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PharPoint Video Series: How and When to Consider a Data Monitoring Committee


Have you heard the following acronyms: DMC, DSMB, DSMC, IDMC and wondered what they stand for? In this video, PharPoint Biostatistician Alison Miller and Senior Director of Consulting Operations Amy Flynt provide you with information that can help you answer this question and many more.

  • How and When to Consider a Data Monitoring Committee Transcript

    “Have you heard the following acronyms: DMC, DSMB, DSMC, IDMC and wondered what they stand for? Or have you ever wondered what is the difference between a DMC and an IDMC? Have you ever wondered if you needed one of these committees for your study and how you should go about forming that committee? Today, we are going to provide you with information that can help you answer these questions.

    These acronyms are all different terms used to describe the same thing; an independent group of experts who monitor patient safety and may also monitor treatment efficacy data while a clinical trial is ongoing.

    This group advises the sponsor regarding the continuing safety of trial subjects and those yet to be recruited into the trial, as well as the continuing validity and scientific merit of the trial. Here is what the acronyms mean:

    • DMC = Data Monitoring Committee
    • DSMB = Data Safety Monitoring Board
    • DSMC = Data and Safety Monitoring Committee
    • IDMC = Independent Data Monitoring Committee

    For consistency purposes we will refer to this group of experts as a DMC for the rest of the discussion today, but any of the previously mentioned acronyms would be acceptable.

    Reasons for a DMC:
    The fundamental reasons to establish a DMC is to enhance the safety of trial participants and if regular interim analyses of the accumulated data are needed. When determining if you need to have a DMC, consider if your study meets any of the following criteria:

    • Large, randomized, multisite studies
    • Controlled trial that compares rates of mortality or major morbidity
    • Known safety concerns regarding adverse events, a specific laboratory parameter for the product, or serious toxicity with the study treatment
    • If there are a priori reasons for a safety concern due to the invasive administering of the study treatment
    • An adaptive design with a formal interim analysis planned
    • If the study endpoint is such that a highly favorable or unfavorable result, or even a finding of futility at an interim analysis might ethically require termination of the study before the planned completion
    • Study being performed in a fragile population such as children, the elderly, pregnant women, or other vulnerable populations such as terminally ill or diminished mental capacity
    • If the trial population is at an elevated risk of more severe outcomes even when the study objective addresses a lesser endpoint
    You would not need a DMC for the following:
    • Trials at early stages of product development
    • If the trial addresses outcomes such as relief of symptoms or something similar that is of less concern than mortality and morbidity

    Selection Process/Forming the Committee:
    The selection of the DMC members is extremely important since the DMC responsibilities relate to the safety of trial participants. The committee should be made up of a minimum of 3 people:

    • A medical doctor who specializes in the area being studied
    • A medical doctor who either specializes in clinical trials or in the area being studied
    • And a statistician with knowledge of statistical methods for clinical trials and sequential analysis of trial data
    PharPoint recommends an odd number of voting members to ensure that the vote does not end in a tie. The committee will also usually have a non-voting statistician who serves as the unblinded DMC support statistician.

    Consider prior DMC experience when selecting your members. Prior DMC experience for the statistician is particularly important if there is only one statistician serving on the DMC. The other main concern to consider when selecting your members is an evaluation of their potential conflicts of interest.
    These could be financial conflicts, study conduct conflicts for investigators enrolling subjects in the trial in which their knowledge of interim results could influence their conduct of the trial, and intellectual conflicts for individuals known to have strong views on the relative merits of the interventions under the study and as such may not be able to review the data in an objective manner.

