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 155 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


  • Early-Phase Oncology – Emerging From The Crisis & Innovating For The Future

    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.

  • Minimizing Early-Stage Oncology Failures With Adaptive Design Approaches

    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.

  • Considerations For Applying Adaptive Design Approaches To Early Oncology Studies

    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.

  • Adaptive Trial Designs In Early Oncology: Minimizing Risk & Accelerating Timelines

    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.

  • Long-Term Follow-Up In Gene Therapy Trials: Ensuring Patient Engagement & Regulatory Compliance

    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.

  • Creating A Compliant eCTD: Avoiding Common Regulatory And Technical Pitfalls On The Path To Submission

    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.

  • Addressing A Critical Unmet Need: Considerations For Designing And Conducting Neonatal Clinical Trials

    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.

  • Oncology Trials In The Time Of COVID-19: Expert Insights

    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.

  • PREMIER VOICES #12: Direct-to-Patient – Is This The ‘New Normal’ For Clinical Trials?

    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.

  • Real-World Data And Real-World Evidence: What Is Their Value As A Synthetic Control Arm?

    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.

  • Managing Pandemic-Related Protocol Deviations: Tips For Clinical Trial Conduct

    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.

  • Statistical Considerations In The Wake Of COVID-19

    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.

  • Delivering The Global Expertise Required To Coordinate A Pediatric Gene Therapy Trial

    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.

  • iPSP Considerations For Molecularly Targeted Cancer Drugs

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

  • Data Driven: How Electronic Patient-Reported Outcomes (ePROs) Provide A Vital Opportunity To Capture Key Data Beyond The COVID-19 Crisis

    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.

  • How Ramping Up Communications Helped Overcome A Next-To-Impossible Recruitment Challenge

    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.

  • The State Of Cell Therapies In Dermatology

    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.

  • Direct-To-Patient Model Keeps Research Moving And Patients Safe

    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.

  • Snapshot: A New Guidance Document Program From The FDA

    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.

  • Understanding New FDA Guidance For Pediatric Oncology Studies

    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.

  • ICH GCP E6 (R2): A Primer For Small Biotech And Specialty Pharma Companies

    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.

  • Updates To The Guideline For Good Clinical Practice: Quick Review

    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

  • ICH GCP Update: Sponsor Control Of Clinical Trial Data

    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.

  • FDA Issues Guidance On Clinical Trials During COVID-19

    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.

  • Global Health Authorities Push For Rapid Development Of COVID-19 Tests, Drugs And Vaccines

    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.

  • What Is FDA’s Emergency Use Authorization (EUA) And How Can It Help Develop Therapies Faster?

    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.

  • Considerations for Ensuring Trial Continuity in the Wake of COVID-19

    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.

  • Why Forecasting Trial Costs Is So Hard (And Getting Harder)

    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.

  • Learning To Listen: Voice Of The Patient Grows Louder In Product Development

    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.

  • Study Start-Up: Why You Really Need To Get This Right

    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.

  • Why And How Global Healthcare Product Regulators Are Joining Forces

    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.

  • Current Challenges (And Solutions) In Global Regulatory Harmonization

    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.

  • From KOLs To Patient Advocates, Using Every Tool To Fight A Rare Disease

    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.

  • Understanding Recent Updates To The Classification Of Rosacea

    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.

  • Conducting Clinical Trials In The Asia Pacific Region: Opportunities And Obstacles

    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.

  • INTERACT Meetings: Streamlining Early Clinical Development

    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.

  • Cleaning Up Patient Data … Quickly

    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.

  • Getting Creative: A Hybrid Answer When The Conventional Won’t Do

    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.

  • A High Unmet Need That No One Wanted To Talk About

    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.

  • Clinical Trial Start To Finish …. That Was Easy

    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.

  • Just How Big Is The Placebo Problem?

    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?

  • Analgesia eBook: The Placebo Problem

    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.

  • Developing Analgesia Studies: Use Of Placebo Control In The EU

    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.

  • CRO Clears Path For Success For Rosacea Trial With Enrollment Hurdles

    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.

  • Current Rosacea Treatment Algorithms: Guide To Subtypes And Existing Therapies

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

  • The Role Of E-Health Technologies In Clinical Care And Research

    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.

  • Four Things You Need To Know About eHealth And The Dermatology Field

    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.

  • Getting Every Detail Right – With No Time To Think About How

    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.

