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.


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.


Premier Research

One Park Drive

Durham, NC 27709


Phone: + 1 910 447 3156

Contact: Karen Brown


  • 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.

  • Section 505(b)(2) of the Food, Drug, and Cosmetic Act describes a 505(b)(2) new drug application (NDA) as an application where at least some of the information required for approval comes from studies not conducted by or for the applicant and for which the applicant has not obtained a right of reference.

  • Read the available case study to figure out the solution to how a biotech company could not afford the time and expense to conduct studies recommended by its consultant and agreed on by the FDA.

  • In 2020, CDER approved 68 NDAs that used the 505(b)(2) pathway, representing important advances in patient care across a wide range of therapeutic areas.

  • As the 505(b)(2) expert, Camargo, a division of Premier Research, is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Here is the final installment in the four part series of frequently asked questions (FAQs)

  • As the 505(b)(2) expert, Camargo is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Here is Part 3 in the series of frequently asked questions (FAQs).

  • As the 505(b)(2) expert, Camargo is frequently asked questions about how to get a product approved via the 505(b)(2) regulatory pathway and if this pathway is appropriate. Here is Part 2 in the series of frequently asked questions (FAQs).

  • The topic of this post will be general 505(b)(2) questions, including what is and is not allowed for an approval via the 505(b)(2) regulatory pathway.

  • In this paper, we will highlight those designation programs available specifically for products with rare disease indications: their criteria, timelines, maintenance, and benefits.

  • In this two-part blog series, we will explore the regulatory strategy considerations sponsors should bear in mind when working with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), with a focus on the development of drug and biologic products.

  • Development and Reproductive Toxicology studies, or DART studies, are required for most non-oncology programs between IND and NDA filings. This blog post discusses how their goal is to detect any effects of a drug within a complete reproductive cycle as relevant to humans: from initial conception to reproductive capacity in the next generation.

  • To help sponsors understand how the FDA uses the eCTD technical validation rules to verify conformance, the FDA developed the technical rejection criteria (TRC) for study data. Since its inception, the FDA monitored and analyzed conformance and revised the TRC but has not implemented them until now.

  • Read about the tools or making the Pre-IND meeting successful.

  • The 505(b)(2) pathway can yield significant benefits in drug development cost and time. But what are the differences in 505(b)(1) versus 505(b)(2)? They are not the same.

  • 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.

  • For developers seeking to obtain approval for previously approved drug products in the United States, the U.S. Food and Drug Administration (FDA) offers two abbreviated approval pathways — an abbreviated new drug application (ANDA) and a 505(b)(2) application. Read about these in the available blog.

  • 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.

  • Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life. Read how that’s changed with the recent publication of a new draft guidance from the U.S Food and Drug Administration’s (FDA) Oncology Center of Excellence (OCE), Core Patient-Reported Outcomes in Cancer Clinical Trials.

  • 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. Faced with limited treatment options, researchers, clinicians, and patients may be seeking approaches to accelerate the development and approval of novel therapies. In this blog post, we review 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 evaluated both non-disease-modifying and disease-modifying transgenes for Parkinson’s disease (PD) treatment, with encouraging results. 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 – potentially as long as 15 years – 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 learn how the Study Start-Up application delivers fast, efficient, accurate trial management and superior study quality from project start-up to project closure.

  • 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. 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.

  • 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.

  • n 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.

  • Drug development is a resource-intensive endeavor. Seeking input from the U.S. Food and Drug Administration throughout the journey can help optimize those resources and maximize the likelihood of regulatory approval. When preparing to submit a new drug application (NDA), a pre-NDA meeting with the FDA can be a critical step in ensuring the submission of a well-organized NDA that fits the expectations of agency reviewers.

  • 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.

  • As our understanding of the molecular characteristics of tumors has improved, there has been a tremendous leap forward — not only in targeted therapies but also in the development of tumor-or tissue-agnostic treatments. Now, with precision or personalized medicine, treatments can be targeted to a subgroup of patients rather than relying on a one-drug-fits-all model. In this blog post, we explore the changing rare oncology landscape and novel approaches to rare cancer trials.

  • 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.

  • A clinical hold from the U.S. Food and Drug Administration can significantly prolong the time and increase the cost of drug development, which is particularly concerning for emerging/small biotech and specialty pharma companies. In this blog, we discuss common reasons for clinical holds and provide useful tips for both avoiding and addressing them.

  • 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.

  • Failure to comply with the regulatory and technical requirements when creating your eCTD could jeopardize the success of your submission and your product development plan as a whole, especially for early-stage biotechs working with limited resources. Here are strategies for avoiding some of the most common electronic publishing pitfalls on the path to submission.

  • 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.

  • The Research to Accelerate Cures and Equity (RACE) for Children Act requires that any original new drug or biologics license application submitted on or after August 18, 2020, for a new active ingredient must contain reports of molecularly targeted pediatric cancer investigations if the drug is directed at a molecular target that the FDA determines to be substantially relevant to the growth or progression of a pediatric cancer. Learn more about the changes in this article.

  • 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.

  • Earlier this week, the FDA issued a guidance on the conduct of clinical trials during the ongoing Coronavirus Disease 2019 (COVID-19) pandemic. In line with the challenges created by the COVID-19 pandemic, the FDA provided some recommendations with regard to the conduct, monitoring, and data aspects of ongoing and future clinical trials. We’ve listed some of the key considerations for sponsors below. For more information, consult your project manager or contact Premier Research’s COVID-19 Task Force.