    Things to consider/Recommendations:
    We recommend that each DMC maintains a charter that becomes the governing document for that committee. This document needs to include well-defined standard operating procedures. Per the FDA Guidance: “Such charters are important for the same reason that study protocols and analytical plans are important—they document that procedures were pre-specified and thereby reduce concerns that operations inappropriately influenced by interim data could bias the trial results and interpretation.” The charter should include the following information at a minimum:

    • Schedule and format for meeting
    • Format for presentation of data
    • Information regarding who will have access to interim data and who may attend all or part of DMC meetings
    • Procedures for assessing conflict of interest of potential DMC members
    • The method and timing of providing interim reports to the DMC
    • Information regarding who is involved in DMC-related activities
    • Define a communication plan to establish who may directly communicate with the DMC (this is often only a DMC coordinator and/or an unblinded DMC support statistician)
    • Roles and responsibilities of the DMC members
    • Roles and responsibilities for other persons involved with DMC related activities (e.g. the sponsor, DMC coordinator, unblinded DMC support statistician)
    • Information regarding meeting minutes
    • Options for DMC recommendations

    Some people may tell you that you must use a different group to run the DMC than the group that does the analysis. This is not the case. What is required is that everything remains independent and the unblinding information is only shared with the approved personnel as stated in the charter. This can be achieved within one single organization if that organization has the necessary firewalls in place.

    In the first 10 years of operations at PharPoint, the biostatistics team has provided independent data review committee support for over 100 different protocols, most which involved multiple meetings. Our statisticians have served the role of both unblinded statistician and voting statistician for these meetings.

    If you are interested in hearing more about DMCs, you need support for a DMC, you need help determining if you need one for your study, or you need help recruiting members, please contact me at 919-433-2474. You can also reach me via email at”


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PharPoint & Medrio Video: Smart Designs for Data Collection with Adaptive Clinical Trials


Join our Director of Programming, Jayme Swinson, as he discusses smart designs for data collection with adaptive clinical trials using Medrio software.

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Beginnings Charity

Beginnings and PharPoint Research

PharPoint Gives BackEach month, PharPoint Research selects a non-profit to support through fund-raising and awareness building to aid in research and/or resource development. Also known as our Working Together for Change campaign, this company-wide initiative is dedicated to serving communities in need.  During the month of March, PharPoint is working to give back to Beginnings, a non-profit for parents of children who are deaf or hard of hearing that helps parents and families understand hearing loss and the diverse needs of children who are deaf or hard of hearing.

PharPoint Gives BackHearing loss is one of the most common birth defects. Nearly three out of every 1,000 children are born with a permanent hearing loss and another two or three in 1,000 will develop hearing loss later. By identifying a hearing loss early in a child’s life, families can help ensure age-appropriate development. Most children born with hearing loss have parents with typical hearing. A parent who has not previously been exposed to hearing loss may not understand the consequences for their child and their family.

Help and Hope

In the first few months after learning of a hearing loss, parents and families face an overwhelming number of choices. The decisions parents make will have an impact on their child’s emotional and language development. It is important for parents to understand the challenges of hearing loss, the resources available, and the many pathways to a happy and successful adulthood.

PharPoint Gives Back

Beginnings provides families with accurate and objective information so they can make the best decisions for their child and family.

Beginnings remains in contact with families as children transition from early intervention to preschool, and along the sometimes confusing paths through elementary, middle and high schools. They are there to help parents at all times understand available options and to become their child’s best advocate.

Beginnings collaborates with state agencies and organizations and serves on state and national committees, representing parents of children with hearing loss. Every day in North Carolina, they meet with parents and professionals to improve children’s lives.

Free Services Offered

Beginnings provides free services to North Carolina residents who are:

  • Children – birth to age 22 – who are deaf or hard of hearing
  • Parents or caregivers of children who are deaf or hard of hearing
  • Professionals who serve these families


Beginnings helps parents understand:

  • Hearing test results
  • Communication strategies
  • Amplification options
  • Language and speech development
  • How to implement strategies for school success
  • Education law
  • Programs and resources available to their family


Get Involved

Interested in contributing to Beginnings to help support their programs? Your contribution to Beginnings will support education, training, publications and outreach for families of children who are who are deaf or hard of hearing and educators/professionals in the field of deafness.