  • Involving Children, Adolescents, And Parents In Pediatric Drug Development

    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.

  • Finding The Path To Disease Modification In Parkinson’s Disease

    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.

  • Making The Case For Long-Acting Antipsychotic Treatment

    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.

  • A Vast Unmet Need: Challenges In Alzheimer’s Disease Clinical Trials

    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.

  • Challenges In Medication Development For Addictions

    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.

  • Dealing With the Unexpected: Surprisingly Fast Clinical Trial Enrollment

    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.

  • CNS Trials: Challenges and Opportunities for Neuropsychological Testing

    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.

  • From Psoriasis To Behçet’s: The Evolution Of Biologics In Dermatology

    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.

  • Ashanthi DeSilva’s Story: A Look Back At The First Gene Therapy Trial

    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.

  • Mapping The New Landscape Of Orphan Drug Development

    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 101: From The 1960s To Today

    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.

  • Back On Track: Data Rescue On Pediatric Rare Disease Trial

    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.

  • The Case Of The Elusive Protocol

    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.

  • 6 Reasons Why Early Oncology Drug Trials Fail (And How To Avoid Them)

    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.

  • Key Considerations When Designing A Phase 1 Oncology Trial

    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.

  • 5 Reasons To Consider APAC For Clinical Trials

    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.

  • Considerations In Conducting Pediatric Clinical Trials

    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.

  • Establishing Risk-Based Monitoring Within A Quality-Based System As “Best Practice” For Clinical Studies

    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.

  • Considerations For Operationalizing Pediatric Oncology Studies

    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.

  • Unlocking The Full Potential Of Precision Medicine In 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.

  • Risk Based Quality Management (RBQM) - A Collaborative Approach To Holistic Clinical Trial Oversight

    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.

  • Understanding Recent Regulatory Changes For Pediatric Oncology Trials

    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 Renews Sponsorship For World Orphan Drug Congress Europe

    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."

  • Parkinson’s: Why Has Disease Modification Failed — And What Now?

    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.

  • Regulatory Oversight On Gene Therapy In The U.S. And EU

    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.

  • Planning A Gene Therapy Trial? Ask An Expert — And Pay Attention

    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.

  • Rare Disease: Operationalizing Gene Therapy Trials

    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.

  • Principles Of CAR T Cell Therapy And The Pathway To Regulatory Approval

    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.

  • Registry And Natural History Studies: Vital, Contrasting Roles In Rare Disease Clinical Research

    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.

  • Complete Response: Lymphoma Trial A Success For Five Patients

    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.

  • A Primer On Cancer Immunotherapy

    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.

  • A Brief Introduction To CAR T-Cell Therapy

    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.

  • Six Issues To Consider For Rare Disease Pre-IND Meetings

    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.

  • 5 Operational Considerations For Rare Disease Trials

    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.

  • The FDA’s New Guidance On Natural History Studies In Rare Diseases: What You Need To Know

    In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases that addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack of natural history data to guide the design of successful clinical trials. Read more to learn about the important role natural history studies play in drug development and their benefits.

  • Data Protection Part 1: Understanding How GDPR Affects Clinical Trials

    Since it took effect May 25, 2018, the European Union’s General Data Protection Regulation has been reshaping the way data is handled across every industry sector, including clinical research. GDPR contains a number of articles that present unique challenges to the pharmaceutical clinical trials industry. In this blog series, we focus on defining the regulation, on key aspects of GDPR that are relevant to clinical trial professionals, and on providing insight on how CROs can achieve compliance in data handling throughout the clinical trial process.

  • Investigational New Drug Applications: Five Solutions To Common Problems

    There are many pitfalls that come with preparing an Investigational New Drug (IND) application for FDA submission, but with the right plan – and the right partner – there are also plenty of ways to avoid them. Here are five actionable steps can help ensure your next IND is a successful one:

  • Investigational New Drug Applications: Four Common Mistakes

    Before starting Phase I trials, an Investigational New Drug (IND) application must be approved by the FDA. This critical early step in clinical trial development grants an exemption to laws prohibiting the transportation of drugs across state lines prior to market approval. Here are the most common errors we’ve seen made with IND submissions.

  • When Other CROs Said No, We Said Yes — To The Nearly Impossible

    The sponsor was finishing its Phase III study for an antimicrobial, anti-infective drug and needed to complete its NDA submission in just 6 months. The development effort went back more than a dozen years, and having performed none of the clinical studies on the drug. This CRO took the unusual step of negotiating a rolling submission, providing by the due date enough information for the agency to begin its review, and filling in the rest over the following weeks.

