ABOUT PREMIER RESEARCH

Built for Biotech

Your compound shows spectacular promise, but it seems there’s an obstacle at every turn – finding hard-to-reach patients; defining relevant endpoints; navigating the regulatory maze; and ultimately, obtaining clean, conclusive data.

Premier Research is a clinical research organization that leverages leading technologies and predictive recruitment models so our teams can build statistical, adaptive approaches and find efficiencies that support Fast Track designation and orphan drug status. Our Built for Biotech model is a compilation of the most successful techniques and capabilities observed across our experience, applied to meet your highly specific needs.

It’s amazing and often very complex science, and we’ve positioned ourselves right in the middle of the action, where pioneers like you are doing the most exciting work. More than just clinical services, we offer unique perspectives, intelligent study designs, and relentless focus on compliance and providing conclusive data.

Firsthand experience with many different systems and processes informs our decisions on what will work best for individual customers. We complement your team with specialized expertise in your therapeutic area to reduce the burden of analysis. 

Our specialized global task forces are dedicated to study optimization and risk management throughout each trial to support drug development strategies, and our ongoing investments in innovative technologies for smart study design and trial management allow us to continually improve product development and study outcomes. Our regulatory consultants are available throughout development from Phase 1 through post-marketing to support trial design, regulatory submissions, and compliance.

We are adaptable, passionate, and customer-focused – smart, experienced, and united by a desire to help change the course of medical science. We’re constantly building on our successes to directly influence the outcome of our customers’ development plans. If you’re looking for a clinical development partner that delivers outcome-focused insight and shares your commitment to developing life-changing therapies, we should talk.

FEATURED PRODUCTS

Pioneers developing the latest targeted therapies turn here for innovation and expertise. We’ve conducted over 170 trials across numerous indications in the past five years.

CONTACT INFORMATION

Premier Research

3800 Paramount Parkway Suite 400

Morrisville, NC 27560-6949

UNITED STATES

Phone: + 1 910 447 3156

Contact: N/A

FEATURED ARTICLES

  • Given the right expertise, Rx-to-OTC conversions are easier to achieve with less time and expense than one might expect for a regulatory exercise.

  • Patient enrollment and retention for investigational trials are notoriously difficult. How is enrollment affected when there is no direct benefit to the patient? Find out in this blog post.

  • This blog discusses key rare disease recruitment challenges and explores strategies for differentiating trials in today’s competitive landscape.

  • Registry and natural history studies can be very important to the clinical development of therapeutics in rare diseases. To accommodate these advances in knowledge, study design flexibility is a must.

  • Explore seven tips on designing and operationalizing natural history studies to support successful rare disease therapeutic development.

  • This blog post explores the distinctions between registries and natural history studies, highlighting the potential value of each in informing and shaping clinical development in rare diseases.

  • Oncology clinical trial activity in the Asia-Pacific (APAC) region has been robust over the past decade, increasing 138 percent from 2010 to 2020. In this blog post, we highlight seven factors contributing to oncology clinical trial activity in the APAC region.

  • This webinar examines the advantages of conducting trials in this fast-growing region and delves into other issues sponsors should consider when choosing sites for upcoming trials.

  • Explore the advantages of conducting oncology clinical trials in the APAC region as we share strategies to help ensure study success.

  • Explore the growth of oncology clinical trials in APAC countries and strategies to support global study success in this dynamic and complex region of the world.

  • Sponsors are increasingly turning to DCT approaches to accelerate their development programs.This expert guide offers five strategies for addressing DCT challenges and optimizing study success.

  • Each year, nearly 85 million Americans see a physician for at least one skin disease, with $75 billion in direct costs to the U.S. healthcare system. This blog post explores the top seven challenges in decentralized dermatology trials and how to address them.

  • This webinar identifies key points to consider when designing and planning decentralized dermatology clinical trials, and discusses reporting technologies such as photography (at home and on-site), electronic diaries, wearable biomarker devices, and mHealth.

  • Discover how mobile health (or mHealth) solutions provide increased access to care, making it easier for patients to receive care and for healthcare professionals to deliver that care.

  • Discover tips on operationalizing gene therapy studies in order to optimize site selection and patient recruitment and engagement.

  • Explore the the key considerations for designing successful gene therapy trials in the available blog.

  • The promise of gene therapy is to provide transformative treatments that meaningfully improve quality of life for patients, many of whom are currently living with debilitating diseases. Explore the emerging landscape of gene therapy and high-level considerations for studies of these advanced therapeutics in this blog.

  • Find out how recent approvals of targeted therapies have led to meaningful improvements in patient care and outcomes and have also stimulated research, particularly in combination trials.

  • In this blog post, learn about the strategies sponsors should consider during the study planning process to maximize patient engagement and retention.

  • Dive into this case study to see how a U.S.-based developer of an investigative therapy for mCRPC was able to increase enrollment in a Phase 2 mCRPC study involving 100 patients at six sites in the United States.