You can also join Beginnings as they celebrate 30 Years of Guidance for New Journeys by attending the Annual Spring Gala and Auction. The event will be held on Wednesday, April 5, 2017 from 6:00pm-8:30pm at The Umstead Hotel in Cary, NC.  Tickets can be purchased online at or by calling (800) 541-4327.

For another chance to give back, join Beginnings at the Celebrate Sound Charity Walk in Raleigh as they walk with the Sertoma Club of Raleigh and the Capital City Sertoma Club on March 25, 2017 to raise awareness and funding. These clubs have made it possible for Beginnings to help families in the area pay for items not covered by insurance, such as ear molds, batteries, and the cost of traveling to doctor appointments.

Have you or someone you know been helped through the services of Beginnings? Please share your experience in the comments below.

The Conservation Fund and PharPoint Research

The Conservation Fund

The Conservation Fund and PharPoint Research

Each month, PharPoint Research hand-selects a non-profit to support through fund-raising and awareness building to aid in research and/or resource development. Also known as our Working Together for Change campaign, this company-wide initiative is dedicated to serving communities in need. Therefore, we are excited to announce The Conservation Fund as January’s selected non-profit for the Working Together for Change campaign.

About The Conservation Fund

The Conservation Fund is a non-profit that pursues environmental preservation and economic development in the United States. Since its The Conservation Fund and PharPoint Research

inception in 1985, the organization has protected more than 7 million acres of land and water in all 50 states. Most relevant areas include parks, historic battlefields, and wild areas. In addition, The Conservation Fund works with community and governmental leaders, businesses, landowners, conservation non-profits, and other partners.

Primary Programs

In addition, The Conservation Fund operates nine programs to support its mission.

  1. Conservation Acquisition: Protecting land and water that also have high conservation value.
  2. Conservation Leadership Network: Developing networks of shared knowledge and technical skills for on-the-ground conservation solutions. Most relevant initiatives include collaborative solutions between communities and corporations.
  3. Freshwater Institute: Conducting research, development, and training in water resource management. This includes developing technology and providing engineering services for aquaculture systems.
  4. Land Conservation Loans: Providing bridge financing and real estate expertise to land trusts.The Conservation Fund and PharPoint Research
  5. Mitigation Solutions: Achieving important conservation results by protecting high-priority wildlife habitat, clean water, and recreation areas.
  6. Natural Capital Investment Fund:  Helping innovative entrepreneurs build locally-owned enterprises. Most noteworthy, this creates lasting jobs and community wealth, while using natural resources responsibly.
  7. Resourceful Communities: Assisting under-served communities protect and restore natural resources.
  8. Strategic Conservation Planning: Offering solutions that government leaders, conservationists, and others need to create systemic and lasting change in major cities, watersheds, and multi-state regions.
  9. Working Forest Fund: Acquiring and permanently protecting ecologically significant forestland. Rather, this supports forests’ role in providing clean air and water, wildlife habitat, and economic benefits for communities across America.

Get Involved

Interested in contributing to The Conservation Fund to help support their programs? There are many opportunities to suit your time and resources:

Donate: A one-time gift or monthly contributions help The Conservation Fund to conserve high priority lands.

Partnerships: The Conservation Fund partners with businesses, state and local governments, and private landowners. As a result, this helps advance social responsibility and sustainability priorities.

Join the Online Community: Stay up-to-date with the latest news and events, manage donations, and communicate with The Conservation Fund.

In conclusion, have you or someone you know worked with, or benefited from The Conservation Fund’s work? We invite you to share your experience in the comments below.

PharPoint Research and Medrio Expand Partnership

PharPoint Research Expands Collaboration with Medrio amid Recent Growth

PharPoint Research Expands Collaboration with Medrio amid Recent Growth

SAN FRANCISCO, CA–(Marketwired – December 14, 2016) – PharPoint Research, a North Carolina-based Contract Research Organization (CRO), has committed to a contractual expansion in the number of clinical trials they will conduct with Medrio, a Software as a Service (SaaS) company providing electronic data capture (EDC) services to clinical trials. This agreement is a direct result of the growth of PharPoint Research, as evidenced by their recent Fast 50 award by the Triangle Business Journal.