  • Improving Regulatory And Operational Performance In Orphan Drug Development

    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.

  • Untapped Potential: Asia-Pacific’s Growing Role In Biotech Drug Research

    For all its demographic promise, the Asia-Pacific (APAC) region has been slow to emerge as a center for US and EU biotech clinical drug research. Patient access in this region is now a strong motivator. Read further observations about the future of the APAC region in biotech drug research.

  • Recent FDA Updates And Guidance For Rare Disease Drug Development Webinar

    In January 2019, the US Food and Drug Administration (FDA) updated its 2015 draft guidelines for drug discovery in rare diseases. The update, Rare Diseases: Common Issues in Drug Development, seeks to help pharmaceutical companies and other sponsors perform more efficient development programs for drugs and biological products and provides new insights on complex elements of the development process. In this webinar, learn about the core elements of the new guidance plus the latest recommendations and instructions from the agency along with practical examples and applications.

  • CAR T Cell Therapy And Other Technologies Emerging In Oncology Research

    Some unique CAR T cell therapies have already been approved by the US Food and Drug Administration (FDA) and are the first steps toward further groundbreaking science. In this webinar, Emile Youssef, Premier Research’s Executive Medical Director, reviews the operational and clinical pathways that are helping to bring these technologies to patients.

  • Boosting Immuno-Oncology’s Effectiveness Against Cancer

    The current limitations on the use of immunotherapy drugs could be swept away thanks to the development of immune checkpoint inhibitors. They block disruptive proteins that limit the body’s natural immune response and stop T-Cells from destroying cancer cells. Some of the biggest factors stopping the more widespread use of these drugs are the limitations of current standards in effectively measuring how well they work. Read how improvements in the methods used for immuno-oncology drug trials could benefit both researchers and patients.

  • Premier Research Champions Patient Advocacy Community

    As part of its commitment to supporting the patient advocacy community and as a kickoff to Rare Disease Day on February 28 and March as Rare Disease Month, clinical research company Premier Research is announcing both a new scholarship for rare disease patient advocates in partnership with Professional Patient Advocates in Life Sciences (PPALS) and the launch of a new Patient and Stakeholder Engagement (PASE) capability led by Juliet Moritz.

  • The Placebo Problem In Pain Research Keeping Up With The ‘Mrs Joneses’

    Pain trials are uniquely challenging because they rely so heavily on patients’ own assessments. While researchers will always need to account for the placebo effect when studying indications as subjective as neuropathic pain, there are ways to increase the likelihood of achieving objective trial outcomes.

  • It’s About How The Social System Works, Not Just The Science

    After a failed attempt from their first CRO, this manufacturer turned to Premier Research to successfully recruit and retain 24 patients for a Phase I proof-of-concept study of inflammatory bowel disease.

  • A Strategic Partner, Or A Vendor Looking To You For The Answers?

    Any capable CRO can provide manpower and execute orders on behalf of a sponsor, no questions asked, with little stake or interest in the endeavor’s ultimate success. What you need is a strategic ally, a company that joins you, lockstep, in pursuing shared goals and greater outcomes. Not a vendor, but a partner.

  • 6 Early Phase Dose-Finding Trial Designs For Oncology Therapeutics

    The optimal dose-finding strategy for a given therapeutic agent and indication is based on a multitude of factors, accentuating the need for individual tailoring in oncology drug trial design. Read on for a look at six of the most common dose-finding trial designs in oncology today.

  • Ethical Considerations In Adaptive Design Clinical Trials

    While adaptive design is associated with many potential benefits, it may also present challenges to observing the basic ethical principles of research in human subjects. In this white paper, we review the features of particular clinical trial design adaptations and discuss the ethical obstacles they can present and those they can potentially resolve. Using examples of both published and unpublished clinical studies, we highlight the importance of proper design and planning and appropriate ethical due diligence in the successful conduct of an adaptive design clinical trial.

  • 5 Must-Dos To Build Trust Between An Early-Stage Biotech And CRO

    Take these steps to create a trusted partnership with your CRO and achieving a much more accurate and financially workable CRO agreement.

