AMIT KATOCH’s Post

Day 2 of learning Pharma Business Research Today, we will explore various special regulatory pathways and their impact on the drug regulatory review process. [Simple yet complex] I will highlight the designations granted by the US FDA, along with their specificities. You can compare these with the equivalent EMA designations in the attached image. 1. Orphan Drug Designation (ODD): This designation includes benefits such as tax credits (25%), R&D grants, accommodating trial design during phase 2 & 3, reduced review fee with comprehensive regular FDA communications, and 7 years of market exclusivity 2. Fast Track Designation (FTD): This designation allows for more frequent communication with FDA and rolling FDA review starting from phase 2 (if successful, this phase 2, instead of phase 3, would, therefore, be sufficient to prove a drug’s safety and efficacy). Additionally, FDA may require a post-marketing trial to address uncertain side effects, toxicity, or treatment outcomes 3. Accelerated Approval (AA): This pathway allows the use of surrogate endpoints instead of regular clinical endpoints during phase 2 or/and 3. Just like FTD program, the FDA may require post-marketing trials for drugs approved under the AA program 4. Priority review: Guided by PDUFA, this program replaced a more complex system by prioritizing and allocating more resources to NDAs/BLAs based on their therapeutic gain. It expedites the review period, reducing it from the standard 10 months to 6 months for priority reviews. 5. Breakthrough designation (BTD): This designation offers advantages like adaptive trial design and the use of surrogate endpoints. It includes all fast track benefits along with early communication with the FDA starting from phase 1, meetings with senior FDA staff, and a cross-disciplinary project lead. However, the FDA may request a phase 4 trial For more details you may read the following article: https://lnkd.in/gfJUsfZp [Image credits: Michaeli, D.T., Michaeli, T., Albers, S. et al. Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy. Eur J Health Econ 25, 979–997 (2024)]

Bringing a new drug to market in the United States and Canada involves navigating complex approval processes. Both the FDA in the U.S. and Health Canada share a focus on ensuring that new medications are safe, effective, and high-quality. The process starts with preclinical studies, followed by clinical trials in three phases to assess safety and effectiveness in humans. After successful trials, companies submit a New Drug Application (NDA) to the FDA or a New Drug Submission (NDS) to Health Canada, which includes all study data, manufacturing details, and risk/benefit analyses. Key differences between the two systems include the time for review, economic evaluation requirements, and approval documentation. The FDA’s standard review period is typically 10-12 months, with options for expedited reviews, while Health Canada's process can take longer due to additional pharmacoeconomic evaluations. Both countries also have pathways like Priority Review and Fast Track to accelerate approval for urgent therapies, though they require follow-up studies to confirm long-term safety and efficacy. Post-market surveillance plays a crucial role in monitoring drug safety after approval, involving Phase IV studies and adverse event reporting. Pharmaceutical companies must also consider reimbursement processes, which are handled separately by insurers in the U.S. but require further reviews for public drug plans in Canada. Understanding these differences is essential for pharmaceutical companies looking to enter both markets. Early engagement with regulatory bodies, leveraging expedited pathways, and preparing detailed submissions can help streamline the approval process. With the right strategy, companies can bring innovative therapies to patients more efficiently. ROBOREG offers expert guidance to help navigate these regulatory challenges, ensuring new drugs meet the requirements of both the FDA and Health Canada. Read more (link in the comment 👇)

✨BioPharma Services Just Published a New Blog ― Discover the critical role of controlled clinical trials in drug development. Phase 1 trials are the essential foundation for any drug's development, setting the stage for safety, dosage, and potential efficacy. These trials use different control methods—placebo, active controls, and dose-comparison groups—to ensure objective results that validate treatment potential while minimizing variables that could skew the data. By establishing rigorous controls early, we make informed decisions that shape each next step in the development journey. As a trusted partner, BioPharma Services brings the highest standards of data integrity and trial management to early-phase studies. Our goal? To give sponsors the confidence they need to advance their pipeline assets with accuracy and transparency. Read the full article to understand how controlled trials drive progress in drug development: https://hubs.li/Q02WF5pw0 BioPharma Services Inc. specializes in #Phase1 clinical trials, bioavailability, bioequivalence, First-in-Human and Human Abuse Potential Studies (#HAP). Let our world-renowned #Pharmacokinetics team execute your next clinical trial with confidence. . . . #clinicalresearch #clinicalstudies #clinicaltrials #bioanalsysis #cro #clinicaltrials #Biopharma #drugdevelopment #biotech #blog #pharmaceuticalindustry