The commitment between the two companies strengthens their partnership and reflects the value that PharPoint Research has placed in Medrio’s services during this period of rapid growth. PharPoint Research views Medrio as one of the top EDCs for quick study build and high data quality, and therefore as conducive to the efficiency that PharPoint Research will require as it continues to emerge as an industry leader and one of the busiest CROs in the region. “With our growth, we continue to utilize Medrio as our primary tool for data capture,” said Moya Hawkins, Director of Data Management at PharPoint Research. “Medrio helps us to facilitate a streamlined and rapid approach to study start-up while maintaining clean data throughout a study’s lifecycle.”

PharPoint Research and Medrio have enjoyed a mutually productive relationship for many years, beginning in 2010 when PharPoint put their first project in Medrio’s EDC system. In 2013, PharPoint Research joined Medrio’s Partner Program, through which Medrio provides extensive sales and marketing support, offers previews of upcoming product developments, facilitates new business opportunities for participating CROs, and more. PharPoint Research has found the Partner Program to be a valuable resource throughout the journey toward its current growth, and continues to take advantage of the program’s benefits.

Of the increased use of Medrio by PharPoint Research in the coming year, Mike Novotny, founder and CEO of Medrio, said, “Nothing could be more rewarding than knowing that PharPoint Research has found the capabilities of our software, and the benefits of our partner program, to be so helpful during their explosive growth. We’re honored that they’ve chosen to expand their business with us over the next year.”

About PharPoint Research

PharPoint Research is a full-service contract research organization (CRO) that specializes in Project Management, Clinical Monitoring, Data Management and Biostatistics. The company is uniquely poised to serve the needs of pharmaceutical, biotechnology, and medical device development organizations. PharPoint Research is experienced in strategic consulting engagements and Phase 1 through Phase 4 clinical research projects across a variety of therapeutic areas, including infectious disease, oncology, CNS, cardiovascular, and rare disease indications. PharPoint Research is headquartered in North Carolina, with strategic operations service provider partners around the world, including North and South America, Europe, and Asia-Pacific. For more information, please visit

About the Fast 50 Award

The Triangle Business Journal, a weekly business newspaper, is the leading provider of local business news for the Raleigh-Durham–Chapel Hill area of North Carolina. Each year the Triangle Business Journal honors the fastest growing companies in the Research Triangle area of North Carolina with the Fast 50 Awards. PharPoint Research received the award in recognition of the significant growth that the company has experienced over the last 12 months. This growth is the direct result of providing excellent client service, expertise and uncompromised quality to the pharmaceutical and biotechnology industry. This is the fourth time since 2011 that PharPoint Research has won this annual award.

About Medrio

Medrio offers an integrated eClinical software platform with a fully hosted EDC system. Our technology features an easy-to-use drag-and-drop interface, allowing study managers to build their studies in days instead of months without requiring any programming. Medrio’s new mobile suite, mSource, supports both event-based and subject-based workflows, offline data entry, and Patient Reported Outcomes (PRO). Although Medrio has extensive experience in all study phases, our m1 application leads the way in early-phase and Phase 1 trials, with over 500 Phase 1 trials to date. Medrio costs up to 75% less than traditional EDC solutions. For more information, please visit our website. To schedule a demo and learn more about Medrio’s Partner Program, please submit your information here and a member of our team will be in touch shortly.


