Clinical Trial Fundamentals

🚨 Major News in IBD: The FDA has approved TREMFYA® for subcutaneous induction in adults with moderately to severely active ulcerative colitis. TREMFYA® is now the first and only IL-23 inhibitor to offer a fully subcutaneous induction and maintenance regimen for UC and Crohn’s disease, giving patients and healthcare providers flexibility and choice from the very start of treatment. The European CHMP today has issued a positive opinion recommending the same regimen, with final European Commission approval expected soon. Clinical Highlights (ASTRO Phase 3 trial): - 26% achieved clinical remission at Week 12 vs 7% on placebo - 36% achieved endoscopic improvement at Week 12 vs 12% on placebo - Symptomatic improvements seen as early as Week 2 SC efficacy comparable to IV induction, with sustained results through Week 24 TREMFYA® represents: ✔ First IL-23 inhibitor with dual induction routes (SC & IV) ✔ Fully subcutaneous option for self-administration ✔ Clinically meaningful improvements in both clinical and endoscopic outcomes 👏 Congratulations to the IBD community and Johnson & Johnson on this landmark advancement #immunology #CGTweekly #biotech #biopharma

Most people arguing about drug pricing don’t understand all the steps it takes for drugs to actually get approved. --- Understanding the FDA approval process helps explain why new drugs cost what they do, why launch timelines matter for coverage decisions, and how safety data evolves over a drug's lifecycle. Here are the five phases every drug must work through. 1️⃣ DISCOVERY AND DEVELOPMENT Researchers identify promising compounds through disease mechanism insights, molecular screening, and new technologies, then study how the drug is absorbed, metabolized, dosed, and how it compares to existing treatments. 2️⃣ IND AND EARLY HUMAN TESTING Before human testing, compounds undergo in vitro and in vivo studies following Good Laboratory Practice standards to establish basic safety and toxicity profiles. 3️⃣ CLINICAL RESEARCH After FDA reviews the Investigational New Drug application, multi-phase human testing begins. Phase 1: 20-100 participants, several months ↳ Tests: Safety and dosage ↳ 70% advance to Phase 2 Phase 2: Up to several hundred patients, months to 2 years ↳ Tests: Efficacy and side effects   ↳ 33% advance to Phase 3 Phase 3: 300-3,000 patients, 1-4 years ↳ Tests: Confirming efficacy, monitoring adverse reactions ↳ 25-30% ultimately receive approval 4️⃣ FDA DRUG REVIEW A multidisciplinary FDA team conducts a 6-10 month comprehensive review of all preclinical and clinical data, inspects trial sites, and may convene Advisory Committees before making an approval decision. 5️⃣ POST-MARKET DRUG SAFETY MONITORING FDA continues monitoring through facility inspections, adverse event reporting systems like MedWatch, and active surveillance using electronic health databases to track safety as real-world use evolves. Phase 4: Post-approval surveillance ↳ Thousands of participants in real-world monitoring --- The complete process takes 10-15 years and $1-3 billion per approved drug. Many drugs will continue to pursue new indications post-launch, which further increases the cost, but also the return (to pharma) in increased utilization and patent extension. The patients with those new indications are also more likely to get coverage with a formal approval. Does understanding this timeline change how you view drug pricing? ♻️ Repost to help pharmacy leaders understand the drug development process. 🔔 Follow me for more content like this (Bryce Platt, PharmD)