  • Discover the critical unmet need for better maintenance therapies to improve the quality of life and to achieve improvement in the overall survival for women with advanced ovarian and endometrial carcinomas.

  • Healthcare payers, regulators, and physicians focus on clinical utility when evaluating molecular diagnostics tests. However, establishing that the test can make a diagnosis or predict clinical outcomes may not be enough to garner acceptance. Explore ways to approach designing and conducting a randomized clinical trial (RCT) to demonstrate clinical utility.

     
  • Walk through the four primary challenges to In Vitro Diagnostic Regulation (IVDR) conversion and discover solutions to simplify and de-stress the process so that IVD manufacturers can approach deadlines confidently and avoid having products pulled from the market.

  • On May 26, 2017, the EU Regulation 2017/746 of the European Parliament and the Council on in vitro diagnostic medical devices (IVDR) became active under the EU Regulation on Medical Devices (MDR). Discover answers regarding the expected changes in this blog.

  • A client developed a unique solution for Colorectal cancer screening – a multi-target, noninvasive screening test that could be facilitated by patients in their homes. Gather information on how the pre-market approval (PMA) study was conducted.

  • Point-of-care (POC) tests offer significant convenience for patients and physicians, as well as cost savings for payers. Yet, achieving regulatory approval can be daunting. Discover how a clear strategy was developed to ensure that the necessary data to support both 510(k) clearance and CLIA waiver would be collected within a single clinical trial.

  • Healthcare payers, regulators, and other stakeholders increasingly focus on clinical utility when evaluating molecular diagnostics tests. Learn why this focus makes it vitally important for diagnostics developers to design and conduct trials that demonstrate the clinical utility of their tests.

  • Regulatory approval is the final hurdle sponsors face when launching an In Vitro Diagnostic (IVD). After spending many years and millions of dollars on development, find out how it can prove frustrating when the finish is in sight, but there are barriers to success.

  • In this blog post, regulatory experts highlight eight new concepts in the published In Vitro Diagnostic (IVD) Medical Devices Regulation (EU) 2017/746 (IVDR) document and identify ways to think about IVD approvals to help prepare for the IVDR switch.

  • Heavy menstrual bleeding (HMB) is one of the most common gynecologic disorders among women of reproductive age, accounting for up to one-third of all gynecologic office visits. Discover key operational and methodological considerations for heavy menstrual bleeding clinical studies.

  • This blog describes how effectively involving physicians and site staff during the trial design process can drive efficiencies that may improve recruitment efforts.

  • The focus on women’s health research means sensitivity to the concerns and needs of the female volunteers contemplating clinical trial participation. To effectively enroll women, it’s important to acknowledge these volunteers and consider their perspectives in every step of the clinical research process.

  • When sponsors develop drugs and devices specifically for women, they need to create trials that are uniquely female-focused. Explore trial considerations, including the unique needs, expectations and regulatory requirements of the female population.

  • With investment in research on the rise, explore the challenges investigators face in patient identification, site selection and the ever-changing regulatory landscape in the available webinar.

  • Women are demanding more power, information, and control when it comes to their health. Learn how women now have a stronger voice when it comes to clinical research for treatments that impact their care.

  • This white paper explores key considerations for site selection and dose-escalation design strategies in early-phase oncology trials.

  • The European regulatory landscape for advanced therapy medicinal products (ATMPs) is complex, and the approval pathway depends on product classification. This case study describes how product classification may not always be clear-cut, given the proliferation of novel approaches to ATMP development.

  • This article examines the rationale for combination therapies, mechanisms of resistance, and where to go from here.

  • Patient recruitment gets all the attention at an oncology study’s inception, but enhancing patient engagement throughout the study bolsters retention, long-term follow-up participation, and the likelihood of patients volunteering in future trials.

  • This post provides an introduction to cancer immunotherapy, exploring its immunological basis and the fundamental principles guiding the development of new treatments.

  • Gene therapy holds great promise as a potential treatment for Parkinson’s disease, a disorder for which currently available medications do not causally treat the underlying disease mechanisms. Download the available white paper to find out more.

  • Premier Research, a leading provider of clinical research and development and advisory services to the biotechnology, specialty pharma, and medical device industry, today announced the launch of PremierPredict™, a powerful clinical trial enrollment probability simulator.

  • Premier Research today announced that it has acquired Health Decisions, a Durham, N.C.-based contract research organization (CRO) focused on clinical development in all areas of women’s health and diagnostics.

  • Beginning in 2017 with the approvals of tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™), read how chimeric antigen receptor (CAR) T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies.

  • With the approvals of tisagenlecleucel (KYMRIAH™) and axicabtagene ciloleucel (YESCARTA™) last year, chimeric antigen receptor (CAR) T-cell therapies have changed the treatment paradigm for patients with certain hematologic malignancies. This blog breaks down the development of CAR-T cell therapy as well as its advantages and challenges.

