Estimands have become a central concept in modern clinical trials, particularly in oncology, where treatment pathways are complex and patient outcomes can be influenced by many post-randomization events. At their core, estimands force us to be explicit about the exact question a trial is trying to answer which has not always been clearly defined in […]

Early-phase oncology combination trials often assume the optimal dose is already included in the dosing grid. When it isn’t, sponsors may face subtherapeutic or overly toxic recommendations, protocol amendments, and delays.  This white paper explores a contour-based method for inserting new dose combinations mid-trial, helping improve dose selection while maintaining safety and regulatory transparency.  What You’ll Learn  About the Author  Matt George, Senior Statistician, Phastar Specialist […]

Real-world evidence (RWE) is no longer peripheral to drug development. Across regulatory, HTA and payer environments, expectations have been shifting rapidly, driven by policy evolution, data availability, and the growing influence of AI-enabled analytics. For sponsors, the question is no longer whether to integrate RWE across drug development stages, but how early, how credibly, and how strategically.  This perspective, authored by Billy […]

Billy Amzal, PhD, Head of Strategic Consulting at Phastar, explores the first year of the EU Health Technology Assessment Regulation in a new article for Regulatory Affairs Professionals Society’s Regulatory Rapporteur.  He examines how Joint Clinical Assessments (JCAs) are beginning to reshape how health technologies are evaluated across Europe, the practical challenges faced by national HTA bodies and developers, and what the […]

Billy Amzal, Head of Strategic Consulting at Phastar, and a Bayesian design expert and pioneer, explores the techniques used in Bayesian trial designs, examines regulatory updates, and shares real-world examples of the impact of these innovative methods in a new article for Applied Clinical Trials. “With rare disease a growing area of clinical research and […]

To mark Rare Disease Day 2026, a new article authored by Billy Amzal, Head of Strategic Consulting at Phastar, explores how external control arms, Bayesian borrowing, and AI-enabled digital twins can strengthen evidence generation and accelerate rare disease drug development, while meeting evolving expectations from regulators such as the FDA and MHRA. ”Innovative trial designs […]

You were among the first to submit and defend a Bayesian trial design to the FDA around 20 years ago. How were Bayesian methods viewed by regulators at that time?     At that time, the statistical community was still quite split between frequentists and Bayesians, so it seemed additional educational work was required. In the early 2000’s, Bayesian approaches were viewed mostly as exploratory tools, essentially for early phases, in indications […]

As innovative clinical trial designs gain traction, sponsors are looking more closely at how real-world data can be used to strengthen evidence generation without compromising rigor. Clearer regulatory guidance, improved data access, and advances in analytics are opening the door to more sophisticated approaches, but only when RWD is applied with intent and discipline. In […]

Phastar is excited to be attending European Statistical Forum, 12 – 13 November in NH Budapest City, Budapest. Connect with us at our presentation or request a meeting below: Experiences of Utilizing the BOIN12 Method for Designing Rare/Small Population Oncology TrialsGiles Partington, Consultant Statistician13 November 2026 Schedule a time to meet below:

As patient-centerd research continues to shape clinical trial design, electronic patient-reported outcomes (ePROs) are increasingly being considered for use as primary endpoints. In a new article for the Journal of Clinical Studies, Barbara Arch, Principal Statistician at Phastar, examines the opportunities and challenges of using ePRO data in this way, drawing on learnings from three […]

As APAC continues to embrace digital innovation, hybrid decentralized clinical trials (DCTs) are emerging as a practical and scalable model for broadening patient access. In a new article for BioSpectrum Asia, Ping-Chung Chang, Head of Business Transformation and China General Manager at Phastar, explores how AI-enabled data processes, real-world data growth, and improved digital infrastructures […]

As the pharmaceutical landscape evolves, traditional clinical trial designs are increasingly complemented by innovative, data-driven approaches. Real-world data (RWD) offers the potential to augment trials, simulate patient populations, and generate synthetic control arms. In-silico trials, powered by RWD and advanced analytics, help provide a path to faster, more efficient, and highly informative drug development. This […]

By Billy Amzal, Head of Strategic Consulting, Phastar  Introduction  Rare disease research demands tailored development and access strategies supported by innovative trial designs and dedicated analytics. From Bayesian designs to data augmentation approaches that strengthen probability of success, specialist CROs and experienced consultants are helping deliver smarter, faster, and more efficient drug development, ultimately bringing […]

Introduction  Integrated Summaries of Safety (ISS) and Effectiveness (ISE) bring together data from across clinical trials to give regulators a clear picture of a treatment’s risk and benefit profile. While the Common Technical Document (CTD) format provides clinical summaries of efficacy and safety (SCE and SCS), US regulations still require detailed ISE and ISS documents, […]

Introduction   When preparing a regulatory submission, one of the most critical components is the Integrated Safety Summary (ISS). Far from being a simple aggregation of safety data, the ISS is a detailed and carefully constructed analysis that provides regulators with a complete picture of a product’s safety profile across clinical trials. This blog explores […]

In this exclusive interview for World Pharma Today, Phastar’s Head of Strategic Consulting, Billy Amzal, shares his career insights, regulatory perspectives, and how data-driven strategies are accelerating smarter, safer, and more impactful healthcare decisions. ”We are increasingly seeing regulatory agencies specify the context of use for novel approach methodologies such as Bayesian statistics. I believe […]