Before a psychiatric diagnosis can be made, it has to survive a medical workup. This is one of the most important, and most consistently missed, steps in mental health care. The brain runs on glucose, oxygen, hormones, nutrients, and an immune system it shares with every other organ. When any of those inputs are disrupted, the brain produces symptoms indistinguishable from psychiatric illness. Examples of what gets missed every day: ⚕️ Hypothyroidism can present as depression. Studies show up to 26.2% of depressed patients have abnormal thyroid function. Treating the thyroid condition should be the first step. ⚕️ Hyperthyroidism can look like anxiety or mania: racing thoughts, insomnia, irritability, and palpitations mimicking panic disorder or hypomania. ⚕️ Iron deficiency can mimic ADHD or depression. Iron is important in dopamine synthesis, and testing without ferritin levels routinely misses this etiology. ⚕️ B12 deficiency can manifest as depression, cognitive decline, and in severe cases psychosis. Neurological impairment can occur at levels considered normal by standard reference ranges. ⚕️ Sleep apnea can present as depression and ADHD: cognitive fog and low energy are driven by chronic hypoxia and fragmented sleep. ⚕️ POTS and dysautonomia can look like anxiety, depression, and cognitive dysfunction, driven by cerebral hypoperfusion when upright, not primary psychiatric illness. ⚕️ PANDAS/PANS manifests as sudden-onset OCD, tics, anxiety, and behavioral change in children. The hallmark is dramatic onset, often overnight, following streptococcal or other infection. ⚕️ Autoimmune encephalitis can present as psychosis. 75% of anti-NMDA receptor encephalitis patients first present to a psychiatrist. It is treatable, but can be fatal when missed. ⚕️ Cushing's disease can look like bipolar disorder: cortisol excess produces mood instability, insomnia, and cognitive impairment. Here's what a basic workup for any psychiatric condition should include: → CBC, CMP, TSH, free T4, B12, folate, vitamin D, iron studies with ferritin, fasting glucose, HbA1c, lipid panel, CRP, urinalysis, toxicology screen. Suspected POTS: → Orthostatic vitals (supine, sitting, standing at 1, 3, and 10 minutes); tilt table test if indicated. Suspected PANDAS/PANS: → Throat culture, rapid strep, ASO titer, anti-DNase B antibody, CBC with differential, ANA, ESR, CRP, mycoplasma IgM/IgG. First-episode psychosis: → Add ANA, anti-NMDA antibodies, HIV, RPR, neuroimaging. Treating a medical condition as a psychiatric one can cost years of a patient's life. The most evidence-based first step in mental health care is ruling out the medical conditions that produce psychiatric symptoms. We should be doing it every time. Follow me Britton Ashley Arey, MD MBA for evidence-based perspectives on psychiatry, mental health, and the science of the mind. #Psychiatry #MentalHealth #IntegratedCare #MindBodyMedicine #MentalHealthAwarenessMonth