  • 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.

  • Read how the INTERACT meeting can be an invaluable engagement with the agency to inform the successful planning and execution of a novel product development program.

  • 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.

  • Small and mid-size biopharma companies in the U.S. and Europe are under intense pressure to find more efficient and cost-effective ways to commercialize their products. In an increasingly competitive clinical trial environment, sponsors have begun to shift their focus to the Asia-Pacific (APAC) region for their studies.Here are five reasons why the APAC region may be a good fit for your global clinical trial.

  • There are many challenges inherent in pediatric clinical studies, where the traditional paradigms for evaluating pharmaceutical agents in adults often do not apply. These occur on ethical, physiological, pharmacometrics and economic grounds, among many others. It’s important to recognize that while these efforts are challenging, they are not impossible — and they may be better suited to creative solutions that center on documenting outcomes within these patient populations. In this webinar, the featured speakers discuss challenges in implementing clinical studies in children and creative solutions to effectively implement them.

  • This report based on a survey of ACRO members reveals that Risk-Based Monitoring (RBM) makes clinical trial quality review more efficient and effective. It found that when a company reviews data through a centralized system using the RBM model, CROs and technology companies are better able to detect quality issues earlier and make rapid corrections at the site level. This type of approach is now central to ensuring the safety of patients in clinical trials, and is expected to continue to grow in importance as clinical trials becomes more numerous and complex.

  • Remarkable progress has been made in our understanding of the genomics of pediatric cancers, and these advancements have led to the recognition that products being studied for use in adult cancer indications may have health benefits for pediatric patients. By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research to Accelerate Cures and Equity (RACE) for Children Act has the potential to accelerate the development of novel treatment options for children with cancer. Read more how RACE and the FDA guidance will affect the future of pediatric oncology.

  • Precision oncology promises a new standard of care where therapies are tailored to the molecular profile of a specific tumor. For the full potential of precision medicine to be realized, regulatory, technical, clinical, and economic frameworks will need to evolve to the nuances of these novel treatments.

  • Developing, executing, and overseeing clinical trials is a complex process. This paper shares perspectives that will assist sponsor organizations in the creation of a RBQM system in partnership with their CROs and vendors. When a shared, proactive plan is established, sponsors and CROs/vendors can then tailor their oversight strategy to support improved quality and safety of clinical trial execution.

  • While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for innovative therapies for pediatric cancers is becoming increasingly vocal.

  • Premier Research is proud to once again be a sponsor of the World Orphan Drug Congress (WODC) Europe taking place November 12-14, 2019, in Barcelona, Spain. As part of our ongoing support for this innovative conference, Jonathan Kornstein, Executive Director, Program Strategy, Rare Disease & Pediatrics, will present a session entitled "A Partnership That Lasts: Key Considerations When Working With CROs on Rare Disease Studies."

  • Researchers have made many attempts at disease modification as they pursue breakthroughs in treating Parkinson’s disease, but so far without success. Why have these efforts failed, and what’s next in treating this degenerative disorder that affects an estimated 10 million people worldwide? In this blog post we examine these past attempts, why they failed, and what targets are currently being pursued.

  • Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. With the rapid evolution of the gene therapy field, regulatory agencies have been working to keep pace with these scientific and clinical breakthroughs. An understanding of the regulations and guidance documents reflecting regulator current thinking surrounding gene therapies is essential to success.

  • Whether the experts come from a CRO or a regulatory consultancy, their help will be critical in helping develop the trial, meet with the Institutional Biosafety Committee, establish a timeline, and manage expectations. Here’s why.

  • Even measured against the vast scientific mystery that defines the biotech industry, gene therapy poses extraordinary challenges. This paper explores the history of gene therapy trials, as well as the types of gene therapy vectors and delivery strategies. Also discussed is the regulatory and operational challenges associated with gene therapy trials, including start-up regulations, site selection, recruitment, and retention.

  • Advances in immuno-oncology have led to the advent of Chimeric Antigen Receptor T (CAR T) cell therapy, which combines a patient’s own T cells with engineered T cell receptors known as “CARs”. The CAR enables the final product to produce chemicals in the hopes that the “enhanced” product or cells will bind to the cancer cells and kill them. In this free webinar, learn about the principles of CAR T cell therapy and the ways these technologies can reach patients.

  • Both registry studies and natural history studies play important roles in rare disease research. Understanding the differences between the two types of studies and how they can be used to inform clinical development can help sponsors plan for success.

  • A trans-Atlantic study to evaluate an antibody for treatment of B-cell non-Hodgkins lymphoma overcame patient recruiting challenges and has already succeeded beyond expectations in the form of five patients declared disease-free a year and counting while still three years from completing patient follow-up.

  • Widespread use of immune checkpoint therapy to treat cancers is hampered by unpredictable response rates and immune-related adverse events. To address these challenges, combination therapies are increasingly being studied as a strategy for improving response and overcoming resistance. In this post, we provide an introduction to cancer immunotherapy, exploring its immunological basis and the fundamental principles guiding development of new treatments.

  • Careful planning is important for all early drug development programs, but it is particularly critical in rare diseases where study populations are limited and precedents for drug development are lacking. By proactively preparing for this meeting, sponsors can set themselves up for productive discussions which may help in identifying areas of regulatory flexibility.

  • From researchers to sponsors to patients to advocacy groups to clinicians, in rare disease research all people involved must be utilized to their greatest potential for a study to be successful. Learn more about the operational challenges within rare disease research and how to overcome them.