PharPoint Research
Tim LaCroix

Michelle Wilcox

Orphan Drug Development Patient Centricity

Orphan Drug Trial Solutions


Orphan Drug Development and PharPoint Research


An orphan drug is a product intended for the treatment, diagnosis, or prevention of a rare disease. A rare disease also affects fewer than 200,000 people in the United States.[1]

Although orphan drugs treat only a small number of patients, orphan drug development shows a large return on investment for potential Sponsors. For instance, companies researching solutions for rare diseases have little competition from branded and generic alternatives. Companies also have the opportunity to carve out a specialized orphan market that no other company is actively pursuing. In addition, the FDA offers a fast-tracked regulatory review process and lower-cost late-stage development to aid in the development of orphan drugs because they treat serious or life-threatening conditions.[2]

To reduce challenges associated with orphan drug development and improve the patient experience, Sponsors should take a patient centric approach to clinical trials.[3] Shifting a trial design to a patient centric approach for rare disease trials does not require an overhaul of current trial standards. However, it does mean that researchers must change their focus to see the trial through the eyes of patients and caregivers. Sponsors can reduce risk and increase effectiveness of orphan drug clinical trials as a result of making these changes in the study design.

History – The U.S. Orphan Drug Act

From 1967 to 1983, only 34 drugs were approved by the FDA to qualify as orphan drugs. The FDA recognized the lack of incentive forOrphan Drug Development Timeline
pharmaceutical companies to develop cures for rare diseases. As a result, the FDA established the Office of Orphan Product Development (OOPD) in 1982.[4] In addition, Congress passed the Orphan Drug Act with financial incentives for companies developing products for rare diseases in 1983. The three primary incentives that the Orphan Drug Act gives manufacturers are:

  1. Federal funding of grants and contracts to perform clinical trials of orphan products
  2. A tax credit of 50% of clinical testing costs
  3. An exclusive right to market the orphan drug for seven years from the date of marketing approval. This allows for greater discretion in pricing.


Other benefits for Sponsors for orphan-designated products include:

  • Close coordination with the FDA throughout the drug’s development
  • Priority FDA review
  • A waiver of drug application fees


As a result, the OOPD assigned a total of 2,112 orphan drug designations and the FDA approved 347 between 1983 and 2009. The Orphan Drug Act has played a major role in the development of certain rare disease products that may have never made it to market without the OOPD. For example, an effective treatment for infant botulism (a rare neurological disease affecting approximately 100 children in the United States annually), was developed because of the California Department of Health Services and OOPD grants, and was also in close coordination with the FDA.[5]

Today, as a result of the implementation of the OOPD, more than 575 orphan drugs have been successfully developed and marketed. Worldwide sales of these drugs are predicted to reach $176 billion by the end of 2020.[6]

Logistical Challenges of Orphan Drug Development

As orphan drug development expands, clinical trials are becoming more complex and expensive. For instance, the average cost of moving one orphan drug to market can cost $350 million. Clinical trials account for a large percentage of the overall price. In addition, a drug typically takes 10 to 15 years to make it to market.[7]

When designing an orphan drug trial, Sponsors may have difficulty finding historical disease information such as disease prevalence and examples of treatment patterns.  Developing a study protocol without this information can be challenging. In addition, for many rare diseases no standard clinical trial designs or study outcomes exist. This makes it difficult to select endpoints, outcome measures, tools, and biomarkers. The lifelong nature of a rare disease also makes it difficult to determine appropriate study durations.[8]

The potential patient population of a study is limited since rare diseases affect a small number of individuals. This is because small patient populations decrease study variation. In addition, the genetic basis or associated comorbidities of many rare diseases can be confounding factors in study predictability. Also, potential clinical trial patients, investigators, and clinical centers are rare and located around the world. So, finding individuals to participate in a study can be time consuming and require high travel costs.[9]

In addition, rare diseases are frequently discovered at birth or during childhood. For ethical reasons it is considered appropriate for drugs to be tested in adults before in children.[10] Therefore, pediatric studies require Sponsors to carefully balance the ethical considerations of conducting studies in a vulnerable population with concerns about site selection, recruitment, compliance, and statistical powering.[11]