  • All data generated in a clinical trial must be uniformly interpreted to ensure accurate results. Correct medical coding plays a critical role — and how that coding happens may be shifting. How is technology affecting the process of medical coding in clinical trials? Watch our webinar to find out.

  • Over the past two decades, biologic therapies have revolutionized the treatment of psoriasis, with more than half of treated patients now able to achieve essentially complete clearing of their disease. Learn more about these therapies as well as new topical and oral therapies that are emerging.

  • With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. This blog post reviews the regulatory programs available to expedite treatments for rare disorders and serious diseases.

  • New treatment options for medical aesthetic indications are in record-high demand, fueled by growing awareness of the effects of physiological and environmental aging and the influence of lifestyle on skin health. Premier Research explored this evolving landscape in depth in our recent webinar Drug and Device Development Secrets in the World of Medical Aesthetics. In this blog, we discuss five of the most important takeaways for creating a competitive, differentiated aesthetic development program.

  • While treatment of Parkinson’s disease has traditionally been limited to dopamine replacement therapy to alleviate symptoms, gene therapy studies have recently shown encouraging results for both non-disease-modifying and disease-modifying transgenes. Conducting these trials requires careful attention to investigator and site selection and adherence to local regulatory and protocol design requirements. The need for long-term patient follow-up puts added demands on patients and sites.

  • Preparing your market application and planning the lifecycle management of your development program are critical to moving your product through early-phase trials and achieving commercial success. Approval in the United States and European Union paves the way for a drug’s acceptance in many, but not all, other parts of the world — so which additional countries should you consider incorporating in your trial strategy?

  • For a biotech company, sudden growth can come in many forms. When your company finds it necessary to scale quickly while still maintaining complete oversight over every aspect of your study, partnering with a functional service provider (FSP) may be an excellent solution. Learn how a FSP can provide the expertise and instant infrastructure to move things ahead quickly while still retaining complete control.

  • While the shift to DCTs is well underway, concerns remain around patient monitoring and engagement and data reliability and quality. In this blog post, we discuss three takeaways from our experience designing and implementing DCTs to help protect patient safety and preserve data quality.

  • Every drug development program must begin with an understanding of how the relevant key stakeholders factor into the development process. It’s not just a matter of courtesy; a comprehensive examination of the roles played by all participants has the potential to produce faster and more concrete results, leading to improved patient outcomes.

  • Industry-wide, spreadsheets have long been the go-to method for tracking clinical trial start-up. Premier Research developed the Premier One Ecosystem to create a paperless, electronic data process, start to finish. Watch this video to see how the Study Start-Up application delivers fast, efficient, and accurate trial management.

  • During Premier Research’s recent webinar Alternative Designs to the Traditional 3+3 Design in Phase 1 Dose Escalation Studies, Abie Ekangaki, Vice President, Statistical Consulting, and Andreas Schreiner, Vice President, Medical Affairs, Neuroscience & Analgesia, discuss alternative dose-escalation paradigms introduced into the clinical trial landscape for Phase 1 trials. In this blog post, we share five of their key insights on alternative dose escalation strategies for Phase 1 studies.

  • In February, the European Medicines Agency (EMA) released the fourth version of its Guidance on the Management of Clinical Trials During the COVID-19 (Coronavirus) Pandemic. As the pandemic continues to impact clinical research worldwide, these updated guidelines reflect the EMA’s evolving stance, clarifying questions raised by previous versions and taking into account the most recent changes in the global health landscape. Here’s an overview of what’s new.

  • Originally presented at the 2021 LSX World Congress virtual conference, this webinar series by Premier experts in oncology, rare disease, and cell and gene therapies focuses on development considerations for advanced therapies, highlighting these products’ unique challenges as well as their powerful potential to address a broad array of unmet patient needs.

  • A new class of medical software known as Software as a Medical Device (SaMD) is offering innovative ways to capture, optimize and analyze healthcare data with the goal of improving health by utilizing analytics to manage and optimize health outcomes. However, this rich field of technology is evolving more quickly than regulators can keep up. In this webinar, the panelists will define what is considered a SaMD and explore key strategies to bring it to market successfully through regulatory strategy, clinical impact and considerations for patient engagement and participation.

  • The Ministry of Health and Family Welfare, responsible for all health policy in India, took steps to clarify their regulatory requirements and address many of sponsors’ concerns with the publication of its New Drugs and Clinical Trials Rules in 2019. With these changes, it is worthwhile for sponsors, especially biotech and specialty pharmaceutical companies, to consider the many opportunities that India and other Asia-Pacific countries offer. We have summarized some of the most significant changes in this article.

  • Pandemic-related disruptions have accelerated much-needed change in clinical operations, but this change has been accompanied by questions about data collection and data quality. While the adoption of DCT approaches can benefit patients, sites, and sponsors alike, successful implementation of these approaches requires careful consideration of the regulatory guidance, processes, and technologies necessary to ensure data quality and manage risk.

  • Designing and conducting a gene therapy trial is a complex undertaking. Learn how understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help bring safe, breakthrough treatments to patients with unmet medical needs.

