Ryan Kilpatrick

Today’s positive announcement about Gilead’s investigational PrEP medication among cisgender men, transgender men, transgender women, and gender non-binary individuals in our PURPOSE 2 trial is a remarkable achievement. I’m so proud that our commitment to inclusion was central to the success of the trial. Read the full press release here: http://bit.ly/3XlBEn7 Cisgender men, particularly those who identify as gay and are white, have been at the forefront of PrEP advocacy, inclusion in research, and PrEP uptake and persistence globally. But due to socio-structural factors like stigma, discrimination, health equity and access, PrEP uptake and clinical trial participation have lagged among cisgender men of color, transgender men, transgender women and gender non-binary individuals. Those most disproportionately affected by HIV are also the most underrepresented in clinical trials. We worked to address these kinds of disparities in multiple ways in PURPOSE 2, including:    ·      Establishing a trial-specific global community advisory group ·      Setting nuanced site-specific diversity goals based on local epidemiology ·      Selecting sites with expertise in delivering gender-affirming care and those with experience and trust in engaging with communities of color, underserved and vulnerable populations ·      Continually engaging with community advocates These and other efforts resulted in more diverse trial participation, including first-time PrEP users from historically underrepresented communities and, for the first time in a Phase 3 PrEP trial, intentional inclusion of transgender men and gender non-binary individuals . By the time we completed enrollment, PURPOSE 2 included more than 3,200 people across 88 sites in Argentina, Brazil, Mexico, Peru, South Africa, Thailand and the United States.   To help reach every person who needs or wants PrEP, across all populations, we know that we need new kinds of PrEP choices that could help increase uptake and persistence among people who have not benefited as much from existing PrEP options, helping to potentially reduce disparities in HIV transmissions globally.   Milestones like this remind me of Gilead’s commitment to helping to end the HIV epidemic for everyone, everywhere. Today, we celebrate PURPOSE 2 and how – through its exceptional level of inclusion and geographical representation, coupled with the innovation in the science, both in the investigational medicine and in the novel clinical trial design – we’re making that ambition a reality.   A huge thank you to our clinical team for designing and recruiting for such an innovative, intentional and inclusive trial, and to the community members and other key partners around the world who worked alongside us at all stages. We look forward to sharing more about the data at an upcoming scientific meeting. Charles Peterson Martez D. R. Smith Wakefield Jenna Rapues #PutPeopleFirst #GileadEmployee #PreventionWithPurpose For U.S. Audiences Only

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GLP-1 receptor agonists, a popular class of drugs including Novo Nordisk's #Ozempic (semaglutide) and #Victoza (liraglutide), do not seem to increase the risk of #thyroidcancer, according to a study published in The #BMJ. The Scandinavian study conducted by researchers at #KarolinskaInstitutet analyzed data from national registries in #Denmark, #Norway, and #Sweden. It compared around 145,000 patients treated with GLP-1s to over 290,000 patients using other diabetes treatments. The study found no significant increase in thyroid cancer risk among patients using GLP-1s over a follow-up period of nearly four years. The research offers strong support that GLP-1 analogs are not linked to an increased risk of thyroid cancer. However, the researchers caution that some subtypes of thyroid cancer may carry risks for smaller, unstudied patient groups. GLP-1 receptor agonists are a leading class of diabetes and obesity medications, primarily represented by Novo's #semaglutide and Lilly's #tirzepatide. These companies are expected to control around 80% of the multibillion-dollar market by 2030. GLP-1s have been flagged for potential safety issues, including associations with suicidal ideation and self-harm, which both the #EuropeanMedicinesAgency and the #FDA are investigating. However, neither regulator has found conclusive evidence to support the link. #MedicalResearch #Pharmaceuticals #Diabetes #Obesity #GLP1 Novo Nordisk Eli Lilly and Company

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How can sponsors prioritize diversity and inclusion of underrepresented populations in clinical trials and adhere to new guidelines from the U.S. Food and Drug Administration? Learn about strategies and recommendations for success in our latest publication: https://lnkd.in/egiDZtEg #ClinicalTrialInclusion #ClinicalTrialDiversity #ClinicalTrials

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The most interesting thing this week: You should not feel guilty anymore if your guilty pleasure is dark chocolate! 🎉 Increased consumption of dark chocolate was associated with a lower risk of T2D, according to three large prospective cohorts studies. Finally! 🎉 The power of Data-Driven but Human-Centred Evidence in action 😉 https://lnkd.in/gJdeqcpf #realworlddata #realworldevidence #rwe #rwd #data #healthcare #biopharma #medicalaffairs #patientcentricity #patientcenteredcare #medicalcommunication #themostinterestingthingthisweek

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My friends in biopharma are increasingly worried about the headwinds to the industry’s traditional business model. The impact is real - constant organizational transformation and layoffs. We predicted the challenge in our book - Redefining Innovation, and suggested pathways to new business models. However, we also understand the inertia and the inability to change. Overall, we foresee the industry becoming more like the utilities- regulated and price capped. As individuals passionate about the industry, it is time to rethink how we will disrupt and innovate ourselves, for our own viability. That in turn might be the impetus for large scale organizational transformation the industry needs now.