Patient Centricity as a Solution for Orphan Drug Trials

To best meet the needs of the scientific, and regulatory communities, Sponsors must employ new approaches to accelerate the research process and also encourage more interest from Sponsors to explore cures for rare diseases. Sponsors can meet the needs of orphan drug development by focusing on patient centricity. Click To Tweet

Since historical research on rare diseases is typically sparse, investigators can look to patients, patient advocacy groups, and families as the thought leaders on their disease. These individuals and groups can identify the clinical outcomes that are most meaningful to a small subset of individuals with highly specialized needs. [12]

Orphan Drug Development Patient Centricity

Research and Development Patients can provide input on identifying health research priorities and participate in the design and undertaking of research projects. For instance, one study on patient involvement in orphan drug trials found that patients prefer equal collaboration with researchers. This is because researchers are guided towards designing more relevant trials with useful outcome measures.[13] Patient experience and input can be especially beneficial when:

  • Setting the research agenda
  • Promoting collaborative research networks
  • Initiating studies
  • Providing financial support for research infrastructures


Research and development organizations or departments should engage with patient associations and families that have a special interest in, and experience with, rare diseases. As a result, researchers gain a greater understanding of the natural history of the disease and the potential therapeutic impacts that would be most meaningful for patients to find relevant trial patients and produce accurate outcomes. Researchers can also conduct qualitative research studies on the internet habits of these groups provide insights into what a patient population will need from a clinical trial. Additionally, advocacy groups can provide feedback on the appropriateness of a trial’s protocol and help drug developers reach patient populations. Sponsors must use these varieties of communication methods to embrace patient centricity and focus on the quality of life for potential patients.[14]

Clinical DevelopmentPatient involvement in clinical development may contribute to the successful delivery of trials because of the increased likelihood of recruitment and retention.  Some companies investigating orphan drugs involve patients at all stages of clinical development, from providing input into study protocol, the selection of endpoints, to the dissemination and implementation of findings.[15]  Since they often have advanced networks, patients can help to enhance communication about research participation opportunities. In addition, patients can disseminate trial-related findings in a manner that is most meaningful to other patients.

Also, consideration of acceptable physical burden, treatment durations, and travel requirements is necessary when looking towards optimal recruitment and retention. Rare diseases affect individuals on a global level and many patients are children so caregivers and parents must be accounted for when evaluating travel time and costs. In addition, implementing trial centers across several countries may enable higher levels of participation. An alternative to traditional trial design is the idea of in-home clinical services, where the majority of simple clinical procedures (treatment administration, blood draws, and health monitoring) are administered by trial professionals at home. Therefore, travel to designated trial sites is reduced as is the disruption to the life of the patient and their family.[16]

Approval and Market Access The FDA has introduced a Patient Engagement Advisory Committee to facilitate patient involvement in the regulatory process. Companies also collaborate with patient advocates to demonstrate the burden of the disease, add it to the policy agenda, and help to incorporate the benefit-risk preferences into a structured evaluation process.[17]

Educating a small but dispersed market is essential for market success. As a result, disseminating knowledge and awareness amongst physicians, patients, and payers is necessary for orphan drug advocacy. One way to approach drug education is by leveraging clinical programs to establish an active and engaged treatment community. Consequently, this aids in rapid product uptake because the clinical program may have already identified a high proportion of available patients. Sponsoring patient registries can also enhance disease understanding during development and enable tracking of patient progress post product launch. Since education is critical for successful market access for orphan drugs, targeted communications are also more effective than traditional, large-scale sales forces. Target audience assessments are less complex for orphan drugs than non-orphan drugs because the patient population is less diverse. With fewer disease experts, micro-targeting and micro-messaging personalized to individual needs is feasible and therefore yields favorable results.[18]


While orphan drug clinical trials may require customized patient centric design, these trials have the potential to produce effective outcomes for patients and Sponsors alike. Furthermore, with regulatory support, financial incentives, and effective study design and management, orphan drug trials can be a valuable to any Sponsor’s clinical trial landscape.




















