Jim Hoffman

How do we minimize the number of protocol amendments and increase the operational feasibility of our clinical trials? This article below points out the benefits of using data, conversations and real world data to enhance the feasibility of the protocol design and inclusion/exclusion criteria: 1) Are we being too strict on our criteria in order to "control" for data variability? 2) Do are criteria allow for the intended patient population in the real world (if not, they need to) 3) How realistic is your design in how the study sites actually work? How they see and treat patients? How is your company ensuring the design meets these requirements? https://lnkd.in/gzJzFd-3

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ChatGPT Social Media Post: The Rare Disease Innovation Hub 🌟 The Rare Disease Innovation Hub: Transforming Rare Disease Treatment 🌟 Recent rapid advances in identifying promising drug targets and developing gene therapies offer momentum and potential to meet the needs of patients with rare diseases. In 2023, over half of all the novel drugs and biologics approved by the FDA’s Center for Drug Evaluation and Research (CDER) and the FDA’s Center for Biologics Evaluation and Research (CBER) were to prevent, diagnose, or treat a rare disease or condition. Cost Effectiveness & Risk Management Investing in the development of treatments for rare diseases not only saves lives but also proves to be cost-effective in the long run by reducing the burden of untreated or poorly managed conditions. Effective risk management practices ensure patient safety and regulatory compliance, further solidifying the importance of initiatives like the Rare Disease Innovation Hub. Environmental Sustainability By focusing on sustainable practices in drug development and manufacturing, the Rare Disease Innovation Hub aims to minimize the environmental impact. This includes using eco-friendly materials, reducing waste, and ensuring responsible sourcing of ingredients. Interactive Questions & Poll Q1: How can the Rare Disease Innovation Hub improve the development of treatments for rare diseases? A1: By fostering collaboration across agencies and leveraging expert insights to streamline drug development processes. Q2: What innovative approaches do you think will be most effective? A2: Incorporating real-world evidence and novel trial designs to accelerate approval timelines and enhance patient outcomes. Poll: Which aspect of the Rare Disease Innovation Hub do you find most promising? Single Point of Connection Intercenter Collaboration Advancing Regulatory Science Environmental Sustainability Call to Action Join the Conversation! Share your thoughts on the importance of the Rare Disease Innovation Hub. Have you or someone you know been affected by a rare disease? What role do you think pharmacists can play in this innovative initiative? Comment below and let's discuss! Hashtags #RareDiseaseInnovation #FDA #DrugDevelopment #GeneTherapy #PatientAdvocacy #HealthcareInnovation #RareDiseases #Biologics #ClinicalResearch #CostEffectiveness #RiskManagement #Sustainability #PharmacyHeroes References and Citations United States Food and Drug Administration. (2023). FDA Drug and Biologic Approvals. FDA Cavazzoni, P., & Marks, P. (2023). Statement on the Rare Disease Innovation Hub. FDA. Engage with Us What role do you think collaboration and cross-agency expertise play in advancing treatments for rare diseases? Share your thoughts in the comments!

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#inflactionreductionact, #bigpharma, #Financial Times Joe Biden hails $7.5bn savings after striking Big Pharma drug price deal Executive Summary: Biden Administration's Drug Price Deal Joaquim Cardoso MSc The Biden administration has celebrated a significant achievement in healthcare policy by negotiating price caps on ten high-cost medications with leading pharmaceutical companies, including Johnson & Johnson and Merck. This initiative, part of the 2022 Inflation Reduction Act, aims to reduce drug costs for Americans, projecting savings of $7.5 billion in its inaugural year. Key points of the deal include: Savings Impact: The program is expected to lower Medicare spending by 22%, equating to $6 billion, and reduce out-of-pocket expenses by $1.5 billion for patients. Legislative Context: Enacted under the 2022 Inflation Reduction Act, championed by Vice President Kamala Harris, this initiative underscores Biden's commitment to healthcare reform ahead of the upcoming elections. Pharmaceutical Response: Industry reactions have been mixed, with concerns raised over potential impacts on research funding and access to medications, despite negotiated discounts of up to 79% off list prices. Looking forward, the administration plans to expand negotiations to include up to 50 more drugs, aiming for $100 billion in savings over the next decade. However, legal challenges from pharmaceutical companies and industry skepticism regarding patient benefits remain ongoing concerns. This policy marks a pivotal moment in healthcare pricing regulation, shaping the debate on pharmaceutical affordability and access in the United States.