  • The placebo effect can be problematic in analgesia clinical trials with a symptom-based approach, as placebo-related analgesic responses may occur and persist for some time in up to 60 percent of study participants. Here, we explore how trial designs and training programs may be used to help reduce the placebo effect in analgesia clinical trials.

  • Dermatology clinical research is undergoing a rapid transformation in response to new demands for clinical trials that are more justifiable, safer, and less burdensome for patients. In dermatology, a patient-focused approach can be applied to all phases of the research process. In this blog post, we explore a strategic, patient-focused approach to planning and implementing dermatology clinical trials.

  • The use of placebo control in clinical trials has long been a topic of spirited debate. In analgesia studies, the question of whether or not to use placebo control is further complicated by the placebo response, which can make it difficult to detect efficacy signals. In this blog, our industry-leading analgesia experts take a fresh look at the key considerations surrounding the use of placebo control in chronic pain studies.

  • In the normal course of a clinical trial, delays are often accepted as part of the process. For instance, data entered by the site may not auto-encode correctly and therefore require further review by a medical coder, generally within five days. Under an FSP model, you gain access to a dedicated corps of experts that works alongside your study teams to deliver long-term support for key functions and meet project-specific needs. Read more about the critical advantages global FSO resources offer.

  • There is a longstanding debate on the use of placebo control in clinical trials. Critics argue that it sacrifices the welfare of patients and is unethical if a proven therapy exists. Proponents argue that placebo control is crucial for proving the safety and efficacy of new treatments. This white paper reviews the regulatory, ethical, and cultural considerations surrounding the issue of placebo control in analgesia clinical trials and explores approaches to mitigating the placebo response.

  • Today’s technology enables us to seamlessly integrate data for every stage of a clinical trial, from lab results to mobile health apps, patient-reported data, and much more. These new sources of data allow us to better interpret, predict, and quickly turn around a study that may be stalled due to lagging enrollment, regulatory challenges, or patient safety. See how the ePremier Integration Hub can bring study data and data quality processes together under one platform.

  • In a new and extremely competitive era, there is more pressure than ever to optimize your clinical development strategy and planning for successful study recruitment, retention, and implementation. In this blog post, based on our recent webinar, we explore critical study design and operational success factors for this new wave of dermatology clinical trials.

  • The global pandemic has exacerbated the operational challenges intrinsic to gene therapy at the site level, the project team level, and the sponsor level. To ensure that studies continue to move forward, strategies need to be considered not only for operational efficiency and patient centricity but also as contingency planning in the case of a subsequent wave or quarantine. Here we continue our case study of a rescue Phase 1/2 gene therapy trial involving localized administration of gene therapy using specialized equipment with a look at the operational challenges of these trials during COVID-19.

  • Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help you bring safe, breakthrough treatments to patients with unmet medical needs. Here we introduce a case study as a framework for exploring critical study design considerations of gene therapy trials and offering strategies for addressing those hurdles.

  • As scientific knowledge, clinical experience, and acceptance of gene therapy products have evolved, so have the regulatory frameworks for ensuring the safety of these novel treatments. In this blog post, we review the regulatory frameworks for gene therapies in the U.S., EU, and Japan, with a focus on programs designed to streamline the product development and approval process.

  • Most patients with Binge Eating Disorder (BED) seek help from psychiatrists, nutritionists, or obesity specialists, but there was no approved, effective pharmacologic treatment. With unanticipated interest in pivotal Phase 3 studies, we needed to line up and train the right resources while collaborating effectively with the central laboratory, data manager, and other participants.

  • With billions of neurons, the brain is the body's most complex organ, so it’s not surprising that neuroscience clinical trials face major challenges. In this new blog series, we’ll look at the challenges sponsors face when operationalizing clinical trials and share lessons from our experience in this therapeutic area.

  • The Atopic dermatitis (AD) treatment paradigm has significantly evolved in the last five years. Currently, AD is an area of intense focus for clinical developers. Globally, more than 100 pharmaceutical and biotechnology companies are investigating new therapeutic solutions for AD. This white paper will explore the pathophysiology of atopic dermatitis, as well as the rationale and mechanisms of action of existing and emerging therapies for AD.

  • In drug development, there is always pressure to move quickly, and with today’s push to develop treatments and vaccines for COVID, those pressures are exacerbated. In this webinar, the speakers will ground their predictions for the future in the historic response of regulatory bodies to epidemics, then explore recent shifts in the regulatory environment in response to COVID and the resulting new efficiencies in drug development.

  • The evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy. Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials.

  • There has been a tremendous leap forward in the understanding of the molecular characteristics of tumors— not only in targeted therapies but also in the development of tumor-or tissue-agnostic treatments. In this blog post the changing rare oncology landscape and novel approaches to rare cancer trials are explored.