PharPoint press release
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Press Release: PharPoint Research Named One of Triangle Area’s Fastest Growing Companies for 2016

October 4, 2016

RESEARCH TRIANGLE PARK, N.C. – PharPoint Research, a contract research organization (CRO) headquartered in North Carolina, is once again a recipient of the Triangle Business Journal’s Fast 50 award, which highlights 50 of the fastest growing companies in the Triangle area. This is the fourth time the company has received this honor.

Each honoree is selected and ranked based on a formula that counts revenue growth and profitability in the preceding three years (2013 – 2015). The data are processed and analyzed by PricewaterhouseCoopers and the rankings will be revealed during the Fast 50 Awards dinner on November 9.

PharPoint has increased their presence in the pharmaceutical industry over the past 12 months, by now providing clinical development services for global trials. “Our global expertise offers Sponsors access to emerging markets and heightens our management of clinical trials,” says PharPoint CEO, Art Holmes.

The company has evolved into a full-service CRO over the past decade and developed processes to manage trial logistics in a completely virtual environment to improve transparency and information exchange with Sponsors. “Our clients are a testament to our success,” continues Holmes. “Our growth is a direct result of the strong relationships we build with Sponsors and partners alike. Companies turn to PharPoint for our team’s breadth of therapeutic and program expertise, horizontal business structure, and study execution capabilities. We are proud to be recognized by the Triangle Business Journal for our continued level of service to our clients.”


About PharPoint

PharPoint Research is a full-service contract research organization (CRO) that specializes in data management, biostatistics, clinical monitoring, and medical writing. The company is uniquely poised to serve the needs of pharmaceutical, biotechnology, and medical device development organizations. PharPoint Research is experienced in strategic consulting engagements and Phase 1 through Phase 4 clinical research projects across a variety of therapeutic areas, including cardiovascular, central nervous system (CNS), infectious disease, oncology, and rare disease indications. PharPoint Research is headquartered in Research Triangle Park, N.C., with strategic technology partners and service providers around the world, including North and South America, Europe, and Asia-Pacific. For more information, please visit



PharPoint Research

Tim LaCroix, Head of Business Development


patient-centric clinical trials

Patient Evolution in U.S. Clinical Trials

Patient Diversity in Clinical Trials

Patient enrollment for clinical trials is a complex process that involves not only patients, but physicians and trial professionals. Clinical trials strive to execute drug treatments on a sample group that is large enough to be statistically significant while also being representative of the population that the drug intends to serve. Differences in age, gender, race, and ethnicity all play a part in how effective a drug will be on an individual patient, which is why pharma companies must be diligent to include a diverse group of patients in their sample population.  But this hasn’t always been easy to do.  Join PharPoint Research as we explore who participates in clinical trials, why patient diversity is important, and what we can do to increase patient enrollment.

The History of Clinical Trial Participation

Diversity in Clinical Trial TimelineCompanies performing clinical research trials are required to follow stringent guidelines set forth by the National Institute of Health (NIH), which is the primary agency of the U.S. government responsible for biomedical and health-related research. The NIH is part of the U.S. Department of Health and Human Services and is the largest biomedical research agency in the world.

Over the years the NIH has enhanced their clinical trial policies to increase safety and patient representation in clinical research that better accounts for variations of a drug’s effects across gender, ethnic, and racial bounds. But these policies have evolved over time and have not always been in place.

For example, as late as the 1990s some researchers were hesitant to include women in clinical trials who had likelihood of becoming pregnant. The researchers feared the drugs being evaluated could lead to birth defects, so certain experimental drugs were mainly tested in men. Makes sense, but when a subset of these drugs finally made it to market there were select instances where female patients experienced unforeseen adverse events.  While not the only factor, the lack of an all-encompassing sample population was a major component that allowed these adverse events to occur.