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Genes & GLPs - Nature Versus Pharma With GLP-1 RA (glucagon-like peptide-1 receptor agonist) medications making headlines as 'game-changing' for weight loss. And with trials taking place for cardiovascular disease and neurodegenerative conditions hope is growing and hype is building. But GLPs are a natural part of our biology. So do we really need drugs to mimic them. This webinar will explore mechanisms, and gene-nutrient interactions to support GLP function naturally, and without side effects. Live from 7pm, Monday 29th April. Recording available to pre registrants. Register Free at: https://lnkd.in/dj5WMMRK #weightloss #wegovy #GLP #semaglutide #nutrigenomics #metabolichealth #healthspan

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No mention was made of the “underwater #biosimilar” issue that is occurring secondary to over aggressive rebating by some bio similar manufacturers trying to gain #failfirst position. Unfortunately, all it has done is drop the Asp so low that not even ASP +8 comes anywhere near reimbursing providers for acquisition cost. It has become an acute problem because patients who were stable on either the now underwater biosimilar or the reference product or a different biosimilar can no longer obtain that medication because the “fail first” drug is unaffordable. Patients have lost all access to the drug, reference or biosimilar. The greed that incentivized the over-rebating, to gain fail first position, has only succeeded in no one being able to use their medication. this problem is being felt the most in the G.I. and Rheumatology spaces. Coalition of State Rheumatology Organizations (CSRO) arranged a meeting with #MedPAC and now #CMS along with a coalition of organizations, to make them aware of this problem. Unfortunately this may also end up being a problem with the #IRA negotiated part B drugs, as well as any provider administered drugs drugs that are picked by the #PDABs. As Adam Fein referred to- just more #UnintendedConsequences

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Nice short article on medtech ups and downs. Don’t need a subscription on this one! Reduced biopharma funding could slow medical device growth—50% of revenue in life sciences comes from biopharma. Physician visits suggest fewer surgeries—impacting demand for surgical devices. FDA LDT rules could raise costs & limit competition in diagnostics. R&D spending & clinical trials remain strong, a high number of device approvals & active clinical trials — 756 as of May 2024 — point to a promising future. Automation & supply chain optimization can improve margins during lower sales periods. Data can optimize inventory management, logistics, and geographic footprint. How can MedTech companies adapt further? #FutureOfWork #MedTech #RandD

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Could aspirin be the Lp(a) preventative treatment we’ve been looking for? An *observational* study out of Emory suggests it could be, finding that high-Lp(a) aspirin-takers have far lower ASCVD mortality risks than people who don’t regularly take aspirin --- including people who have low Lp(a). PS - We're definitely covering this in the next Cardiac Wire. https://lnkd.in/grkuFgWN

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Biomarkers can impact colorectal cancer treatment options. By testing for biomarkers, your medical team can personalize your treatment based on the unique mutations in your tumor, choosing the most effective treatments and helping you avoid unnecessary treatment or side effects. These test results can also connect you with clinical trials testing targeted therapies.    Today, on World mCRC Day, we join our international advocacy partners to raise awareness of metastatic CRC (mCRC) and biomarkers, including the impact biomarkers have on mCRC treatment.     Please visit  https://lnkd.in/eGcbN9xb learn more. #worldmCRCDay #colorectalcancer #coloncancer #bowelcancer 

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Excel hidden tab(s)!!! Hidden data on obesity prospect wipe $12B off Amgen market cap By Nick Paul Taylor Nov 13, 2024 6:35am A hidden tab in an Excel spreadsheet published nine months ago has wiped $12 billion off Amgen’s market cap. The tab contains data on bone density changes in patients who received Amgen’s obesity prospect MariTide in a phase 1 trial. Amgen published data from the study in the journal Nature Metabolism in February. The paper makes no mention of bone density. Amgen also shared the source data in Excel spreadsheets. At first glance, none of the spreadsheets contain bone density data either. However, one of the files contains hidden tabs that can be revealed by right-clicking on one of the visible tabs. The tabs remained hidden until an analyst at Cantor Fitzgerald spotted them and published a note about the bone density data this week. Three of the hidden tabs contain femur, lumbar and hip DEXA bone density scan data. A fourth tab, called “DEXA cohort 9 four,” contains patient-level data for the top MariTide dose. Cantor analyst Olivia Brayer called the data a “big unknown” in a research note, according to CNBC, and suggested bone density is a potential risk of GIPR antagonists such as MariTide. Amgen’s share price fell 7% in the wake of Brayer’s note, wiping $12 billion off the biotech’s market cap.  https://lnkd.in/exuq_c3z