Access to affordable medicines remains a significant public health priority for FDA. The agency regularly conducts economic analyses to measure the impact additional approved generic equivalents can have on consumer generic drug prices. These studies have continually demonstrated that greater competition from generic drugs can help affordability and improve access to drugs for patients and consumers. This study estimates savings from the 633 #generic drug applications fully approved by #FDA in 2021. This work is a continuation of previous research using similar methods to estimate savings from generic drug approvals in earlier years, all of which shows the relationship between generic competition and drug prices. We recognize that there has been increasing attention on whether the lower cost of generic drugs, especially as more generics enter the market over time, may place pressure on companies to adopt strategies that lower the cost of manufacturing, which in turn may lead to supply disruptions and shortages. This paper focuses on the savings from generic drug approvals in 2021 and this larger policy question is not the focus of this analysis. In 2017, FDA approved the most generic drug applications ever, with 843 fully approved applications and 184 tentatively approved applications • FDA estimates total cost-savings associated with 2017 ANDA approvals to be $11.8 billion through February 2018, with $8.8 billion in savings occurring through December 2017 • FDA estimates total cost-savings over one full year after each generic approval in 2017 to be $16.0 billion. • Approved Drug Products with Therapeutic Equivalence Evaluations (FDA’s “Orange Book”) – Identifies 2017 ANDA approvals with approval dates2 • IQVIA National Sales Perspectives (sales through February 2018) – Sales volume ($) and quantity sold (what IQVIA calls “eaches”, such as pills), monthly3 • FDA Electronic Drug Registration and Listing System (eDRLS) – Marketing status and NDC identifiers4 • FDA Document Archiving, Reporting, and Regulatory Tracking System (DARRTS) – Additional reference listed drug information used for matching5 • Bureau of Labor Statistics, Consumer Price Index – Inflation adjustment (of sales) to January 2018 base.

FDA: 7 Things to Know About Drug Shortage Management The U.S. Food and Drug Administration (FDA) has published an article on how the FDA Center for Drug Evaluation and Research (CDER) manages drug shortages. The FDA works closely with the pharmaceutical industry and other stakeholders to help prevent drug shortages and lessen the impact a delay in the availability of medicines will have on patients. The article lists how the FDA CDER manages drug shortages, such as having the Drug Shortage Staff (DSS) acting as CDER’s focal point during drug shortages. The DSS evaluates and manages drug shortages by establishing and maintaining a network of designated contacts within CDER to address drug availability issues comprehensively, monitoring drug supply and demand to help ensure availability, and serving as a liaison to private industry, other FDA centers, and other government organizations. The DSS will consider that a product is in shortage if certain parameters are met such as the national supply available for a product within the U.S. is not adequate, the manufacturer of the product is not able to meet demand or to increase production to cover the shortfall, and the public or practitioners report a lack of product available in the market. CDER posts drugs on the Drug Shortage Database once they are determined to be in shortage. The DSS is responsible for coordinating all activities related to preventing and mitigating drug shortages. Drug companies must notify the FDA about permanent discontinuances in manufacturing and manufacturing interruptions that are likely to lead to a meaningful disruption in the supply of that drug. Patients, health care professionals, and organizations can report new shortages through a public notification portal or [email protected] or 240-402-7770. The DSS will consider that a product is resolved if the “current” shortage is moved to “resolved” status when all the manufacturers combined can meet total national pre-shortage supplies as seen with market data, or what meets current market needs, as supply amounts level out. View the FDA article here: https://lnkd.in/gZgAwj6X

🔬 **Exploring the World of Investigational Drugs: A Key Step in Medical Advancement** Ever wondered what "investigational drugs" are and why they're essential to healthcare innovation? Here’s a breakdown: 💡 **What Are Investigational Drugs?**   An investigational drug is a substance under study but not yet approved by regulatory authorities (like the FDA) for widespread use. These drugs undergo rigorous clinical testing to ensure they’re safe and effective before they reach the market. 📈 **Applications of Investigational Drugs:**   1. **Treatment of Unmet Needs**: They offer hope for conditions lacking effective treatment options.   2. **Expanding Disease Understanding**: Research on these drugs provides critical insights into diseases and potential cures.   3. **Compassionate Use Programs**: Sometimes, investigational drugs are used under special programs to treat patients with severe or life-threatening conditions.  ⚙️ **Principles Guiding Investigational Drug Use:**   - **Scientific Rigor**: Extensive preclinical and clinical testing in phased trials.   - **Ethics**: Focus on patient safety, with informed consent as a cornerstone.   - **Regulatory Oversight**: Ensuring transparency and safety under the watchful eye of regulatory bodies.  Investigational drugs are at the heart of transforming possibilities into realities in medicine. 🌐 If you’re passionate about pharmaceutical research or simply interested in medical advancements, this is an exciting area to follow! #Pharmaceuticals #DrugDevelopment #ClinicalResearch #InvestigationalDrugs #MedicalInnovation #Healthcare #Biotechnology #ClinicalTrials #PharmaceuticalIndustry #NewDrugApproval #DrugDiscovery #PatientSafety #HealthcareInnovation #FDA #LifeSciences #Biopharma #PharmaResearch