  • Use of long-acting injectable antipsychotics is often reserved for patients with chronic or treatment-refractory schizophrenia or repeated non-compliance issues. However, recent studies have consistently found a role for these treatments both soon after diagnosis and in the treatment of chronic disease. In this white paper, we will review the symptomatology and pathophysiology of schizophrenia and discuss the pros and cons of treatment with LAIs. We will also explore the challenges of conducting studies in schizophrenia and offer suggestions for maximizing the likelihood of success in clinical trials of these drugs.

  • Sponsors of psychiatric clinical trials may face significant challenges in collecting robust, quality data to support the efficacy and safety of investigative compounds. Identifying and mitigating study design-, subject-, and site-related factors that may influence data quality as early as possible in program development can help to drive study success. In this white paper, we explore a range of factors that, if identified and mitigated early in the development process, can maximize the potential for conclusive study results in psychiatric clinical trials.

  • When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. In our first article, we reviewed gene transfer techniques and gene delivery systems that could potentially be used for treating dermatologic conditions. Now we turn our attention to the current state of gene therapy for dermatologic conditions.

  • This webinar explores the latest changes in dermatology trials in the areas of study design, patient access, data quality, regulatory considerations, and more. Learn what’s required to understand the disease burden, patients’ treatment goals, and other critical elements when devising effective recruitment and retention strategies.

  • This webinar examines the evolving science of gene therapy, covering current approaches such as gene transfer and gene silencing, safety considerations, and delayed adverse events. Learn about the current regulatory landscape, identify opportunities to more closely engage with regulators, and more.

  • Of all the tasks that are handled by executives in clinical operations, few bring as much anxiety and stress as the selection of a CRO partner. Make the right choice and your trial will be a breeze. Make the wrong choice and your trial can go off the rails and require the necessary but hated task of CRO replacement.

  • This shifting paradigm may well underestimate the incidence of rare cancers, and further underscores the need for innovative approaches in developing new therapies. Challenges include patient access, biomarker testing requirements, selecting the right endpoints, developing alternative study designs that minimize sample size and improve data outcomes, and an ever-changing regulatory landscape. This webinar will explore the promise and challenges associated with the planning and execution of rare cancer trials.

  • The clinical research environment has been impacted in multiple ways during the pandemic, ranging from new regulatory requirements to supply chain interruptions and shipping delays. In addition, many of the standard clinical trial logistics are being reassessed. For example, patients are becoming much more comfortable with mobile health (mHealth) devices, accelerating plans for remote data capture, analysis, and sharing. In this post, we’ll cover the importance of process in ensuring a robust strategy is in place for the acquisition and analysis of trial data in the face of a changing clinical research environment.

  • Non-traditional data points, in particular real-world data (RWD) and real-world evidence (RWE), are becoming more and more important in the current research climate. The FDA and other regulators have started to embrace the use of real-world, unstructured data alongside traditional RCT data. In this post, we will cover non-traditional data points, their use in randomized control trials (RCTs), and how they contribute to a successful data strategy.

  • Since early this year, many standard clinical trial processes have been significantly disrupted. In these excerpts from their recent presentation, Premier Research’s Stacy Weil, Senior Vice President, Clinical Data Operations, Strategic Business Optimization, and Nach Dave, RPh., MSc, Vice President, Development Strategy, discuss the need to change the way patients participate in trials and how we collect and monitor data.

  • As the world emerges from the pandemic of novel coronavirus disease (COVID-19), the treatment need for cancer patients is greater than ever. In this episode of Premier Voices, we present highlights from a recent Premier Research webinar on managing ongoing cell and gene therapy (CAGT) trials while looking ahead to the future of oncology research.

  • Despite advances in our understanding of the complex genetic, molecular, and immunological factors which lead to cancer, the success and likelihood of approval rates for oncology remain low. Advances in our understanding of the genetic underpinnings of cancer have led to a growing number of phase 1 studies that are driven by a common mechanism of action or molecular alterations rather than specific disease type. Adaptive design approaches are well suited for helping sponsors optimize dose and dosing regimen, while also narrowing down the indications of interest.

  • Adaptive design approaches can be applied across all phases of clinical development, including early oncology studies. These designs introduce real-time flexibility while a trial is underway, including the capability to select biomarker subgroups that identify patients more likely to respond to treatment, allow dynamic adjustment of dose schedules, adjust the size of the trial, or even combine two separate trial phases into a single seamless trial.

  • With the emergence of personalized medicine, we are seeing a shift in how early-phase oncology trials are conducted, including a growing number of Phase 1 trials reporting preliminary response rates. This shift is due in part to an increase in adaptive trial designs that seek to limit the number of patients exposed to ineffective doses or treatments while accelerating the timeline to the detection of efficacy signals. In this white paper, we address clinical trials in personalized medicine and explore the expanding role of adaptive trial designs in Phase 1 and Phase 2 oncology studies.

  • To understand and mitigate the risk of these delayed adverse events, participants in gene therapy trials may be monitored for a long-term follow-up (LTFU) period, which may be as long as 15 years. During this period, sponsors are challenged with navigating complex regulatory requirements as well as finding innovative ways to keep patients engaged for a decade or more.