With major changes ahead for EU Health Technology Assessments (HTAs), including the introduction of joint clinical assessments from 2025, developers must adapt quickly to new regulatory expectations. In a recent article for PharmaPhorum, Stephen Corson, Associate Director, Statistics and Technical Solutions and Head of Statistical Consultancy at Phastar, outlines how developers can prepare for the […]

The implementation of the New Health Technology Assessment (HTA) framework in Europe is a pivotal moment for the region’s healthcare landscape. While this initiative promises to streamline and harmonize the evaluation of health technologies across the European Union (EU), it also presents several significant challenges that stakeholders must address.  This blog explores the challenges and […]

Introduction  The EU Health Technology Assessment (HTA) is set to undergo significant transformation at the beginning of next year, with the upcoming joint clinical assessments (JCAs) aiming to streamline processes across member states. The new framework marks a substantial departure from the previous decentralized model of HTA in Europe.   This shift presents both opportunities and […]

Introduction Health Technology Assessments (HTA) are a vital process within healthcare systems, evaluating the value of medical interventions, such as drugs and medical devices, based on their clinical, economic, and ethical impacts. Traditionally, HTAs have been conducted on a national level across Europe, leading to significant variations in how health technologies are assessed and adopted […]

Our recent webinar explored the complexities of Health Technology Assessments (HTA), their current role in evaluating healthcare technologies, and the upcoming changes to HTA regulations in Europe.   The upcoming regulatory shift will require developers to generate a substantial body of evidence within much shorter timelines than current guidelines require. To help you prepare for this […]

As oncology drug development continues to evolve, the FDA’s Project Optimus is reshaping how clinical trials approach dosing—moving away from the traditional maximum tolerated dose (MTD) model towards a focus on dose optimization. This initiative aims to enhance the safety and efficacy of cancer treatments, ensuring patients receive the best possible therapeutic outcomes with minimal […]

With the upcoming introduction of the new EU HTA guidance in January 2025, sponsors in the pharmaceutical, Biotech and medical device sectors will face a significant regulatory shift. This change will demand the generation of a substantial body of evidence within stringent timelines. To help you prepare for this transition, we are hosting an informative […]

Introduction Project Optimus marks a major advancement in optimizing patient outcomes in oncology. By prioritizing dose optimization, this groundbreaking initiative aims to enhance the efficacy and safety of cancer treatments and improve drug development processes. However, implementing Project Optimus comes with its own set of challenges. This blog discusses the complexities of aligning clinical trial […]

Introduction With the launch of Project Optimus, the FDA is overhauling the paradigm of dose optimization and selection in oncology drug development. This initiative tackles the problem of poorly characterized doses and schedules, which can result in increased toxicity without enhancing efficacy.   This transformation will require substantial changes in the design and execution of early […]

Introduction The Project Optimus initiative is a key part of the U.S. Food and Drug Administration (FDA’s) Oncology Center of Excellence (OCE) and represents a shift in the agency’s approach to cancer treatment dosing. [1] The three main goals of Project Optimus are:  These goals are to ensure that more patients receive the appropriate dose […]

Introduction With the launch of Project Optimus, the FDA aims to reform the dose optimization and dose selection paradigm in oncology drug development. The initiative addresses the issue of poorly characterized doses and schedules, which can lead to increased toxicity without additional efficacy.   This transformation necessitates significant changes in how early phase trials are designed […]

By Wei Liu, PhD, Associate Director, Biostatistics, and Gillian Armstrong, MSc, Consultant Statistician The FDA recently published a draft guidance on the Use of Data Monitoring Committees (DMCs) in Clinical Trials for consultation. Leveraging our substantial expertise in DMCs, a select group of Phastar statisticians thoroughly reviewed and provided insightful comments on the guidance. By […]

With the launch of Project Optimus, the FDA is reforming the dose optimization and dose selection paradigm in oncology drug development. The initiative addresses the issue of poorly characterized doses and schedules, which can lead to increased toxicity without additional efficacy. This transformation necessitates significant changes in how early phase trials are designed and conducted. […]

By Tina Hovey, Principal Statistician, and Gillian Armstrong, Consultant Statistician, Phastar The FDA recently published a draft guidance on Master Protocols for Drug and Biological Product Development (fda.gov) for public consultation and feedback from interested parties (such as the PSI Regulatory Special Interest Group). At Phastar, we have substantial expertise in Master Protocols therefore, our […]

EU HTA regulations are changing the way we conduct HTA submissions with EU regulators. Prior to the introduction of this legislation, submission to each member state’s regulatory body could be done in a staggered approach. This won’t be the case with EUnetHTA. Read our latest article on the impact of these changes and how Phastar […]

The way health technology assessments (HTAs) are conducted in Europe is undergoing a significanttransformation. Starting next year, all new advanced therapeutic products and oncology drugs will be subject to a mandatory, EU-wide joint clinical assessment (JCA). By 2030, this will extend to orphan drugs, class IIb or III medical devices, and class D in vitro […]

A new formulation under investigation was planned to be submitted for use in both the US and Europe. As the reference formulation in the US differs from that in Europe, two separate bioequivalence studies were needed to gain approval from the FDA and EMA. Study 1 was conducted for FDA approval and successfully demonstrated bioequivalence. […]

Presented by: LaRee Tracy, PhD, and Joshua Baker, PhD Complete the form below to watch the webinar

Phastar provided a team of statisticians and programmers to deliver the analysis and reporting of a program of four trials in a genetic rare disease, and the corresponding integrated summary of efficacy and safety, to support a regulatory submission to the FDA. There were four studies comprising twelve, one, two, and three patients. The one […]