Firma Clinical Research’s Post

Two phase 3 clinical trials have completed enrollment and will evaluate sozinibercept for patients with wet age-related macular degeneration (AMD). View the complete article below ⤵️ #MacularDegeneration #AgeRelatedMacularDegeneration #ClinicalTrial

Learn how addressing participant concerns enhances the safety of clinical trials in our latest blog post. https://lnkd.in/eSPTsNdb #clinicaltrials #CRO

Why Sham Studies and Clinical Trials Matter in Efficacy and Resource Allocation In the world of clinical practice, we often encounter a plethora of high-dollar courses and treatments, each marketed with impressive claims and supported by a network of enthusiastic clinicians. While many of these options seem promising, my approach to evaluating their efficacy involves a keen appreciation for sham studies and clinical trials. Sham studies, which include placebo-controlled trials, are incredibly valuable. They help us discern whether a treatment's benefits are genuinely due to the intervention or merely a result of patient expectations and the placebo effect. This approach allows us to differentiate between treatments that truly offer efficacy and those that might only provide superficial or temporary benefits. Understanding the role of sham studies helps me allocate my time, reasoning, and resources more effectively. By assessing whether a technique or treatment has demonstrated true efficacy beyond the placebo effect, I can better decide putting my energy or resources towards learning. This critical evaluation prevents us from being swayed by flashy marketing or popular trends and ensures we focus on interventions that deliver real value to our patients. Sham studies and well-designed clinical trials provide a clearer picture of what works and what doesn’t, helping us make informed decisions and use our resources wisely. This approach enhances our practice and ultimately benefits our patients by prioritizing evidence-based, effective treatments over those that may be more about flair than substance.

In the latest #TalkClinicalTrials blog, learn more about the barriers to #type1diabetes clinical trial participation and JDRF/FRDJ Canada's new T1D Clinical Trials Finder. Read the blog post here: https://bit.ly/48SNR6D #ClinicalTrials #ClinicalTrialParticipation

👏 SpineX Inc. has closed recruitment for a clinical study investigating its neuromodulation device to treat urinary incontinence. Data from the trial, which began in May 2022, will be used to support a De Novo submission to the US Food and Drug Administration “in the coming months”, according to SpineX’s CEO Parag Gad. 🔗 Read more: https://lnkd.in/ehY4xj2n 📰 Follow Guided Solutions to receive the latest #MedTech news daily and subscribe to our weekly newsletter: https://lnkd.in/eBu_EP6V #MedicalDevices #MedicalDevice #MedicalEquipment #Medicine #Surgeons #GuidedSolutions

Study exploring the impact of anticholinergic burden on urinary independence: insights from a post-stroke cohort of older adults. Increased anticholinergic burden in post-stroke patients who require assistance with urination is significantly associated with less independent urination. Anticholinergic agents may need to be introduced cautiously in patients who require assistance with urination. Derek Stewart European Society of Clinical Pharmacy (ESCP) Yolande Hanssens Vibhu Paudyal Shusen Sun Filipa Alves da Costa Matej Štuhec PharmD PhD Univ.-Prof. Dr. Anita Weidmann #pharmacy #pharmacists #pharmacylife #clinicalpharmacy #pharmaceuticalcare #pharmacycollege #pharmacystudents #pharmacytechnicians https://lnkd.in/dVnn_guF

Experience matters when you’re running a clinical trial. Our expert CRAs and project managers apply the knowledge they've gained across a range of therapeutic areas and phases to every trial they work on. Find out more and let us set you up for success. https://buff.ly/2TvQEhr #clinicaltrials #CRO #clinicalresearch #consultants #testimonials

The Swedish National Formulary for Children ePed comments on Shaniv et al. A callout for international collaboration on neonatal (and paediatric) drug formularies. ePed is not only a useful neonatal and paediatric formulary that provides medication information to doctors, nurses and pharmacists in Sweden. It offers much more. Read the article and you will see. #ePed ePed-Nationellt Beslutstöd för Läkemedel till Barn #neonatology, #children, #medicationsafety, #patientsafety, #mdpichildren,

Lutetium Lu 177 Dotate has been approved by the FDA for treatment of pediatric patients aged 12 years and above. According to Dr. Anish K. Shah, "The approval was based on the pharmacokinetic (PK), dosimetry, and safety data frombNETTER-P, an ongoing, international, multi-center, open-label, single-arm study ofblutetium Lu 177 dotatate in adolescent patients with locally advanced/inoperable or metastatic SSTR-positive GEP-NETs." Read to learn more about this option for #pediatriccancerpatients ⬇ https://lnkd.in/gMfHE6Sv

  Last month, ICER’s Chief Medical Officer, Dr. David Rind, published an editorial in JAMA outlining Sarepta’s available evidence for Elevidys, as the company seeks full approval and label-broadening, in the absence of a traditional evidence package.  The editorial is behind the JAMA paywall, so here are some highlights that are worth noting in the ~10 day lead-up to Sarepta’s action date next Friday, June 21 (though, of course, it could come sooner).    Recall, Sarepta holds Accelerated Approval and has a price tag of $3.2 million dollars.  Additionally, Sarepta does not appear to be offering any type of warranty, rebate or Outcomes Based Agreement (please educate me if this statement is incorrect).  In spite of failing their primary endpoint in a confirmatory trial, Sarepta is now asking the FDA for a conversion to full approval, expansion of age on label beyond 4-5 years olds and expansion to non-ambulatory patients. This is a very big, multi-faceted, complex precedent-setting ask. The thing to watch here is what type of signals the FDA decision sends to those seeking Accelerated Approval moving forward.    ICER Highlights:   + "It seems clear that the evidence for net benefit with SRP-9001 is weak. It also appears that if benefits with SRP-9001 are real, they are not large, although it may be that longer trials could show greater effects." + "Although accelerated approval by the FDA was purportedly based on the surrogate outcome of microdystrophin production, the FDA only granted this approval for treatment of boys aged 5 or 5 years." + "Cost is also an issue that cannot be ignored. Sarepta Therapeutics has priced Elevidys at $3.2 million. This is an enormous price tag for a therapy that has failed to meet its primary end point in the 2 randomized trials in which it has been studied and that is clearly not curative." The next question becomes, depending on FDA's response to Sarepta's asks, will payers begin to push back on a $3.2M price? There have been whispers among payers about limited evidence warranting a more limited reimbursement, until confirmatory trials produce evidence to justify the price. Will FDA's pending decision be a tipping point? #sarepta #atmpt #genetherapy #cgt #elevidys #duchenne