  • There is significant unmet need for approved treatments for neonatal-specific conditions. With improved understanding of the unique nuances of research in this young, vulnerable population, sponsors can increase their likelihood of developing and executing successful neonatal clinical trials.

  • In this virtual presentation, we explore how cell and gene therapies are being used to treat tumors, discuss design and operational considerations for new early-phase trials, and review the pandemic’s impact on the clinical trial environment.

  • The direct-to-patient clinical trial model has gained popularity in recent years, and social distancing concerns have only accelerated this trend. This podcast will discuss what to consider when using technology solutions to ensure data integrity, how to protect patient privacy and what recent events suggest about the “new normal” for clinical research if you are considering this arrangement.

  • It is commonly accepted that a single-arm trial utilizing a synthetic control arm (SCA) can also be an adequate design for assessing a new treatment intervention. Single-arm trials demonstrate clinical benefit by showing the positive effects of a new therapy or treatment without the need to use placebo or standard of care as a control. Instead, RWD and RWE comparisons – leveraging a variety of sources – can serve as the comparator.

  • Conducting clinical trials of therapeutics and devices can be daunting, and COVID-19 has made this endeavor even more challenging. Here we share some tips for managing pandemic-related disruptions while collecting the information regulatory agencies will require regarding protocol deviations going forward.

  • COVID-19 has dramatically changed the way we conduct clinical trials and left many sponsors without answers when it comes to the future of their studies. This podcast explores challenges sponsors see as a result of this crisis, the impact those challenges are having on trial procedures, and how modifications to the statistical analysis plan can point a path forward.

  • A European biotech company was conducting a pediatric trial involving a rare, incurable genetic disorder. In addition to new regulatory considerations, this trial also called for an extremely challenging intracerebral administration. To succeed, they needed a partner that could develop a clear, compliant strategy for obtaining informed consent and implementing an innovative data management plan to track patients between surgical and clinical sites.

  • This blog includes what an iPSP for a molecularly targeted cancer should address and what elements it should include.

  • Traditionally, the use of patient-reported outcome (PRO) data has been an adjunct to primary data when it comes to clinical trials; however, in the current environment, reliance on PRO models has gained steam. As the FDA and research community continue to evolve in terms of how and what data gets captured during the COVID-19 pandemic, one of the areas coming to the forefront is data collection using electronic patient-reported outcomes (ePRO) tools.

  • We knew going in that it could be the perfect recruiting nightmare with extremely complex inclusion/exclusion criteria. By letting sites set their own goals and commit to them along with assigning project coordinators that stayed close to those sites the study is off to a good start.

  • Of the many types of skin stem cells that have been identified, epidermal stem cells — primarily keratinocyte stem cells — are recognized to play a key role in tissue repair and skin regeneration. These cells have been characterized as rare, infrequently dividing, and capable of generating the short-lived, rapidly dividing cells involved in regenerating the epidermis and repairing skin injury. This article discusses the challenges of developing cell-based therapies in dermatology.

  • In an effort to maintain the continuity of our clinical trials during the COVID-19 pandemic, Premier Research is helping customers take steps to safeguard the well-being of patients who were previously expected to go to a medical facility to receive treatment.

  • In a progressive effort to mitigate these long-standing concerns and bring a level of clarity to its guidance documents, the FDA has created a pilot program called “Guidance Snapshots.” As experts, we are always debating about what the guidance is asking and how the contents are to be applied.

  • With recent updates to the ICH Good Clinical Practice guidelines biotech and specialty pharma innovators face even greater sponsor responsibilities, the most significant of which may be design and development of Clinical Quality Management Systems to achieve compliance with the revised guidelines. In this white paper, we will explore the revised guideline and discuss its impact on small biotech and specialty pharma sponsors, with a focus on risk-based approaches to quality management.

  • In November 2016, for the first time in 20 years, ICH GCP was updated by means of an addendum that provides additional guidance without altering the existing text. Now that we are in the implementation phase of the ICH GCP revision, it may be a good idea for biotech and specialty pharma innovators to review the key principles of ICH GCP and how they have been updated to reflect the realities to today’s clinical trial landscape

  • Under the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Guideline for Good Clinical Practice (GCP), sponsors have extensive responsibilities for ensuring not only the ethical and scientific quality of clinical trials, but also the protection of study participants and the integrity of clinical trial data. The recent update of ICH GCP provides new, more detailed guidelines for sponsors regarding the handling of clinical trial data, documents, and systems.

  • In response to the Coronavirus Disease 19 (COVID-19) pandemic, the scientific community, industry, and regulatory agencies are pulling together to facilitate the development of diagnostic tests, drugs, and vaccines to help prevent the spread of the disease. Read more about this unprecedented level of collaboration in the scientific community.