🚨 𝗕𝗿𝗲𝗮𝗸𝗶𝗻𝗴 𝗻𝗲𝘄𝘀 𝗳𝗿𝗼𝗺 𝗙𝗗𝗔 Drug approval no longer starts with a trial. It starts with a mechanism. The FDA has introduced the “𝗽𝗹𝗮𝘂𝘀𝗶𝗯𝗹𝗲 𝗺𝗲𝗰𝗵𝗮𝗻𝗶𝘀𝗺” 𝗽𝗮𝘁𝗵𝘄𝗮𝘆: a new route to approve bespoke therapies when classic trials are impossible.  Think N-of-1 gene editing for ultra-rare, often fatal childhood diseases. 𝟱 𝗲𝗹𝗲𝗺𝗲𝗻𝘁𝘀 𝘁𝗵𝗲 𝗙𝗗𝗔 𝘄𝗮𝗻𝘁𝘀 𝘁𝗼 𝘀𝗲𝗲 𝗯𝗲𝗳𝗼𝗿𝗲 𝗮𝗽𝗽𝗿𝗼𝘃𝗮𝗹 1️⃣ 𝗖𝗹𝗲𝗮𝗿 𝗺𝗼𝗹𝗲𝗰𝘂𝗹𝗮𝗿 𝗰𝗮𝘂𝘀𝗲 A single, well-defined genetic or molecular defect driving the disease. 2️⃣ 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗱𝗲𝘀𝗶𝗴𝗻𝗲𝗱 𝘁𝗼 𝗳𝗶𝘅 𝘁𝗵𝗮𝘁 𝗱𝗲𝗳𝗲𝗰𝘁 The product must target the precise mechanism: the edit, splice, or RNA change that corrects the biology. 3️⃣ 𝗪𝗲𝗹𝗹-𝘂𝗻𝗱𝗲𝗿𝘀𝘁𝗼𝗼𝗱 𝗻𝗮𝘁𝘂𝗿𝗮𝗹 𝗵𝗶𝘀𝘁𝗼𝗿𝘆 Strong historical data showing how the disease progresses without treatment, so real benefit is clear. 4️⃣ 𝗘𝘃𝗶𝗱𝗲𝗻𝗰𝗲 𝗼𝗳 𝘁𝗮𝗿𝗴𝗲𝘁 𝗲𝗻𝗴𝗮𝗴𝗲𝗺𝗲𝗻𝘁 Proof the therapy does what it’s meant to do, through biomarkers, biopsies, or validated non-animal models. 5️⃣ 𝗠𝗲𝗮𝗻𝗶𝗻𝗴𝗳𝘂𝗹 𝗰𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗯𝗲𝗻𝗲𝗳𝗶𝘁 Improvements strong enough that they cannot be dismissed as noise. A single patient can serve as their own control. If a platform shows success in several different patients, even with unique bespoke edits, the FDA can move toward platform-level authorization, not just case-by-case exemptions. 👉 𝗪𝗵𝘆 𝘁𝗵𝗶𝘀 𝗺𝗮𝘁𝘁𝗲𝗿𝘀 𝗳𝗼𝗿 𝗽𝗮𝘁𝗶𝗲𝗻𝘁𝘀  For families facing ultra-rare genetic diseases, the old logic was brutal: too rare for trials → no drug → no options. This pathway changes that:  • From “too rare to study” → “biologically defined and actionable.”  • From isolated compassionate-use miracles → a structured regulatory route.  • From decade-long timelines → months from design to first dosing in the most urgent pediatric cases. And it does not end at approval:  • Long-term real-world evidence  • Ongoing monitoring for off-target edits, immune issues, developmental risks  • Registries to track durability and outcomes 👉 𝗪𝗵𝗼 𝗯𝗲𝗻𝗲𝗳𝗶𝘁𝘀 𝗳𝗶𝗿𝘀𝘁?  • Infants with lethal monogenic diseases  • Ultra-rare disorders with a single known driver mutation  • Small, genetically defined subsets in oncology and immunology 𝗜𝘁’𝘀 𝗮 𝗯𝗶𝗼𝗹𝗼𝗴𝘆-𝗳𝗶𝗿𝘀𝘁, 𝗽𝗹𝗮𝘁𝗳𝗼𝗿𝗺-𝗯𝗮𝘀𝗲𝗱 𝗮𝗽𝗽𝗿𝗼𝗮𝗰𝗵 𝗯𝘂𝗶𝗹𝘁 𝗳𝗼𝗿 𝗰𝗼𝗻𝗱𝗶𝘁𝗶𝗼𝗻𝘀 𝘄𝗵𝗲𝗿𝗲 𝗹𝗮𝗿𝗴𝗲 𝘁𝗿𝗶𝗮𝗹𝘀 𝘄𝗶𝗹𝗹 𝗻𝗲𝘃𝗲𝗿 𝗯𝗲 𝗽𝗼𝘀𝘀𝗶𝗯𝗹𝗲. It’s a design guide for how to build your next platform and IND package. Because for many families, this is the first time “𝘆𝗼𝘂𝗿 𝗯𝗶𝗼𝗹𝗼𝗴𝘆 𝗶𝘀 𝘂𝗻𝗶𝗾𝘂𝗲” doesn’t automatically mean “𝘆𝗼𝘂’𝗿𝗲 𝗼𝗻 𝘆𝗼𝘂𝗿 𝗼𝘄𝗻.”

Elsevier’s Researcher Academy is offering a set of FREE mini-courses that actually hit the pain points we all have: 📚 How to write a literature review 🔎 How to identify research gaps 🧪 How to conduct evidence-based research 📄 How to write an abstract & cover letter 💰 How to secure funding 🤖 Plus: Using Gen AI in research (with ethics!) These are short, practical videos (8–60 mins) you can squeeze in between experiments, clinic, or teaching. Perfect if you’re preparing a manuscript, supervising students, or just leveling up your research skills. I’m sharing this because good research isn’t just about data — it’s about presenting it so it gets published, cited, and funded. 