Fast-track Your Drug Development: Unlock Time Savings in Canada 🍁 Navigating the landscape of first-in-human (FIH) clinical trials can significantly impact your drug development timeline. With the evolving regulatory environments, initiating Phase I clinical trials in the EU and the UK demands a considerable investment in time and resources due to the complexity of the EU clinical trial application (CTA) preparation and submission processes. Going can-EH-dian can offer a more streamlined approach, potentially saving up to six weeks on your drug development schedule compared to the EU. This time efficiency can be a critical advantage in the highly competitive pharmaceutical landscape. Want to learn more? Click here to get started 👇 https://hubs.li/Q02nWGNS0 BioPharma Services Inc. specializes in #Phase1 clinical trials, bioavailability, bioequivalence, First-in-Human and Human Abuse Potential Studies (#HAP). Let our world-renowned #Pharmacokinetics team execute your next clinical trial with confidence. . . . #clinicalresearch #clinicalstudies #clinicaltrials #bioanalsysis #cro #clinicaltrials #Biopharma #drugdevelopment #genericdrugs #biotech #pharmaceuticalindustry #CTA #drugapplication

Choosing the right clinical outcome assessments (COAs) is a vital step in advancing drug development and securing FDA approval. With 55 new drugs approved last year, COAs play a growing role in capturing meaningful patient outcomes and demonstrating the clinical benefits of novel therapies. Key Insights - ▶ COAs ensure trial outcomes reflect real patient experiences. ▶ They impact the labeling of treatment benefits and risks. ▶ COA selection must consider patient burden, reliability, and regulatory standards. Questions to Consider - ▶ How can we ensure COAs align with patient health concerns? ▶ What are the risks of choosing an ineffective COA in a clinical trial? #DrugDevelopment #ClinicalTrials #Pharma #COA #FDAApproval #HealthcareInnovation

🤝 European Sponsors, Meet Your Perfect Clinical Trial Partner! Clinical drug development in Europe? It requires a deft touch, deep understanding, and a reliable partner. BioPharma Services has overseen 2,200+ clinical trials and boasts regulatory expertise across Europe and beyond. 🇪🇺 🇨🇦 Let's innovate and elevate your drug development journey together. 🔬 🔗Learn more here: https://hubs.li/Q02VG6Mx0 BioPharma Services Inc. specializes in #Phase1 clinical trials, bioavailability, bioequivalence, First-in-Human and Human Abuse Potential Studies (#HAP). Let our world-renowned #Pharmacokinetics team execute your next clinical trial with confidence. . . . #clinicalresearch #clinicalstudies #clinicaltrials #bioanalsysis #cro #clinicaltrials #Biopharma #drugdevelopment #genericdrugs #biotech #pharmaceuticalindustry

🚀 Exciting Insights on New Drug Approvals! 📈 I would like to share key findings from the source: CIRS - Centre for Innovation in Regulatory Science R&D Briefing 93 focused on new active substance (NAS) approvals across six major regulatory agencies from 2014 to 2023. This analysis highlights the evolving regulatory landscape and the impact of facilitated regulatory pathways (FRPs) on drug availability. Key Highlights: 1. Approval Times:   - In 2023, the PMDA had the fastest median approval time at 332 days, followed closely by the FDA at 333 days. The EMA lagged with a median time of 453 days.    2. NAS Approvals:   - The FDA led in approvals with 61 NASs, while Swissmedic and **Health Canada followed with 35 and 34 NASs, respectively. 3. Facilitated Regulatory Pathways:   - The usage of FRPs has increased significantly, with 74% of FDA approvals utilizing at least one FRP. This trend underscores the importance of expedited pathways in addressing unmet medical needs. 4. Orphan Designation:   - A notable 63% of NAS approvals in Swissmedic were for orphan drugs, indicating a growing commitment to rare disease treatments. 5. Therapeutic Areas:   - Anti-cancer and immunomodulator therapies represented 42% of all approvals, reflecting the pressing need in this area. 6. Submission Gaps:   - The FDA had a 0-day submission gap, indicating companies often submit first to the U.S. agency, followed by EMA (25 days) and others with significantly longer gaps. These insights not only reflect the current trends in drug approvals but also the ongoing efforts to enhance patient access to innovative therapies. For those interested, you can read the full briefing here: PDF #Pharmaceuticals #RegulatoryScience #HealthcareInnovation #CIRS #DrugApproval