WCG IRB’s Post

FDA - Rare Disease Day - on March 1, 2024. 9:00 AM - 4:30 PM EST Topics include:  1. The legal framework for approving studies and medical products at FDA  2. What FDA does during review processes to approve medical products  3. Decentralized clinical trials and digital health technologies  4. Where to find important information and documents related to clinical trials  5. Information that can be obtained from medical product labels  6. Legal and ethical requirements for consent forms in clinical trials  7. FDA initiatives to advance medical product development for rare diseases  8. Ways for patients to engage with FDA 

Last week, we've had a listening session with the FDA discussing out Leigh syndrome global patient registry, including the ways we follow FDA registry guidance, and data interoperability with CDISC and OMOP. This listening session is our 2nd, following one in 2023. We are grateful for the opportunities to discuss our registry and hear FDA guidance, comments, and questions. #leighsyndrome #mitochondrialdisease

Good news for MD manufacturers involved in orphan or rare diseases, we now have an MDCG, MDCG 2024-10! Why is it important? As written in the introduction: "The level of clinical evidence that is required to place medical devices on the market has been increased by the MDR, including an increased need for pre-market clinical investigations for certain higher risk devices to verify their safety and clinical performance. These increased clinical evidence requirements present a challenge for devices specifically intended for use in rare diseases/conditions, or in specific indications for rare cohorts of patients with an otherwise non-rare disease/condition." #eumdr #clinicalevaluation #MDCG #orphandiseases #rarediseases #medicaldevices

#ExpandedAccess may be an option for patients with life-threatening diseases or conditions to try investigational medical products when no satisfactory or approved therapies are available and there is no opportunity for the patient to enroll in a clinical trial. Learn more about Expanded Access and how it could help your patients here: https://loom.ly/wVLb_4I #ExpandedAccess #physicians #healthcare

We are delighted to inform you and your team that the Clinical management of diphtheria guideline has been published. The guideline includes 3 recommendations on : Antibiotic treatment: In patients with suspected or confirmed diphtheria, WHO recommends using macrolide antibiotics (azithromycin, erythromycin) in preference to penicillin antibiotics [Strong recommendation, low certainty evidence]. DAT sensitivity testing: In patients with suspected or confirmed diphtheria, WHO recommends not to perform routine sensitivity testing prior to administration of diphtheria antitoxin [Strong recommendation, moderate certainty evidence]. DAT dose: In patients with suspected or confirmed symptomatic diphtheria, WHO suggests administration of a single dose of diphtheria antitoxin with choice of dose based on disease severity and time since symptom onset, in comparison with a fixed dose for all patients [Conditional recommendation, very low certainty evidence]. The guideline is available on the following links: IRIS: Clinical management of diphtheria: guideline, 2 February 2024 (who.int) MAGICapp: Clinical management of diphtheria: guideline (magicapp.org) WHO website: Clinical management of diphtheria (who.int) Many thanks for your great support and continued collaboration with all our great experts ! #safescalableclincalcare #clinicaloperations #diphtheria #WHE #gdg Steve McGloughlin Jamie Rylance Julie Viry Gordon Guyatt Srinivas Murthy

Need some inspiration for the new year? KCR Head of Trial Execution Services, Erin Phillips Philips, can help! Check out this clip to hear Erin explain how prioritizing #solutiondelivery allows her to focus on the thing that matters most: helping #patients. #KCRCRO #ClinicalResearch #innovation #medicine #KCRSolutions #KCRTrialExecution #KCRatWork #PatientFocused #Research #ClinicalTrials

#TARGET BP I is a prospective, international, sham-controlled, randomized, patient- and assessor-blinded trial investigating the safety and efficacy of alcohol-mediated RDN. After 301 #hypertensive uncontrolled patient randomisation, #RDN was associated with a significant reduction in 24-hour ambulatory SBP at 3 months (mean ± standard deviation -10.0 ± 14.2 mmHg versus -6.8 ± 12.1 mmHg, treatment difference -3.2 mmHg, 95% confidence interval [CI] -6.3, 0.0 mmHg; P=0.0487). Adverse safety events through 6 months were uncommon. This is the 7th trial positive in the field.

How wrong and misleading can Physicians for Informed Consent be ? Or stupid ? Or at least lying to you all. The #MRR is one of the most studied vaccine within more than half a billion doses given so far and such extent of data dismisses any rare #adversevents or AEs with delayed onset. A recent Cochrane review of 124 studies assessing vaccine effectiveness showed that two doses of MMR vaccine were 96% effective in preventing measles, and one dose 95% effective. Two doses of MMR vaccine are also around 86% effective against mumps, and 89% effective against rubella. Public Health England estimates that 20 million cases and 4,500 deaths have been prevented in the UK since MRR vaccine was introduced in 1968. It has prevented around 1.4 million cases of rubella and 1,300 cases of birth defects and averted 25,000 terminations. There is no evidence of any link between the MMR vaccine,autism or other pretended rare AEs. Below is a list of studies and their findings. A Danish study of over 650,000 children found no increased risk for autism after MMR vaccination (Hviid et al., 2019 ) An analysis of studies involving over 1 million children found no relationship between vaccination and autism. There was no evidence of a link between the MMR vaccine and autism development in children, and the study also found no evidence of a link between thiomersal and autism development (Taylor et al., 2014 ). There is no increased incidence of autism in children vaccinated with MMR compared with unvaccinated children (Farrington et al., 2001 ;  Madsen and Vestergaard, 2004 ). There is no clustering of the onset of symptoms of autism in the period following MMR vaccination (Taylor et al., 1999 ; Mäkelä et al., 2002 ). The increase in the reported incidence of autism preceded the use of MMR in the UK (Taylor et al., 1999 ). The incidence of autism continued to rise after 1993 in Japan despite withdrawal of MMR (Honda et al., 2005 ) There is no correlation between the rate of autism and MMR vaccine coverage in either the UK (Kaye et al., 2001 ) or the USA (Dales et al., 2001 ) There is no difference between the proportion of children with a regressive form of autism (i.e. who appear to develop normally but then lose speech and social skills between around 15 and 30 months) who develop autism having had MMR compared with those who develop autism without vaccination (Fombonne and Chakrabarti, 2001 ; Taylor et al., 2002 ). There is no difference between the proportion of children developing autism having had MMR who have associated bowel symptoms compared with those who develop autism without vaccination (Fombonne and Chakrabarti, 2001 ; Taylor et al., 2002 ) No vaccine virus can be detected in children with autism using the most sensitive methods available (Afzal et al., 2006 ; D’Souza et al., 2006 ). https://lnkd.in/gy5cwp2C https://lnkd.in/gyAiWiNj

Considering a Clinical Trial for IPF?   If you or a loved one are living with #IPF (Idiopathic Pulmonary Fibrosis), you might be exploring every avenue for better treatments and a brighter future. Clinical trials could be the key to unlocking groundbreaking advancements in IPF care.  It's important to weigh the benefits and potential risks and have open discussions with your healthcare provider before selecting a clinical trial as a treatment option. Stay connected with us on our Clinical Trial for patients with IPF by following the link below: https://lnkd.in/gXRNn-3A #IPFAwareness #ClinicalTrials #HopeForIPF

Mealtime Insulins: get updated on the newest formulations, differentiating factors, and clinical evidence | Leveraging Clinical Evidence on Prandial Insulins to Achieve Patient Goals | 0.25-hour CME/CE | Booster Quiz | #MedEd #cme #diabetestreatment | https://bit.ly/3wsj6rT