Mikael Dolsten’s Post

Dr. Jorge Estrella, M.D., CMO/President, alongside Dr. Charles Schmidt, M.D., Head of the Scientific Clinical Medical Advisory Board at ESTERN Medical CRO Life Sciences, recently convened at the prestigious World Orphan Drug Congress USA 2024 in Boston, Massachusetts. This key event in the international calendar brought together major stakeholders in the field of Life Sciences to focus on rare diseases and innovative treatments.   The Congress served as a critical platform for dialogue among pharmaceutical leaders, research bodies, healthcare providers, patient advocacy groups, and regulatory agencies. Such interactions are pivotal to advancing clinical trials and research and development efforts.   This year’s conference successfully bridged gaps within the rare disease community, enhancing cooperative efforts that are essential for the progression of clinical trials and for improving patient access to crucial therapies. Key industry players, including leading pharmaceutical and biotech firms, regulatory bodies, and investors, engaged extensively to propel forward the development of orphan drugs.   As the need for clinical trial development surges, smaller and mid-sized biotech and pharmaceutical companies in the USA and Europe are encountering notable challenges due to limited operational capacities at all stages of their INDs and NDAs—from Phase I, II, III through IV.   At ESTERN Medical, we are poised to mitigate these challenges. We provide comprehensive, customized solutions and in-depth expertise to support your R&D across the entire lifecycle of product development. Our services are tailored to address the specific needs of your projects in North America and the burgeoning markets of Latin America and South America.   Together, let’s champion innovation in Life Sciences, transform the landscape of clinical trials and R&D, and ultimately revolutionize global healthcare. Join us in shaping a future where advanced therapies become accessible to those in dire need. #biotech #biotechs #boston #california #canada #clinicaldevelopment #clinicalregulatory #clinicalresearch #clinicalresearchorganization #clinicalresearchtrials #clinicalstudies #clinicaltrials #cro #cros #crolatinamerica #croservices #drugdevelopment #emergingmarkets #estenmedical #europe #france #genetown #germany #globalhealthcare #healthcare #healthcareIndustry #healthcareinnovation #internationalcro #japan #latinamerica #latam #latamcro #lifesciences #massachusetts #medicaldevices #ESTERNMedicalCRO #medicalresearch #medicalsciences #medicine #medtechs #netherlands #newengland #newjersey #northcarolina #northerncalifornia #pharmaceuticalindustry #pharmaceuticals #regulatorycompliance #researchanddevelopment #rtp #sanfrancisco #sandiegoca #scientificresearch #southamerica #southerncalifornia #spain #sweden #switzerland #texas #uk #usa #inds #ndas #WorldOrphanDrugCongressUSA #rarediseases #innovation #DrCharlesSchmidtMD #DrJorgeEstrellaMD #WorldOrphanUSA #RnD

Dr. Jorge Estrella, M.D., CMO/President, alongside Dr. Charles Schmidt, M.D., Head of the Scientific Clinical Medical Advisory Board at ESTERN Medical CRO Life Sciences, recently convened at the prestigious World Orphan Drug Congress USA 2024 in Boston, Massachusetts. This key event in the international calendar brought together major stakeholders in the field of Life Sciences to focus on rare diseases and innovative treatments.   The Congress served as a critical platform for dialogue among pharmaceutical leaders, research bodies, healthcare providers, patient advocacy groups, and regulatory agencies. Such interactions are pivotal to advancing clinical trials and research and development efforts.   This year’s conference successfully bridged gaps within the rare disease community, enhancing cooperative efforts that are essential for the progression of clinical trials and for improving patient access to crucial therapies. Key industry players, including leading pharmaceutical and biotech firms, regulatory bodies, and investors, engaged extensively to propel forward the development of orphan drugs.   As the need for clinical trial development surges, smaller and mid-sized biotech and pharmaceutical companies in the USA and Europe are encountering notable challenges due to limited operational capacities at all stages of their INDs and NDAs—from Phase I, II, III through IV.   At ESTERN Medical, we are poised to mitigate these challenges. We provide comprehensive, customized solutions and in-depth expertise to support your R&D across the entire lifecycle of product development. Our services are tailored to address the specific needs of your projects in North America and the burgeoning markets of Latin America and South America.   Together, let’s champion innovation in Life Sciences, transform the landscape of clinical trials and R&D, and ultimately revolutionize global healthcare. Join us in shaping a future where advanced therapies become accessible to those in dire need. #biotech #biotechs #boston #california #canada #clinicaldevelopment #clinicalregulatory #clinicalresearch #clinicalresearchorganization #clinicalresearchtrials #clinicalstudies #clinicaltrials #cro #cros #crolatinamerica #croservices #drugdevelopment #emergingmarkets #estenmedical #esterncro #europe #france #genetown #germany #globalhealthcare #healthcare #healthcareIndustry #healthcareinnovation #internationalcro #japan #latinamerica #latam #latamcro #lifesciences #lifesciencesinnovation #massachusetts #medicaldevices #medicalresearch #medicalsciences #medicine #medtechs #netherlands #newengland #newjersey #northcarolina #northerncalifornia #pharmaceuticalindustry #pharmaceuticals #pharmatechsolutions #regulatorycompliance #researchanddevelopment #rtp #sanfrancisco #sandiegoca #scientificresearch #southamerica #southerncalifornia #spain #sweden #switzerland #texas #uk #usa #WorldOrphanDrugCongressUSA #rarediseases #innovation  #inds #ndas #DrCharlesSchmidtMD #DrJorgeEstrellaMD

