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TY Epstein Becker & Green, P.C. for this latest read on the #FDA's draft guidance "Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies Guidance for Industry" Enhancing diversity within clinical studies facilitates the broader applicability of results across a broad spectrum of patient populations, as well as enhances understanding of the disease or medical product under study. Valuable insights inform the safe and effective use of the medical product among patients. Read up all #HealthEquityWarriors Michael Garrett RISE CMSA Dr. Ulysses Grant Baldwin, Jr. Daniel Dawes DeAnna Minus-Vincent #DEIB #HealthEquityPillarsforSeriousIllness #HEPSI #ClinicalTrialsAccess #DiversityActionPlan #ClinicalStudies #HealthInequities

How can life sciences companies strengthen post-market surveillance of ADEs? Longitudinal linking may be the key 🔑 Individual social media posts contribute somewhat to safety surveillance, but limited character counts and spur of the moment posts that only tell part of the story create bias and research challenges. Inspire conducted a study on behalf of a major life sciences corporation that linked Inspire member-generated content from the same individual over a 10-month period. The goal was to determine whether longitudinal linking of individual posts could provide a more holistic assessment of an individual’s chronic disease journey. Inspire uncovered that linking this data provided richer relevant medical findings and nontraditional insights, such as: -adverse events reporting -prevention and impact of adverse events -medical history -use of non-medical treatments -concomitant medications -environmental factors Read the full case study here: https://lnkd.in/eZfscV8b

The first ever treatment for metabolic dysfunction-associated steatohepatitis (MASH) was approved earlier this year, and several other promising therapies are progressing through clinical development. How does your investigational MASH drug compare? Certara's CODEX clinical outcomes database contains curated trial data for the first approved MASH drug and the most promising therapies in clinical development. The clinical outcomes data available via CODEX gives you the ability to compare your drug profile against competing products for efficacy and biomarker endpoints. Download our CODEX MASH brochure today: https://lnkd.in/eNmTp-ri

How could the IRA change the economic incentives surrounding the clinical development of new treatments and impact patients? Read the new study published in Health Affairs Forefront examining what we can learn from three case studies. https://bit.ly/3Smn3XL

Speed up clinical trials with #syntheticdata 🚀

Welcome back to TUESDAY THEOREMS! Here's Theorem #4 for Tuesday, March 26 Is the Hallowed Gold Standard in Clinical Research Fool's Gold?   Virtually every textbook on clinical research or biostatistics describes randomized control trials ("RCT" for short) as "the gold standard" in clinical research. But an important question remains: is that 24-karat, 18-karat, or 10-karat gold?   In some circumstances, RCTs, even in their most rigorous versions, have been shown to be much less than 24-karat. Uncritical acceptance of a claimed gold standard creates the risk of being deceived by "Fool's Gold".   The main weakness of even the best RCTs is easy to summarize: the strict inclusion and exclusion protocols for subjects to be included in an RCT trial (whether for a new drug, surgical procedure, or clinical care practice) prevent RCTs from fully reproducing the actual use experience of patients in the "real world".   Phase IV tests designed to pick up actual problems after months or years of use in the real world are far less frequently done than the Phase I, II and III trials typically done to get a new drug to market for large pharmaceutical manufacturers. According to the U.S. Govt's Central Registry of clinical trials at ClinicalTrials.gov, only 25%, or one in four, of newly marketed drugs receive a Phase IV assessment.   This explains the somewhat unusual excitement in the medical literature when research - often from smaller American locations or Scandinavian research centers (where drug company funding for medical research is more closely monitored) - appears with titles such as "Real world results with Drug X,” or “Real world results with Procedure Y."   Of course, one might well wonder when these relatively rare Phase IV or "real world” results are published in reputable peer-reviewed journals, to what world then do the much touted Phase III trial results - upon which FDA approval often depends – actually apply?   Technically, RCTs provide only summary results, even for subpopulations of the RCT study population. Within those summary results lie patients for whom the new drug or procedure is not effective; and, for some others, is even harmful.   This problem with RCTs, the much acclaimed “Gold Standard”, has given rise to a new kind of experimental design termed “N of 1” trials, wherein patients become their own controls.   While RCTs are not perfect, (and leaving for another day the many technical problems with the multivariate statistical procedures usually employed to analyse RCT data), RCT trials remain an indispensable tool in the medical researcher's tool kit.   In the difficult and complex quest to unravel disease and formulate better treatments, we cannot afford to let the perfect be the enemy of the good. Nonetheless, “Caveat Emptor” applies.   Stay tuned for next week's Theorem: "The Strange Case of Dr. Moore" all best, BRJ        

What an inspiring and insightful conversation today about best practices and future opportunities for including the voice of the patient in every step of the #drugdevelopment process. The discussion quickly ran out of time and many of the more than 100+ audience comments unfortunately had to go unaddressed. But, this is just the beginning of the conversation - submit your thoughts and recommendations to the docket using this link https://lnkd.in/dBYZp63A Some of the key take-aways for me included the need to evolve the public discourse on the issue from the theoretical and abstract to tangible recommendations and practical advice; the richness and value that differences in opinion, cultural heritage, and lived experience bring to this important discussion (and make for a very lively debate); and the foundational importance of trust, shared expectations, and clear and effective two-way communication. Our patients do not have time to waste on failed clinical trials and ineffective drug development programs. The best (and often only) way to ensure rare disease drug development programs have the maximum chance of success is to ensure everyone - sponsors, CROs and trial sites, patients and families, regulators, academic researchers, and more - is on the same page and that no program for our patients is developed without meaningful engagement of our patients. Nowadays, nobody would even think to develop a household good or service without meaningful end-user engagement - and rare-disease therapies are infinitely more complex and important to our patients and families. We see that patient experience data is increasingly central to #raredisease drug development and today's workshop certainly reinforced that. Thank you to FDA and the Duke-Margolis Center for Health Policy for including me and the National Organization for Rare Disorders in this important discussion!

Sy Mukherjee at Fast Company notes it is ‘…just the beginning for biopharma’s obesity-drug arms race.’ Eli Lilly and Company raked in over $5 billion in sales in 2023, whilst Novo Nordisk landed almost four times as much at over $18 billion. The potential market size for obesity and related health conditions may serve ‘…numbers in the tens of millions…’ of Americans, with over a billion worldwide.   Reports of an obesity drug market to reach over $100 billion in sales by the end of the decade are not wild or out of sorts. Competitors want to relieve costs for the pricey treatments, resolve supply issues, and sell better drugs – superior weight loss, less frequent dosing, and possibly oral GLP-1 pills. Not unsurprisingly, there are ‘…486 active studies of GLP-1 drugs and their effects on various types of patients and diseases currently listed in the federal ClinicalTrials.gov database…’ which makes me shudder at the total costs being poured into drug development, clinical trials, marketing, and distribution of these medications – anyone want to pick a number? https://lnkd.in/eVeYFUGH

Do you know the importance of your loved ones' participation in a clinical trial? In this link you'll learn about the pros and cons, what can you expect, myths, more background information, and featured clinical trials. LEARN MORE: https://lnkd.in/ehCSUijK

Bridging the gap between clinical trials and real-world patient experiences is crucial for advancing healthcare. Explore the evolving role of real-world evidence in drug development with insights from Rebecca Miksad, MD, MMS, MPH.