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Don't Stop Believin': Mastering CTAs and Budgets for Clinical Trial Success About ten years ago, we set an ambitious goal: to establish Master CTAs with at least half of the sponsors PCRS Network work with. We met that goal! Well, sort of. Having worked with all the major Pharma companies and many smaller ones, we knew that therapeutic areas within larger companies operate in silos. This presents significant challenges when trying to standardize legal or budgeting processes. Even when a master CTA was approved at the corporate level, legal teams in each silo often wanted to negotiate their own versions. Fortunately, in every instance, we were able to facilitate communication between these teams and resolve the issues efficiently. At the same time we set the goal for the master CTA, we also set a more conservative target for master budgets. The concept of a master budget is straightforward: pre-negotiate standard items to minimize the number of negotiable items study-to-study. As we anticipated, sponsors were generally receptive to this approach. Moving forward, our long-standing relationships with sponsors facilitated smooth negotiations on future studies, often with just a brief conversation or email exchange. Although we continue to experience some delays because of the silos, we remain committed to implementing master CTAs and budgets wherever possible. We've learned not to be discouraged by occasional refusals. Our experience shows that having master agreements and budgets, or just standardized language for key clauses, even if they need periodic revisions, saves both sponsors and sites valuable time. Encouraging the industry to adopt master agreements as a standard practice can significantly improve the study startup timeline, contributing to saving and improving lives. Supporting Insights A 2021 article from Applied Clinical Trials highlights the importance of master CTAs, stating, "Master CTAs streamline the contracting process, reducing administrative burden and accelerating study startup times." Another study from Clinical Trials Arena in 2022 found that "adopting master agreements can lead to a 30% reduction in study startup timelines, enhancing overall efficiency." Call to Action Let's work together to streamline clinical trials and bring treatments to patients faster. If you’re interested in learning more about how master CTAs and budgets can benefit your organization, reach out today. Together, we can rise to new heights of efficiency and success in clinical trials. Citations 1. "Streamlining Clinical Trials with Master CTAs," Applied Clinical Trials, 2021. 2. "Efficiency Gains in Study Startup with Master Agreements," Clinical Trials Arena, 2022. 3. Lyric reference from "Don't Stop Believin'" by Journey, 1981.

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How much do we get for our public investment? An extract from my Monday presentation to the Pharma Forum. We often consider public reimbursement in pharmaceuticals as an isolated item. In reality, governments reimburse many types of goods - health, transportation, education, agriculture, etc. The question arises: Can one quantify and standardize the return on investment (ROI) for every dollar spent? So that is what we did in our research. Not all data is available, but as we can see, and contrary to populists' arguments, public RoI for purchasing pharmaceutical products is similar to other areas. In the case of pharmaceuticals, we have "reversed engineered" costs per QALY. Where do things get tricky? In funding medicines that are considered ultra "premium". We can see that the RoI in these cases is lower than in other instances. #RoI #Publicreimbursement #Pharmaceuticals #Publicfunding #Health #Cures

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✍️ #WarningLetter to a clinical investigator. #DataIntegrity is not named but if you read through, you see that the observations speak about contemporaneous, accurate and complete as well as original. Always keep #ALCOA+ in mind and be precise on what, how, when and where it gets documented, processed, altered, reviewed and archived.