  • As we continue to face health emergencies and imminent threats to our safety and well-being, the medical community is tasked with bringing solutions to the patients in an expedited and safe manner. One such opportunity is to partner with the US FDA by submitting an Emergency Use Authorization (EUA) for any products that could immediately and effectively address a health emergency.

  • We’ve gathered questions from our customers and vendors regarding potential impact of COVID-19 on current and planned studies. Although we may not have answers for every situation, here are some areas where sponsors should be focusing.

  • A good budget is critical at the outset of every trial, but on Day 1 that budget becomes a forecast as you begin accounting for enrollment of new sites, protocol amendments, and change orders. Trials are moving targets — and the more complicated and global they become, the more they challenge our forecasting acuity. Using purpose-built budgeting and forecasting tools you can see what’s happening in the moment as study parameters change and adjust quickly.

  • Patient-driven changes are systematically beginning to inject more than token patient participation and viewpoints into all stages of drug and device development and slowly, but methodically, chipping away at the clinically isolated way drugs and medical devices are developed.

  • You get one chance to initiate a clinical trial. Botch the start-up and you’ll expend great effort correcting course and playing catch-up. The waste of money and time — commodities that are chronically scarce among the biotech and specialty pharma companies that comprise most of our customer base — can be devastating. Finding a partner that consolidates all the expertise needed can get your trial off on the right foot.

  • The ICMRA is now considered a strategic cross-border leader among a select group of global medicine and device regulatory policy bodies. Read how the coalition is working across the world to help sponsors develop medical products in a more globally harmonized way moving forward.

  • According to a Council of Foreign Relations report, ensuring a safe and secure healthcare marketplace that can provide innovative therapies is no longer an undertaking for a single nation. Larger harmonization agreement efforts, even with intervention by the World Health Organization (WHO), ended many times when country regulators bickered over who had the power to develop the standards and whose standards should be followed. The ICMRA, ICH, and other affiliated harmonization groups are acting to correct this.

  • With a recruiting target of 18 patients for a condition that afflicts just one in 31,000 people this CRO had to make full use of what few resources were avaiable. By working with KOLs and a patient advocacy group they located their 18 patients ahead of schedule and completed the 26-week trial early.

  • Since 2002, doctors and researchers have used a standard rosacea classification system to provide consistent terminology as well as to facilitate studies, clinical diagnosis, and treatment. However, in 2018, the Journal of the American Academy of Dermatology published a new standard classification system that replaces the previous one. Following is some of the most important information found in this publication.

  • As the cost and complexity of drug development continue to increase, the Asia Pacific (APAC) region is a key destination for the conduct of clinical trials. Lower-cost and a large population of patients are just a few of the advantages. This blog weighs the advanatages and challenges of studies in APAC countries.

  • A sponsor needed to deliver data so a go-/no-go decision could be made at a major internal meeting that couldn’t be rescheduled. The problems stemmed, in part, from a failure to communicate between a large, somewhat impersonal CRO and a small sponsor that was used to close personal relationships and more or less constant interaction with the team managing its studies. We picked up the project in March 2013 and completed data lock by the end of September.

  • A small oncology-focused biotech company presented such a challenge when seeking to contract out its data management services. This CRO combined outsourcing, insourcing, and geographic flexibility to devise a hybrid solution.

  • What do you do when patients are too embarrassed to talk about what’s wrong with them? Recruiting “hidden” patient groups for rare disease studies is always challenging, and flexibility is the only constant.

  • A sponsor needed to test a new nonsteroidal anti-inflammatory drug (NSAID). Working with an experienced CRO allowed them to line up the 40 best sites in the country for this type of analgesia study and have the best training and tools at each site.

  • The placebo response is a real psychological, physiological, and ultimately statistical phenomenon that can be a powerful therapeutic tool in the world of medicine, especially when it comes to chronic pain conditions. Unfortunately, placebo response rates and effect sizes have increased considerably over the last few decades. Consequently, successful clinical trials must be able to accurately measure and mitigate placebo responses. But how do researchers quantify such a diverse range of phenomena?

  • The placebo effect is broader than just patient improvement in response to this inactive treatment; it encompasses the patient’s response to the entire therapeutic context in which treatment is administered. In this eBook, we examine issues surrounding the placebo response and its rise in more detail.

  • There is longstanding debate on the use of placebo control in clinical trials. This white paper reviews the regulatory, ethical, cultural, and even financial considerations surrounding the issue of placebo control in analgesia clinical trials.

  • Two large, parallel trials were being conducted with 107 sites spread across the United States for a topical drug to treat rosacea. They were challenged to come up with an advertising strategy to needed to attract more than 1,400 subjects fast enough to meet the aggressive schedule.

  • Cases of rosacea are traditionally classified into three different subtypes. This blog describes how to determine the different subtypes and how to treat them.

  • Telehealth (or e-health) solutions are providing increased access to care, making it easier not only for patients to receive care, but also for healthcare professionals to deliver that care. Advancements in medical technology that enable the capture and transmission of high-definition digital images have opened the door for dermatology e-health programs, and these same technologies have the potential to radically change dermatology trial designs.

