA-approved PI3Kα-specific inhibitor for patients with PIK3CA-related overgrowth spectrum (PROS). During its development, in patients with the MCAP subgroup of PROS, there was no specific, standardised evaluation of the effect on neuro-cognitive functioning. Moreover, it remains unknown if the molecule crosses the blood-brain barrier. Our objective is to evaluate the efficacy of a 24 month treatment with alpelisib on adaptive behaviour in patients with MCAP syndrome.

Methods and analysis
SESAM is an industry-sponsored two-period multicentre French academic phase II trial, with a 6-month double-blind, placebo-controlled period followed by an open-label period. The primary endpoint is a ≥4-point improvement in the Vineland II Adaptive Behaviour Scale (VABS), 24 months after treatment initiation. Secondary objectives are safety, VABS improvement at 6 months, impact on the quality of life, epilepsy and hypotonia. 20 patients aged 2 to 40 years with an MCAP diagnosis and neurodevelopmental disorders of various degrees, will be followed monthly in local centres, centrally assessed (clinical, biological, neuropsychological and functional evaluation) at baseline and every 6 months. Patients will be evaluated by volumetric MRI at baseline and at 24 months. An optional lumbar puncture will be performed to investigate blood-brain barrier crossing. Inclusions were completed by April 2024, with the end of follow-up in November 2026.
Given the efficacy of alpelisib in patients with PROS, if the drug crosses the blood-brain barrier, we can expect a clinical benefit for patients with neurocognitive disorders.

Ethics and dissemination
Ethical approval was given by CPP Sud-Ouest et Outre-Mer I (reference: 2022-500197-34-01). Findings from this study will be disseminated via publication, reports and conference presentations.

Trial registration number
NCT05577754

Objective
The COVID-19 pandemic required the rapid and often widespread implementation of medical practices without robust data. Many of these practices have since been tested in large, randomised trials and were found to be in error. We sought to identify incorrect recommendations, or reversals, among National Institute of Health COVID-19 guidelines and Food and Drug Administration (FDA) approvals and authorisations.

Design
Retrospective cross-sectional study.

Participants
Recommended medical practices and FDA authorisations or approvals for COVID-19 prevention, treatment and/or management.

Main outcome measures
The frequency and characteristics of COVID-19 medical reversals, defined as practices that were implemented and/or recommended during the pandemic, but were later tested in randomised trials that failed to find benefit.

Results
We found 332 COVID-19 recommendations. 85 (25.6%) opposed a medical practice, 23 (6.9%) were to continue a pre-COVID standard of care without deviation and 224 (67.5%) reccommended a new medical practice. We found randomised trials assessing 72 of these practices (32.1%), among which 25 (35%) were found to be in error and deemed medical reversals. Among medical reversals, 21 (84%) were prescription medications and 1 (4%) was convalescent plasma. 17 (68%) were repurposed medications. Two (8%) were procedures or mechanical interventions and one (4%) was a device. 16 (64%) reversals pertained to the hospital setting (4 to intensive care units), 4 (16%) were non-specific (ie, applicable to any setting), 4 (16%) pertained to a non-hospital setting and 1 pertained to healthcare workers.

Conclusion
When faced with a novel pandemic, policymakers rapidly made hundreds of specific medical recommendations. More than two out of three were never robustly tested. Among practices tested in a randomised fashion, one in three was made in error. Pandemic recommendation errors were substantial. Early and coordinated efforts to initiate randomised trials, even during dire situations, may mitigate the perpetuation of ineffective practices.

This Viewpoint discusses new pathways for researchers to seek collaboration when conducting studies on treatments for rare diseases and conditions.

This Medical News article is an interview with FDA Commissioner Robert Califf, MD, about the agency’s regulatory powers over artificial intelligence tools for use in health care and biomedicine.

Aideco, ‘il trattamento nasconde insidie se non è eseguito bene’

Aideco, ‘il trattamento nasconde insidie se non è eseguito bene’

Esperto, sempre consigliata specie nei pazienti ‘difficili’

The US Food and Drug Administration (FDA) defines drugs as in shortage when the total supply of all versions of a commercially available product cannot meet the current demand. Drug shortages are increasingly common, with more than 300 drugs classified as actively in shortage each quarter since the start of 2023. Many factors play a role in drug shortages, including low prices and limited competition, but shortages are often the result of issues related to manufacturing and quality as the drug supply chain has become increasingly globalized. These disruptions threaten the care and health of patients due to the unavailability of needed treatments.

To the Editor Robust and high-quality evidence generation for new drugs and devices benefits many parties. Ensuring that this evidence proves that treatments are beneficial is essential. As our health care system operates like a speeding train with no brakes immediately following US Food and Drug Administration (FDA) approval, reasonable evidence that benefits exceed harms must be obtained before approval. Although in the Special Communication article in JAMA, Dr Abbasi and colleagues proposed that it is up to payers to ensure that benefits exceed harms, it is actually the FDA that effectively determines the quality of evidence to ensure safety and effectiveness—especially in the role it plays helping sponsors to design premarket studies and evidence requirements. Although evidence can be gathered after FDA approval in the form of postmarket surveillance studies and registry data, it cannot replace high-quality randomized clinical trial data that once defined FDA approvals. Registry data lack a comparison group, making it unclear whether patients are better off with or without the treatment. Postmarket studies, when required, are often of low quality, use surrogate end points that lack clinical relevance, and may be incomplete or inaccessible. Disappointingly, even when studies are completed and show no benefit, treatments remain on the market.

The US Food and Drug Administration (FDA) recently approved a new treatment to prevent or reduce the frequency of bleeding episodes in patients 12 years or older with hemophilia A or hemophilia B. Whereas traditional hemophilia therapies use products that replace or mimic defective coagulation factors, marstacimab-hncq works by targeting a protein involved in blood clotting.

In prevenzione, diagnosi precoce, trattamento e riabilitazione