Stories making headlines today in the biopharma industry: Johnson & Johnson has announced that the final pricing set under the Inflation Reduction Act (IRA) will not hinder its long-term sales projections. Jennifer Taubert, Worldwide Chairman of Innovative Medicine, confirmed that the company had already factored these figures into last year's financial guidance. This positions J&J as yet another pharmaceutical leader demonstrating resilience through strategic investment and pipeline management. The FDA has launched a Rare Disease Innovation Hub, intended to unify policies and develop new evaluation methods for rare disease treatments. Led by CBER Director Peter Marks and CDER Director Patrizia Cavazzoni, the hub aims to streamline regulatory pathways and provide consistent guidance to companies working on rare disease therapies. Merck has asked the US District Court for the District of Columbia to expedite its decision regarding its challenge to the Inflation Reduction Act. The company is seeking a swift resolution to allow sufficient time for appeals before the IRA's full enactment in 2026. This follows a series of government victories in similar cases against other major pharmaceutical companies such as Bristol Myers Squibb and AstraZeneca. Executives from Cigna, CVS Health, and UnitedHealth Group are set to testify before Congress on July 23. Organized by the House Committee on Oversight and Accountability, the hearing follows a critical Federal Trade Commission report and will focus on how Pharmacy Benefit Managers (PBMs) interact with drugmakers and pharmacies. These three companies collectively hold 79% of the PBM market. London-based hVIVO is actively seeking acquisitions of small contract research organizations (CROs) to meet its £100 million revenue target by 2028. CEO Yamin Khan indicated that the company is particularly interested in UK-based CROs with Phase 1 trial facilities. hVIVO specializes in human challenge trials to assess vaccine efficacy and safety. Gilead Sciences has announced that Chief Medical Officer Merdad Parsey will depart by the first quarter of 2025. Parsey's departure comes amid challenges in Gilead's pipeline and cancer drug development. The news has prompted a slight rise in Gilead's stock, which has been down 10% since the beginning of the year. Analysts suggest this transition could be an opportunity to rejuvenate Gilead's R&D efforts. #pharma #biotech #topstories #biodatastudio

Did you know global refrigerated storage surged by 32% from 2014-2020 (accelerated by a 17% increase during the 2020 COVID-19 pandemic)? Innovative new biological therapeutics (biologics) including cell and gene therapies (CGT) are driving temperature-controlled and cold-chain logistics growth, along with the need for global distribution of mRNA-based vaccines. Yourway's Dublin depot, along with our global operations, orchestrates a fully-integrated symphony. From controlled-room temperature to -185°C cryogenics, our Dublin depot is the backbone of a complete clinical trial value-chain. Storage, transportation, clinical packaging—it's all seamlessly coordinated, ensuring advanced therapies reach their destination at the right time. Read our new blog to learn how we can support your global clinical trials: https://hubs.ly/Q028RlJ40 #ClinicalTrials #Pharmaceuticals #Biologics #ColdChain #TemperatureControl #BiopharmaInnovation #SupplyChain #AlwaysYourway

