Day One Biopharmaceuticals’ Post

In McLean Hospital, Belmont we worked on clinical trials of buprenorphine-naloxone for opioid dependents in the NIDA Clinical Trials network (CTN). In a secondary analysis we identified that presence of pain predicts buprenorphine-naloxone dose levels in opioid-dependent youth, and that patients with pain have comparable opioid use outcomes to those without pain, but require higher buprenorphine-naloxone doses. Chakrabarti A, Woody GE, Griffin ML, Subramaniam G, Weiss RD. Predictors of buprenorphine-naloxone dosing in a 12-week treatment trial for opioid-dependent youth: secondary analyses from a NIDA Clinical Trials Network study. Drug Alcohol Depend. 2010 Mar 1;107(2-3):253-6. doi: 10.1016/j.drugalcdep.2009.10.014. Epub 2009 Nov 30. PMID: 19948382; PMCID: PMC2821971.

#RareDiseaseWeek2024 I am so honored to be advocating in DC this week, with 800 peers from all 50 states! We are asking legislators to 1) create an Interagency Collaboration Committee for Rare Diseases, & 2) to fund additional Rare Disease research for early diagnosis, treatment, & education for medical professionals. Fact 1: There are more than 10,000 known rare diseases and only a few hundred have safe, effective treatments...many still have NO treatments. Only 5% have FDA approved pharmaceutical drugs. #RareDC2024 #BeTheChange #RDLA Rare Disease Day EveryLife Foundation for Rare Diseases National Organization for Rare Disorders Disability:IN The Ehlers-Danlos Society Photo of several hundred people on the steps of the US Capitol building with the caption Rare Disease Week on Capitol Hill - Rare Disease Legislative Advocates, presented by Amgen

Big Pharma doing the right thing! "People with Down syndrome have a 90% risk of developing Alzheimer’s disease, and on average they receive their diagnosis at age 54, decades before the general population. Alzheimer’s disease currently leads to 70-80% of deaths in adults with Down syndrome. To date, no one with Down syndrome has been included in a clinical trial of donanemab or in trials of the other two anti-amyloid drugs: Leqembi (approved by the FDA in 2023) and Aduhelm (approved by the FDA in 2022)." #downsyndrome #downsyndromeawareness #idea #specialeducationadvocate #specialeducation #advocate #advocacy https://lnkd.in/gJWy_3cY

What can the results of a randomized controlled trial (RCT) tell you about a drug’s impact on your health? Well, probably not much, unless you meet the trial's specific inclusion and exclusion criteria—such as being male, between 25-40 years old, diagnosed with a particular stage or severity of the condition the drug treats, and otherwise completely healthy without any other medications. You get the point. This is why real-world evidence (RWE) and "intention to treat" analyses are essential for understanding the broader applicability of RCT results. The FDA’s recent final guidance, "Conducting Clinical Trials with Decentralized Elements," along with its draft guidance, "Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice," reflect this shift. These RCT elements aim to bring trial-related activities closer to patients, facilitating the enrollment of more diverse populations and ultimately leading to more generalizable trial results. We are gradually evolving from a model that emphasizes strictly controlled trials to one that prioritizes broader generalizability, recognizing the need for balance between rigorous study design and real-world relevance. I, for one, support this evolution, despite the challenges it may present. I believe we can navigate these challenges together! 😉 https://lnkd.in/dg-7GMHN #FDA #clinicaltrialinnovation #realworldevidence 

Take a look! NAESS helps improve the overall safety of babies enrolled in a clinical trial. #NAESS #NeonatalAdverseEvents #DrugSafety #PediatricResearch #AdverseEventScale #ReferenceValues #MedicalInnovation #DrugDevelopment #PediatricHealthcare #ClinicalMetrics

This is a good summary of the diversity of participants in clinical trials that led to CDER drug approvals in 2023. It would be useful if the report included information on prevalence of the disease within each the racial and ethnic subgroups reported. Prevalence information could further enhance interpretation of the racial and ethnic composition of the study populations. #FDA #CDER #diversity #clinicaltrials

Industry colleagues supporting neonatal clinical trials and drug development learn more about the Neonatal Adverse Event Severity Scale tool via this publication https://lnkd.in/gcv5ewX9. The paper discusses the potential to incorporate the tool into study protocols to improve efficiency and success of neonatal trials.

Tomorrow, a #FDA advisory committee will meet to discuss whether the #USA should approve its first psychedelic drug. The fate of the [#psychedelic] treatment—MDMA-assisted therapy for post-traumatic stress disorder—will turn on how the #FDA interprets data from two clinical trials that, on their face, are promising. Long-suffering patients who took the drug while undergoing intensive talk therapy were about twice as likely to recover from #PTSD as patients who got the placebo with therapy. If the treatment is approved this summer, it could bring relief to some of the approximately 13 million Americans with #PTSD. It could also serve as a model for other psychedelics to meet the FDA’s regulatory bar. But there’s a conundrum at the core of these two clinical trials, one that has plagued virtually all efforts to study psychedelics. https://lnkd.in/e7PmAB9K

From the Age Management Medicine Group (AMMG) E-Journal, FDA Fully Approves ‘Novel’ Alzheimer’s Disease Drug Legembi, Will Be Covered by Medicare: https://lnkd.in/evXKKT59. "The Food and Drug Administration (FDA) has fully approved a “novel” drug used to treat adult patients with Alzheimer’s, the agency announced. Leqembi, which is produced by Japanese drugmaker Eisai and American-based drugmaker Biogen, was initially approved in January under the Accelerated Approval pathway. After promising clinical trials, the drug was given traditional approval Thursday. “Leqembi is the first amyloid beta-directed antibody to be converted from an accelerated approval to a traditional approval for the treatment of Alzheimer’s disease,” the FDA explained in a press release. “The drug works by reducing amyloid plaques that form in the brain, a defining pathophysiological feature of the disease,” the statement added." #Legembi #AlzheimersDrug #PrecisionMedicine #AMMG #AgeManagementMedicine #CMEEducation #ContinuingMedicalEducation #CMEConferences

Did you know? There are lots of new drugs under development that are inching their way through the pipeline to one day become available to us to try! https://lnkd.in/gWT_UZxb Check out our Dry Eye Drug Pipeline page to see: -Who's recently submitted FDA approval paperwork? -Who's getting ready to submit? -How many new drugs are currently undergoing human clinical trials? If you ever feel discouraged about the future of dry eye treatment, lists like this can give you a window into the vast research and development going on towards better solutions that will work for more of us. While drugs are not the only pathway to improving life with dry eye disease, drug development remains an important source of hope for our community. #dryeye #optometry #ophthalmology #arvo2024 #dryeyedisease #dryeyezone