FDA just changed the rules of the oncology game: survival is the scoreboard. For years, companies leaned on surrogate endpoints—progression-free survival, tumor shrinkage, biomarkers. Flashy. Fast. But not always life. Now the FDA’s draft guidance makes it clear: → Overall survival (OS) should be the primary endpoint in randomized cancer trials when feasible → Even when it’s not, sponsors must collect and prespecify OS data—because safety and efficacy live there → Design fixes are coming for crossover, unequal randomization, and long-term follow-up Why it matters: as FDA and AACR leaders put it, we’ve seen cases where progression-free survival looked great—but patients didn’t live longer. Sometimes, they lived shorter. That’s unacceptable. This is a reset. Biotechs, CROs, CDMOs: if your trials don’t put survival front and center, you’re not future-proof—you’re playing the wrong game. The only metric patients care about is time. Is your company ready to measure up?

𝕊𝕄𝔸ℝ𝕋 𝕄𝔼𝔻𝕀ℂ𝕀ℕ𝔼 ⁣⁣⁣⁣⁣⁣⁣⁣⁣⁣⁣⁣⁣ #𝐒𝐌𝐀𝐑𝐓𝐌𝐄𝐃𝐈𝐂𝐈𝐍𝐄 Video #intubation: simple and easy steps Used in difficult airway or when better visualization is needed. Preparation Explain to the team what you're doing Check equipment: video laryngoscope, blade, ET tube (with stylet), syringe, suction, bag-valve-mask, monitor Position the patient: sniffing position if no c-spine injury, ramped if obese Pre-oxygenate with 100% O2 for at least 3 minutes or 8 vital capacity breaths Steps 1. Insert blade midline Gently open the mouth Advance the blade midline until you see the epiglottis 2. Visualize glottis Lift the epiglottis with blade tip (Mac) or insert past and lift (GlideScope) Don’t lever on teeth Confirm you see vocal cords clearly on screen 3. Insert the tube Advance the ET tube under direct vision through the cords Watch the tube pass between the vocal cords on the screen Advance until cuff is just past the cords (2–3 cm) 4. Remove stylet and confirm placement Inflate cuff with air Bag the patient while checking: Equal chest rise Equal breath sounds No epigastric sounds Capnography (ETCO₂ wave) Monitor oxygen saturation 5. Secure the tube Note the depth (usually 21 cm for females, 23 cm for males) Fix the tube with tape or tube holder Reassess frequently Tips Use anti-fog if screen is foggy If you see esophagus only, adjust head or blade position Always have backup plan: bougie, LMA, cricothyrotomy kit 🩺 𝐃𝐑 𝐀𝐇𝐌𝐄𝐃 𝐑𝐄𝐃𝐀 ⁣⁣⁣⁣⁣⁣ https://lnkd.in/d875vszs⁣ #AirwayManagement #VideoIntubation #EmergencyMedicine #CriticalCare #Intubation #MedicalEducation #DoctorsOfLinkedIn #PatientSafety #HospitalLife #ClinicalSkills

𝗙𝗟𝗨𝗜𝗗 𝗢𝗙 𝗖𝗛𝗢𝗜𝗖𝗘 1. Diarrhea → Ringer’s Lactate (RL) Rationale: 🔴Diarrhea leads to loss of both water and electrolytes, especially bicarbonate, causing metabolic acidosis. 🔴RL contains lactate, which is converted to bicarbonate in the liver, helping correct the acidosis. 🔴RL also closely resembles extracellular fluid, making it ideal for volume replacement. 2. Vomiting → Normal Saline (NS) Rationale: 🔵Vomiting causes loss of hydrogen ions (HCl) from the stomach, leading to metabolic alkalosis. 🔵NS (0.9% NaCl) helps restore chloride and sodium, which helps the kidneys excrete bicarbonate, correcting the alkalosis. 3. Hepatorenal Syndrome (HRS) → Albumin Rationale: 🟡HRS is a functional renal failure in advanced liver disease. 🟡Albumin, a colloid, expands plasma volume and helps improve renal perfusion by increasing oncotic pressure, especially when combined with vasoconstrictors. 4. Sepsis → Crystalloids Rationale: 🟣Early goal-directed therapy in sepsis includes aggressive fluid resuscitation. 🟣Crystalloids (e.g., NS or balanced solutions like RL) are first-line due to wide availability and cost-effectiveness. 🟣Helps correct hypovolemia and maintain tissue perfusion. 5. With BT (Blood Transfusion) → Normal Saline (NS) Rationale: 🟢NS is isotonic and does not cause hemolysis of red blood cells. 🟢RL contains calcium, which can bind to citrate in stored blood, potentially causing clotting, so it's avoided. 6. Diabetic Ketoacidosis (DKA) → Normal Saline (NS) Rationale: 🟤Initial fluid of choice is NS to correct hypovolemia and electrolyte loss. 🟤Once blood glucose reaches around 200–250 mg/dL, fluids may be changed to 5% dextrose with 0.45% saline. 7. Sweating → ½ Normal Saline (0.45% NaCl) Rationale: ⚪Sweat loss is hypotonic, leading to hypernatremia. ⚪½ NS is also hypotonic, which helps gently lower serum sodium and replenish lost fluid. 8. Head Injury → Hypertonic Saline (HS) > Normal Saline (NS) Rationale: ⚫Hypertonic saline (e.g., 3% NaCl) reduces intracranial pressure (ICP) by drawing fluid out of the brain via osmosis. ⚫NS can be used, but HS is preferred in raised ICP scenarios. Pic credit https://lnkd.in/gU2ttxbS