Fierce Pharma Clinical Summit - great week! Very high energy from the speakers, attendees, and industry friends. What made this year's conference special is the quality and diversity of the topics and speakers. I attend these conference with an eye toward how ScienceMedia, Inc. can continue serving our sponsor clients in new ways and understand the challenges and opportunities for pharma in the clinical realm. Lots of opportunities to learn and collaborate! Check out these highlights: 🔹 Michael Roseblatt from Roche late-stage ClinOps kicked us off with his global perspective on the state of clinical trials and the exciting innovation ahead. 🔹 Andrew Lee from Merck pulled out his crystal ball to predict future trends in clinical trials. He emphasized adaptability, technology and progress! 🔹 Gerri Gallagher from GSK moderated a panel on building stronger site engagement, with insights from Jimmy Bechtel (SCRS) and Leslie Shupenko from Jazz Pharmaceuticals. They discussed the importance of collaboration and shared goals among sponsors, sites, and CROs. 🔹 Janelle Sabo from Eli Lilly and Company led a panel on community-based research approaches, with contributions from Adam Wyckoff (H1), LaShell Robinson from Takeda Alftan Dyson (ViiV Healthcare), and Nisha Pandya Patel (GSK). They explored strategies for increasing representation in clinical trials. 🔹 Reenie McCarthy from Stealth BioTherapeutics shared her thoughts on closing the health equity gap in ultra-rare diseases, highlighting the challenges and opportunities in clinical development. 🔹 Leonard Sacks from the FDA spoke about the progress and challenges in decentralized clinical trials, emphasizing the need for continued innovation. 🔹 Justin Ott from Regeneron moderated a panel on risk-based approaches for inspection readiness, featuring Marna Grim from Chinook Therapeutics, Inc., Lorne Cheeseman (Corcept Therapeutics), Stephanie Haley from Astellas Pharma US, and Mary MacDonald (Takeda). 🔹 Jen Yip from Novartis led a panel on change management for decentralized clinical trials, with insights from Tom Julian from Gilead Sciences Mary Slomkowski (Otsuka), Bradley P. Hightower (Hightower Clinical Trial Services), and Shafaat Ali Khan (Eli Lilly). A big thank you to all the speakers for sharing their expertise! Looking forward to future collaborations. #ClinicalTrials #Innovation #fierceclinicalsummit

Honored to have our 2018 PNAS paper on The National Institutes of Health funding for research contributing to new drug approvals quoted by Jocelyn Ulrich, MPH, vice president of policy and research at PhRMA to the Senate Judiciary Committee May 21, 2024. We are disappointed that she did not reference our more recent 2023 JAMA Health Forum paper, which identified $187 billion NIH spending for basic or applied research directly related to drugs approved from 2010-2019, demonstrating that the average investment cost to the NIH for each approved drug was not less than reported investment cost to industry. https://lnkd.in/esX4fP7r. We agree with her testimony stating that NIH funding complements industry's funding, with the NIH making greater contributions to early stages of basic science and discovery rather than later stages of applied research and development. Her arguments do not diminish NIH's role as the first investor in #pharmaceutical #innovation, performing research that both enables and de-risks subsequent investments by industry. Center for Integration of Science and Industry at Bentley University, Bentley University Center for Health and Business, Bentley University, Institute for New Economic Thinking,

On reading a book chapter on "How to Systematically Assess Biotech Clinical Trials". Whether you're involved in early drug discovery or clinical trials, data about past, present and future clinical trials is important. Lately I've been coursing through ClinicalTrials dot gov to gather insights on participant recruitment and relevant routine health data that support them, I wanted to step back and get a different perspective on how the trials are evaluated by various parties. I really like Magellan's book as it seems to often approach this topic more from a business perspective. It opens one section by asking "What is the intent of the study?" and then "Is the study designed to achieve its goals?". I've been focusining on this second question and going through sections about population, study design and endpoints. "How were patients identified, included and excluded and why?" - this is really important for imagining the trial candidates who would engage in recruitment. "How well validated are tests or biomarkers that were used to define the patient population?" - this is really important for today's precision medicine activities that try to characterise disease patient sub-populations. "What were the endpoints?" (primary and secondary). The ability to measure the end points and referencing them to established end points used in other drugs can be important. "What effect size in the primary endpoint was the trial designed to detect, and why was it chosen?" This can influence how many people you'll need in a trial. If you're measuring an endpoint that has high variability or a small change in an endpoint, you'll need more people enrolled. There are some really interesting insights in the book, like discussion about how the study population can influence what drug comparators you're using, the influences of the trial phase on end points So whether you're interested in large-scale, late phase clinical trials or you're interested in those first phase trials for new drug targets, I'd highly recommend having a look through the book. #drugdevelopment #clinicaltrial #design #precisionmedicine