  • Advancements in medical technology have opened the door for dermatology telehealth programs, and these same technologies have the potential to radically change dermatology trial designs. Here are four ways digital disruption in the dermatology field is benefiting both patients and providers.

  • With a customer racing to beat a competitor to registration with last patient out to database lock in two weeks, this CRO was able to step in and take charge and the study was completed on schedule.

  • Actively seeking the involvement of pediatric patients and their parents throughout the life cycle of a clinical trial can help drive recruitment, retention, patient satisfaction, and, ultimately, the success of the study. In this white paper, we discuss key factors that influence participation in pediatric clinical trials. We also present a case study on a first-of-its-kind survey designed to solicit feedback from children and their families on strategies for encouraging clinical trial participation.

  • The cause of Parkinson’s remains a mystery, and the dopamine promoter levodopa — notwithstanding its limitations — has been the principal treatment for about half a century. But researchers are making headway in discovering potential mechanisms of disease modification. In this blog we provide background on Parkinson’s, identify some of today’s open questions in clinical research, and take a first look at disease modification strategies.

  • There is a prevalence of associated symptoms with schizophrenia that antipsychotic drugs don’t treat. Managing these symptoms is essential to improving patient outcomes but remains an elusive goal, even after decades of progress. Read how effectively treating the core symptoms with long-acting drugs represents an effective strategy for addressing associated symptoms of schizophrenia.

  • Despite intensive research, nearly 15 years have passed since the last new Alzheimer’s disease medication was approved. Understanding the obstacles inherent in Alzheimer’s clinical trials, from high screen failure rates to lengthy trial durations that are demanding for both patients and caregivers, can help sponsors plan for – and overcome – these challenges. This paper reviews the current global pipeline of Alzheimer’s trials and their geographic locations, describes innovations in trial design, and discusses considerations of optimal clinical trial processes, including preclinical patient populations, clinical assessments sensitive to the earliest disease-related changes, and biomarkers as outcomes of clinical trials.

  • There is a significant unmet need for medications for addictions, chronic, relapsing disorders that lead to biological and behavioral changes that can have harmful medical and psychological consequences. This paper presents an overview of the medications that have been developed for addictive disorders, the study endpoints that have been used for market approval, and the challenges companies may face when developing medications for addictive disorders.

  • Faced with an unusual situation with patients rushing to enroll almost as soon as word of the study got out, this CRO quickly recognized the issue and ramped up staffing, right-sized other locations in the study, and collaborated effectively with all parties involved to deliver conclusive, positive results eight months ahead of time.

  • Central nervous system (CNS) disorders represent a major medical challenge and a significant opportunity for therapeutic innovation. Sponsors who have a clear understanding of the regulatory landscape and neuropsychological testing modalities available are poised to address critical unmet needs for the millions of people living with CNS conditions.

  • There is a clear shift in dermatology drug development towards biologics, targeted treatments, and rare skin diseases and away from topicals and symptomatic treatments. Learn more about the number of indications that are in development.

  • In recent decades, clinical gene therapy trials have underpinned the research that has significantly contributed to advancements in the development of therapies for this and other diseases – and these trials can be traced back to a young girl named Ashanthi DeSilva.

  • The marketplace for orphan drugs is growing, and changes in the regulatory landscape are providing favorable conditions for collaboration in the area of drug development in rare diseases. Understanding the regulatory and operational nuances of orphan drug development can help sponsors position their promising compounds for clinical and commercial success.

  • Gene therapy is a hot topic in clinical research today — and for good reason! These technologies have the potential to treat — and in some cases even cure — a wide range of conditions, including rare genetic disorders that previously had no effective therapies. This blog gives a brief history of gene therapy and where we are today with vectors and delivery systems.

  • A pharma company developing a drug to treat urea cycle disorder struggled with a CRO that was not effectively managing the study data. As trouble mounted, the CRO quit, stranding the project at a critical point in the development cycle. Premier Research was able to step in and successfully take over the data management and statistics portion of the trial to keep the trial on track.

  • A client required a chart review to evaluate survivability of pediatric patients with a rare metabolic bone disorder. By trying different protocols and overcoming complex issues of informed consent that had to be resolved before the study was successfully completed, they were able to successfully deliver a final clinical study.

  • Unfortunately, not all oncology trials succeed. In fact, the phase success and likelihood of approval (LOA) rates for oncology are the lowest across major therapeutic areas. Although there are many reasons for these relatively poor success rates, issues determining dose, schedule, and regimen in early phase trials are among the most prominent.

  • Selecting a safe starting dose must be balanced against the proportion of patients treated at sub-therapeutic doses. This is especially important for agents that demonstrate minimal toxicity in preclinical testing or for drugs that are unlikely to ever reach maximum tolerated dose. This approach has the potential to reduce the number of dose escalations while preventing patients from being treated at overly toxic doses that lack incremental biological activity.