No Experience? No Problem. How to Break Into Clinical Research with a Life Science Degree Let’s be real: A four-year life science degree gets you in the door, but without experience, clinical research can feel like a locked room. Here’s the truth: You don’t need years of experience to get started, you need strategy. Here are 4 Ways to Land Your First Clinical Research Role: 1. Start Where You Are, Leverage Your Degree Your background in biology, chemistry, or health sciences is the foundation. Now add ICH-GCP knowledge. Action: - Complete a free GCP training (NIH or CITI). - Highlight coursework in research methods, pharmacology, or statistics on your resume. Entry-Level Roles (less then 2 years are expected) : - Clinical Trial Assistant (CTA) - Data Entry Coordinator - Regulatory Affairs Assistant 2. Internships & Volunteering: The Experience Shortcut No paid experience? Create your own. Action: - Look for research assistant opportunities at local universities. - Volunteer for clinical studies, even supporting data entry or patient follow-ups counts. I remember even looking for front desk options. 3. Get Certified (Even Without a Job) SOCRA, ACRP and CITI certifications often require experience, but some programs don’t. Action: - Explore entry-level certifications like the Clinical Research New Coordinator (CRNC) program. 4. Network Smarter, Not Harder Clinical research is a relationship-driven industry. Action: - Follow CROs and pharma companies on LinkedIn. - Join clinical research groups and attend virtual conferences. - Connect with professionals, ask smart questions, not just for jobs. Clinical research needs fresh talent, people ready to embrace decentralized trials, patient-centric solutions, and digital transformations. Your degree is just the beginning. The opportunities? Endless, if you know where to look. For my experienced CRAs how did you get your first CRA job?

A drug discovered entirely by AI just completed human clinical trials. And it took only 3 years It's called Rentosertib - the first drug where both the target and the molecule were designed by AI, not just optimized. Insilico Medicine used generative AI to find a treatment for idiopathic pulmonary fibrosis (IPF) - a disease where scar tissue builds up in your lungs until you can't breathe. Phase 2 results just came back. And it works. Here's what happened: ▶ AI cut discovery time by 80% Insilico's AI platform analyzed datasets and identified a protein called TNIK that drives lung scarring. Then their chemistry engine designed a molecule to block it. 18 months from finding the target to having a drug candidate. Traditional drug discovery takes 10-15 years. ▶ Patients improved in Phase 2 trials IPF patients taking the drug daily saw their lung capacity improve. The placebo group got worse. Patients also reported less coughing and easier breathing - the things that actually matter to their daily lives. Safe and well-tolerated across all doses in two Phase 1 trials and one Phase 2 trial. This is crucial because IPF has no cure. And 5 million people worldwide have IPF. You get 3-4 years to live after diagnosis. Your lungs slowly fill with scar tissue. You can't get enough oxygen. Eventually you suffocate. Current treatments only slow it down. They can't stop the scarring or reverse it. Rentosertib is showing it can actually improve lung function - something no existing drug can do. This is the first generative AI drug to reach Phase 2 with positive results. AI found what to target. AI designed the molecule. Humans proved it works in clinical trials. What do you think of this? Will AI-discovered drugs become the standard or stay the exception? #entrepreneurship #healthtech #innovation