Are you attending the World Orphan Drug Congress April 23 - 25th in Boston? If you haven't made a decision yet, here are 3 MORE reasons to attend: 1. Strategic Partnerships and Collaboration: Collaboration is essential for addressing the complex challenges associated with rare diseases and orphan drugs. WODC provides a conducive environment for fostering strategic partnerships and collaborations among industry stakeholders, academic institutions, patient organizations, and government agencies. By working together, attendees can leverage complementary expertise, resources, and networks to accelerate research, improve patient outcomes, and overcome barriers to drug development and access. 2. Opportunity to make a tangible difference in the lives of patients, families and caretakers living with a rare disease and contribute to shaping the future of orphan drug innovation and healthcare policy. 3. Gain access to cutting-edge insights, best practices, and the latest developments in drug discovery, regulatory pathways, patient engagement, market access, and commercialization strategies. These insights can inform decision-making, inspire innovation, and shape future initiatives within the industry. And if you need another reason, I'll be there and I'd love to connect with you! If you would LIKE to attend and work at a biopharma/pharma company - you can get a free pass! Just leave me a comment and tag me! hashtag#raredisease hashtag#rarediseases hashtag#rareaccessactionproject hashtag#WODC hashtag#smallbiopharma

Are you attending the World Orphan Drug Congress April 23 - 25th in Boston? If you haven't made a decision yet, here are 3 MORE reasons to attend: 1. Strategic Partnerships and Collaboration: Collaboration is essential for addressing the complex challenges associated with rare diseases and orphan drugs. WODC provides a conducive environment for fostering strategic partnerships and collaborations among industry stakeholders, academic institutions, patient organizations, and government agencies. By working together, attendees can leverage complementary expertise, resources, and networks to accelerate research, improve patient outcomes, and overcome barriers to drug development and access. 2. Opportunity to make a tangible difference in the lives of patients, families and caretakers living with a rare disease and contribute to shaping the future of orphan drug innovation and healthcare policy. 3. Gain access to cutting-edge insights, best practices, and the latest developments in drug discovery, regulatory pathways, patient engagement, market access, and commercialization strategies. These insights can inform decision-making, inspire innovation, and shape future initiatives within the industry. And if you need another reason, I'll be there and I'd love to connect with you! If you would LIKE to attend and work at a biopharma/pharma company - you can get a free pass! Just leave me a comment and tag me! hashtag#raredisease hashtag#rarediseases hashtag#rareaccessactionproject hashtag#WODC hashtag#smallbiopharma

It's been a big year so far for M&A in the life sciences market, and the general attitudes and feeling in the industry are a far cry from last years layoffs and liquidations. There is still a way to go in terms of recovery, and we are still far away from the highs of the COVID market, but it's getting better! Below, BioSpace have compiled some of the biggest M&As of the year so far, all worth over a billion dollars. Having 7 multi billion dollar deals in Q1 is a sure step in the right direction for the market and will hopefully lead to some great new treatments hitting the market soon! AbbVie were the biggest spenders, with $10.1 billion spent on ImmunoGen, Inc., while Bristol Myers Squibb came both second and third in deal size, spending a combined $8.9 billion on Mirati Therapeutics and RayzeBio. Other big spenders in the included AstraZeneca, Johnson & Johnson and Novartis, all spending over a billion. This investment shows a change in opinions in the market, with companies taking bigger risks and investing more in new products, signaling a change of attitudes and hopefully a change in the trends of the recruitment market soon! Follow Warman O'Brien for all the latest in the life sciences sector. #lifesciences #pharmaceuticals #biotech #investment