In 2001 the NIH updated its policy on the inclusion of women and minorities as subjects in clinical research, so that “all NIH-funded clinical research will be carried out in a manner sufficient to elicit information about individuals of both sexes/genders and diverse racial and ethnic groups and, particularly in NIH-defined Phase 3 clinical trials, to examine differential effects on such groups.” These updates help to ensure that clinical trials include a more robust sample population, but this doesn’t mean it is always easy for clinical trials to recruit minorities and patients from certain backgrounds.

Obstacles for Patient Recruitment

So the question becomes “Why can it be difficult to recruit certain patients for some clinical trials?” Unfortunately, there is no one single answer, but rather a group of factors that when brought together can create a burden for patient recruitment.

One problem stems from a clinical site’s geographical location and a prospective patient’s inability to make feasible travel arrangements.  Another problem is the lack of translation resources due to budget restrictions. This leads to information only being provided in English which is a significant deterrent for non-native speakers.  However, one of the most significant indicators of participation for a clinical trial is a prospective patient’s socioeconomic status. Patients with annual household incomes below $50,000 are 32% less likely to participate in clinical trials than higher income patients. This is due to factors such as the inability to schedule time off work, travel costs, insurance co-pays, and insufficient information about clinical trials as a treatment option.

The good news is that trends are changing.  Between 2010 and 2014 the proportion of African American participants in Phase 3 clinical trials increased from 10% to 23% and as of 2016 the average participant population in all NIH clinical trials more or less matches the racial and ethnic makeup of the U.S.  To better serve the needs of our diverse population, the industry is also adopting new ways to include underrepresented communities for clinical trials through patient-centric trials, where patients play an active role in the development of the trial they participate in.

The Growth of Patient-centric Trials

The vast wealth of information available through online resources has led to modern patients who are better informed about the medicines they use. The pharma industry has reacted by experimenting withDiversity in Clinical Trials patient-centric clinical trials that include greater patient involvement in the research, development, and planning stages. Pharma’s goal in using this technique is to boost recruitment, reduce costs and ultimately, speed up a drug’s timeline to market.

So, what does “patient-centric” really mean and how can this approach be used for clinical trials?  Patient-centric trials are designed to focus on society, technology, patient lifestyles and the realization that trials can be better designed and executed if the needs of patients are more adequately addressed.

Some of the ways pharma companies are finding ways to integrate patient voices into clinical trials are through social media and engaging patients early on in the trial process.

Social Media and Wearable Sensors

The capabilities of online communication and technological monitors have made the connection between physicians and patients stronger than ever.  Social media and advocacy groups such as the Michael J. Fox Foundation and the ALS Association provide online forums for participants to share health information, and a similar approach can help clinical trial professionals collect health data from patients and increase communication with physicians. Likewise, wearable sensors provide physicians with up-to-date health information from trial participants when geographic or time constraints may be a factor in clinical trial participation. (For more information on how doctors are communicating with patients through technology, check out PharPoint’s wearable sensor blog post).

Early Engagement

Getting patients involved in the clinical trial enrollment early on is key. This goes beyond just enrolling patients, but also encouraging engagement throughout the process. Inviting patients to review draft protocols and procedural schedules gives patients better insight into the maintenance of a clinical trial and how their own health will be managed. Additionally, asking patients how and when they prefer to be involved can have a big impact on how a development team designs a trial. Educating patients on the drug development process and how the clinical trial will affect their lives gives patients a more active role in the trial and can increase enthusiasm for playing a part in extending medical research.


Innovative drug development continues to work towards more effective patient processes. Through the means of patient-centric trials, enrolled patients are more involved in the clinical process, enabling faster drug development and a more accurate sample population.  This process is only in the beginning stages, but is a step in the right direction and our team at PharPoint is excited to see where it goes.

If you are interested in participating in a clinical trial or would like to find clinical trials near and relevant to you, visit The Center for Information and Study on Clinical Research Participation.  Have you or someone you know participated in a clinical trial? Have you experienced first-hand a patient-centric trial? Tell us about it in the comments below!