  • CAR-T Cell Therapies: Safety Considerations and Toxicity Management

    Along with demonstrated efficacy in hematologic malignancies, CAR-T cells have the capacity to elicit serious toxicities. Safety considerations related to CAR-T cells may impact both trial design and trial management, as the adverse events (AEs) associated with immuno-oncology agents differ from those associated with cytotoxic therapies. Learn how to make anticipating, preventing and managing toxicity a key component of clinical studies involving CAR-T cells.

  • 4 Potential Advantages Of Adaptive Design

    Adaptive design strategies have been especially useful for rare disease research in general and rare oncology in particular. Read more to see how the savvy application of adaptive trial design compares to conventional study designs.

  • 5 Endpoints For Rare Oncology Trials

    For patients battling rare cancers, especially those lacking standard treatments, they don’t have the time to wait for more concrete measures. In these cases, choosing the appropriate endpoint for a trial is crucial. Read on for a rundown of the five major types of clinical endpoints in rare oncology and the best way to use them.

  • From Laboratory To Bedside: Expediting Development Of Novel Rare Cancer Treatments

    This webinar shares new insights on rare oncology drug development and examines how new processes and regulatory pathways are helping speed development of novel therapies.

  • Driving Product Development And Finding The Fast Track In Early-Phase Oncology Programs

    This webinar highlights the many aspects a company must consider in planning and executing a trial, and how a CRO can help throughout the process.

  • Immuno-Gene Therapeutics In Hematological Cancers: How Science Drives Study Strategy

    This webinar reviews the current state of technology in gene therapeutics and examines trends in immuno-oncology and gene therapy for hematological malignancies.

  • The Complex Issues With Developing A Novel Human Antibody From Scratch

    Having the right specialists on their team enables Premier Research to recruit subjects relatively quickly, despite extremely complex selection criteria for this multiple myeloma clinical trial. In addition, maintaining a close and cooperative customer relationship assisted in moving the study forward.

  • Overcoming The Challenge Of Enrolling Sickle Cell Patients In Clinical Trials

    Premier Research was given a tight timeline of less than four months from delivery of the final protocol to first patient in. With proper site selection to accommodate the needs of a fast start-up along with open and frequent communication with the principle investigator and the project team, they were able to meet this timeline.

  • Recruiting Hard-To-Find Patients From A Specific HLA Subgroup

    CRO helps company overcome early patient screening failure rate of 70 percent as well as assisting with instituting new protocols and renegotiating contracts as the study experiences and major change of direction.

  • Lengthy Cancer Drug Study Using Dozens Of Sites Yields Positive Results

    After lengthy recruiting process and a five year long clinical trial spread across more than two dozen sites, sponsor calls study results “an unexpectedly huge step forward” in overcoming the limited efficacy of current treatments and continues partnership with CRO.

  • CRO Helps To Identify A Highly Productive Location For Siting Future Studies

    Compromised data collection effectively stalled a clinical trial study of an inflammation control drug. With help from a CRO, important lessons were learned about the need for discipline and following protocol as well as the identification of Russia as a highly desirable site for clinical trials.

  • Five Things To Know When it Comes To iRECIST

    Here are five things you should know about iRECIST as the standard shapes up as an important tool in assessing solid tumors.

  • Maximizing Success In Early Stage Oncology Trials: Considerations And Strategies In The Era Of Molecularly Targeted Agents

    Making a meaningful impact on the survival and quality of life of patients with cancer remains a significant challenge. This white paper discusses considerations and strategies for maximizing the likelihood of success in early phase oncology trials and developing significantly improved therapeutics for patients.

  • Rapid Change, Real Promise: The Future Of Rare Oncology Research

    Insight on the major issues being raised in the rare oncology space today, including patient perspectives on rare cancer research, innovative trial designs, the regulatory landscape, and pending legislation that may impact how studies are conducted.

  • Six Dosing And Safety Considerations In The Era Of Emerging Therapies

    Key considerations for developing early-phase trials that can more accurately define the recommended dose and identify adverse events for emerging therapies.

  • Driving Product Development And Finding The Fast Track In Early-Phase Oncology Programs

    Explore the many aspects a company must consider in planning and executing an early-phase oncology trial of this hypothetical compound, from performing a regulatory gap analysis and developing a target product profile to identifying the right patients and selecting efficacy endpoints.

  • Opportunities Beyond Hope: Immuno-Oncology Drug Development

    Explore the role of the immune system in cancer development, as well as the history and challenges of developing immunotherapies for cancer.