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Some learnings from a 7-year deep dive into Medical Affairs: I have worked in Medical Affairs since 2002, but 7 years ago was when I really dove deep into the opportunities and hurdles of Medical Affairs. You may think this is a strange statement, but I have found that working IN it is very different from working WITH it. During my years in pharma, I was very often running from deadline to deadline, crisis to crisis and project to project. Very rarely could I find the time to sit back and think about my team's role, our activities and how to do them best. I was just very happy to get things done, keep my team aligned and learn a little in the process. But everything changed when I quit my job 7 years ago, and I started looking at Medical Affairs from the outside. I now had the time to read, to listen and to discuss, and I started seeing the patterns, the joys, the pains and the gaps. This understanding was really accelerated when I trained and worked with more and more Medical Affairs teams; learning through our interactions and discussions. Curious what I have learned? Here are some of them; ·       Medical affairs is a department that is still looking for its rightful place in the company. My guess is that its place is somewhere between where McKinsey sees it and where most people are right now. What I do know is that we are still on the move and have not yet arrived. Not sure we always know which direction to go or what tools to use to get there. ·       Medical Affairs is essential to the success of the company, especially in the changing healthcare environment of cynicism, mistrust and decrease in time and resources. ·       The need and complexity of demonstrating true value is a recurrent theme which often leads to teams feeling undervalued and misunderstood ·       Despite the crucial contribution of the Medical Affairs teams, training in how to optimize their work is not always available and often new hires are left to their own devices on how to fulfill their role ·       Cross functional collaboration is not as easy as it sounds And the best one of them all: ·       Even though daily practice can be hard, it is the best job in the world   What other things do we need to understand about Medical Affairs? Let us know in the comments!! PS to everyone who is working hard in the trenches: I really recommend carving out some time in your working week to think, read and write about your department and role. There is so much information out there and being able to see the big picture will help you tremendously in your work. Also, we need all bright minds out there to share their experiences and opinions to bring on the changes we want. #medicalaffairs #msl #pharma

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Important presentation from Andreas Vychytil - when designing clinical trials for new PD solutions it is vital to use outcomes linked to SONG-PD priority areas

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⚔ Battle of the anti-#FcRNs in myasthenia gravis... 📑 Johnson & Johnson touts its recent nipocalimab PIII data showcased at the #EAN: European Academy of Neurology Conference, possibly competitively positioned well vs. argenx's #Vyvgart. ⏩ Forward looking: 👥 To leverage efficacy as a differentiator; across a range of antibody-positive gMG patients (i.e., MuSK+, anti-LRP4+, and anti-AChR+) 🗓 Regulatory filing est. by EOY #gMG #GeneralizedMyasteniaGravis #Clinical #MedicalAffairs #Nipocalimab #ActivitiesOfDailyLiving #Autoimmune #Neurodegenerative #Pharma #Healthcare

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💥 Leveraging Real-World Evidence (RWE) in MSL Activities 💥 Check out this weeks MSL Talk Podcast with Elizabeth Pash, PhD, MS, RDN, Medical Affairs Executive Leader who shares insights on how to leverage real-world evidence (RWE) in MSL activities. This episode is sponsored by MSL Mastery, helping Medical Science Liaisons and aspiring MSLs to excel in their careers and lives through courses, coaching, and community. For more details, please visit MSLmastery (dot) com. In this episode, Beth discusses… 👉 What is Real World Evidence and how it is different from Clinical Trial Data 👉 How MSLs can effectively incorporate RWE into their discussions with healthcare professionals and key opinion leaders (KOLs) 👉 Challenges MSLs typically face when presenting RWE to stakeholders, and how can they overcome them 👉 Ways RWE can be used to enhance the strategic value of MSL activities 👉 Key skills MSLs need to develop in order to effectively understand and communicate RWE to diverse audiences The MSL Talk Podcast is available on all podcast platforms and the MSL Talk YouTube Channel.     MSL Talk is a podcast that features helpful information for current Medical Science Liaisons, Medical Affairs leaders, job seekers looking to break into the pharmaceutical industry in their first MSL role as well as anyone interested in hearing good industry conversations and medical affairs discussions.   #msl #medicalaffairs #medicalscienceliaison #realworldevidence #careers    

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💥 Leveraging Real-World Evidence (RWE) in MSL Activities 💥 Check out this weeks MSL Talk Podcast with Elizabeth Pash, PhD, MS, RDN, Medical Affairs Executive Leader who shares insights on how to leverage real-world evidence (RWE) in MSL activities. This episode is sponsored by MSL Mastery, helping Medical Science Liaisons and aspiring MSLs to excel in their careers and lives through courses, coaching, and community. For more details, please visit MSLmastery (dot) com. In this episode, Beth discusses… 👉 What is Real World Evidence and how it is different from Clinical Trial Data 👉 How MSLs can effectively incorporate RWE into their discussions with healthcare professionals and key opinion leaders (KOLs) 👉 Challenges MSLs typically face when presenting RWE to stakeholders, and how can they overcome them 👉 Ways RWE can be used to enhance the strategic value of MSL activities 👉 Key skills MSLs need to develop in order to effectively understand and communicate RWE to diverse audiences The MSL Talk Podcast is available on all podcast platforms and the MSL Talk YouTube Channel. MSL Talk is a podcast that features helpful information for current Medical Science Liaisons, Medical Affairs leaders, job seekers looking to break into the pharmaceutical industry in their first MSL role as well as anyone interested in hearing good industry conversations and medical affairs discussions. #msl #medicalaffairs #medicalscienceliaison #realworldevidence #careers