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If the world is relying on the US to get value based drug pricing right (as this article in STAT suggests), we are in trouble. It lays out an argument for how drug pricing should be framed from a societal perspective, instead of just a payer-payment perspective, since the impact of successfully treating diseases impacts to far more than the entity paying for the therapy. But. It misses that the value in value based pricing would be vastly improved if we took into account using diagnostic tools to increase response rates. Instead of accepting FDA approved drugs with response rates between 15-40% and struggling with how to 'value' those, what if we used diagnostic tools to focus prescription on the patients who benefit? The value of the drug is vastly different in patients who respond vs those who do not. Why do we lump them together? What if we paid more for drugs that had a higher response rate in a stratified population? Clinical benefit to the patient should be the center of value based pricing discussions. #diagnostics #precisionmedicine #valuebased https://lnkd.in/eW4mwp7w

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Most months, I try to answer a well-focused question. This month, however, I want to simply take a broad look at how #FDA conducts its postmarket surveillance study program under Section 522 of the federal Food, Drug, and Cosmetic Act. FDA published a new final guidance on this topic on October 7, 2022, and the agency also made corresponding updates to its database. That gave me the chance to study the data, however incomplete they are. More on that in the post.

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I just got around to watching Oppenheimer, and the quote, "The future is no longer what it was," rings true regarding the race to bring breakthrough therapies to market as AI continues revolutionizing drug discovery and development. As startups and pharma giants leverage AI to streamline clinical trials, improve patient targeting, and optimize the drug pipeline, we're entering a new era of precision and efficiency. But, to fully realize AI's potential and subsequent benefits, companies must prioritize transparency, patient safety, and ethical data usage by maintaining robust data privacy standards, ensuring AI outputs are interpretable, and committing to rigorous risk-benefit analyses. Organizations must prioritize a strategic AI implementation plan harmonizing platforms, people, and processes. When AI tools align with these best practices, they accelerate timelines and enhance patient outcomes across the board. As we continue to witness AI reshape life sciences, those who innovate responsibly and with foresight will shape a future where AI-driven drug development meets the highest standards, driving sustainable success for the industry and the patients it serves. https://lnkd.in/erKAM664

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🌟 EXPERT INSIGHTS: Christopher Bunick, MD, PhD, discusses what he calls a “next-generation TYK2 inhibitor,” #zasocitinib, highlighting its high selectivity and potency and spotlighting data from clinical trials shared at the European Society for Dermatological Research meeting. "I think that the goal of this poster and this presentation at ESDR was to really show the dermatology community that there is so much more that can be done to inhibit the TYK2 pathway," Bunick said. 🎥 View the full interview now: https://lnkd.in/gaRpukWc

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Why are Americans paying so much for #prescription medicines? According to the FTC, it’s because the biggest #PBMs -- all owned by health insurers -- are using their market power to drive up costs, pocket #rebate dollars, and force patients to use pharmacies that the PBMs own. At the same time, they’re providing independent pharmacists with “untenably low” reimbursement, undermining the care ecosystem even as costs increase. The scathing new #PBM report from the FTC makes clear that PBMs are often a shell game, using vertical integration and convoluted corporate structures to hide the fact that PBM "benefits" are often just a way to steer dollars to the PBM's other businesses. Patients suffer the most, first paying higher prices than they should be paying at the pharmacy, and later as they will have access to fewer new treatments because #biotech innovation suffers due to PBM greed, unless we call attention to this egregious behavior. #patientsfirst #innovation #biotech Biotechnology Innovation Organization https://lnkd.in/gK2A2zFh

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Great piece by Juliana "Julie" Reed in @Stat outlining the missed savings for the US healthcare system including employers and patients that have been caused by #PBMs greed in choosing higher priced drugs because all of the price concessions and profits they make are based on the list price of the drug. It never was a physician’s choice to choose a biosimilar as the PBMs control that. Now the big 3 ins cos/PBMs are mandating “fail first”biosimilars that physicians can’t afford to buy based on the cost of the drug and underwater reimbursement. We will never realize the true potential of biosimilar savings until the perverse incentive to choose medications that make the most money for these publicly traded PBMs is taken out of formulary construction.

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