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FDA‘s #platformdesignation program promises to be a true gamechanger for #raredisease #drugdevelopment. With more than 10,000 rare diseases, of which 95% have no treatments, developing diseases one at a time is simply not an option. #FDA’s draft guidance on the platform designation program was just released a few days ago and connected are due at the end of July. What a timely discussion at #BIO2024 BIO International Convention today! Some of the key take-aways for me: the goal is to streamline clinical ( and nin-clinical) development as well as #cmc, pre-and postmarket; come talk to #FDA early and often (think applying for the designation at the #IND stage); increase efficiency for #sponsors *and* #regulators; the designation is widely applicable, way beyond #cell and #genetherapies (spanning both #CBER and #CDER) although what qualifies as a platform may still evolve within the confines of the #FDA definition; the onus will be on #sponsors to justify why they qualify for the designation; there will be a need for much more thinking beyond the current guidance ( e.g., how clinical data or lifecycle management issues will be handled); and as the environment evolves ( think #artificialintelligence #ai) the guidance will have to evolve with it, but the guidance provides a very robust start; the guidance provides a framework for conversations between #FDA and #sponsors on how prior knowledge can be leveraged in drug development. One thing that was largely absent from the discussion today, but that will be critical for #raredisease #patients, will be how the Centers for Medicare & Medicaid Services and other public and private #payers will think about the issue, in particular if clinical data will be leveraged across multiple #productdevelopment programs. We’re already seeing an increasing number of #ncds and #ceds at #cms for products that have been #FDA approved through traditional or accelerated approval, making it increasingly burdensome for patients to access life-altering therapies. Hopefully payers will be supportive of platform approaches and recognize the benefits for patients and the broader #raredisease community, but their absence from the conversation today is certainly a concern for me …. certainly a lot more to do… Thank you Nicole Verdun, Dylan Bechtle, Snehal Naik, Rob Berlin, Anne-Virginie Eggimann, and Antonella Lozito-Heerschap, Pharm.D. for a really inspiring conversation!

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Even if a clinical trial doesn’t meet its initial objectives (as is the case with most trials in PSP and other rare Neurogenerative diseases), the data and the learned processes are invaluable to the field. By taking the extra time to publish their insights, organizations like Biogen, in collaboration with the University of California, San Francisco, and Rutgers University, ensure important learnings and outcomes are shared with the broader community. For those in my network who don't know about PSP, it is a neurodegenerative disorder with an estimated prevalence of about 6 cases per 100,000 people. It typically begins in one’s early 60s and can lead to death within 7 years of symptom onset. Characterized by impairments in gait, eye movement, swallowing, speech, sleep, cognition, and behavior, PSP correlates with the distribution and density of 4R tau pathology in neurons and glia. Tragically, there is still no treatment available to slow the progression of this fatal disease. With tau-targeting therapies now in development, the field depends on robust clinician- and patient-reported measures—known as clinical outcome assessments (COAs)—to track meaningful changes in disease progression. FDA guidance on patient-focused drug development emphasizes the necessity of COAs that capture what truly matters to those living with PSP. A newly published paper in Nature Communications, co-authored by Lawrence Golbe, a CurePSP board member, among other leading scientists, describes a 15-item update to the PSP Rating Scale (PSPRS). This refined tool improves clinical relevance and sensitivity to disease progression, aligns with FDA patient-focused drug development principles, and offers researchers a measures of therapeutic impact in PSP. #PSP #NeurodegenerativeDiseases #ClinicalTrials #Biogen #UCSF #Rutgers #CurePSP #Innovation #PatientFocus https://lnkd.in/ernBmqWN

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Of the approximately 4,500  FDA-approved drugs, the vast majority are generic, over 80%. Once a drug goes generic, its price drops as multiple manufacturers begin producing it,  resulting in little financial incentive for further research. Despite their affordability, these generic drugs possess enormous potential, yet our system often overlooks them because they are no longer considered profitable. At Every Cure, we believe this gap in research and innovation is unacceptable. We are dedicated to unlocking the potential of these generic drugs to treat diseases that currently lack effective therapies. #pharma #raredisease #drugdevelopment #drugdiscovery

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