Letter From the Editor

Written by: Scott Finger | sfinger@ciscrp.org

Dear readers,

If you have been following CISCRP and our newsletter, you know about some of the exciting and innovative changes we have made over the last year and a half. While we understand it can be tiring to hear so much about the pandemic and the “new normal,” we can’t overstate how important it is to continually address disparities in health care and clinical research. In this issue, we cover some of the changes we have been making and what you can expect from us in the coming months.

One of CISCRP’s biggest initiatives has been to address the inequity of participation in clinical trials. In other words, we want to turn underrepresented communities into represented communities. Our Health Literacy team is collaborating with members of these communities to learn how we and clinical trial professionals can bridge the gap. We will add this knowledge to our library of educational brochures, so stay tuned for the finalized publications. Also in this issue, we discuss how our AWARE for All live educational series focuses on diversity and inclusion. Our next event is happening October 21, and our final AWARE event of the year will be held on November 18.

Our efforts would not be possible without our Research Services team having important conversations with the community. The results of our Perceptions & Insights Study, conducted every two years, will be available this fall! Read here for a sneak peek of some of the key findings, including responses from members of ethnically and racially underrepresented communities.

In our Supporter Spotlight article, Luther T. Clark, MD, Deputy Chief Patient Officer of Merck, explains some of the other issues surrounding healthcare disparity. These issues include the pandemic, social and economic factors, and certain groups’ lack of access to technology (known as the “digital divide”). With in-person interactions becoming rarer, it is important now more than ever to help communities without the proper resources.

CISCRP is also taking steps to make research results more accessible. The results of clinical trials and other scientific studies are incredibly complex, and there is a large demand for plain language information. In response, we have partnered with sponsors and other experts to create “plain language summaries of publications” (PLSPs), which turn hard-to-understand articles into easy-to-understand summaries. This new offering expands our plain language portfolio to include not only trial results summaries, but all types of scientific and medical articles.

It takes a community to make change, and that is absolutely true when it comes to clinical research. In this issue, we highlight the efforts and the experience of CISCRP’s own Phyllis Kaplan in our ongoing Medical Hero Spotlight series. Read here to learn more about Phyllis’ journey from patient and advocate to clinical research participant to Senior Manager of Events & Community Engagement at CISCRP.

While the clinical research community has become more aware of the disparity in representation, clinical trials are still not as diverse as they should be. We still have much work to do to address the knowledge gaps that exist within the general public. Whether you are a health professional or member of the community trying to do your part, we encourage you to learn as much as you can about clinical trials and the important issues we need to address. As a team, we can make a difference to ensure that all voices are heard, and that research accounts for people of all backgrounds.

Take care, and stay safe,
Scott Finger
Editor, Health Literacy

 

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Medical Hero Spotlight: Phyllis Kaplan & Type 1 Diabetes

Written by: Melissa Daley | mdaley@ciscrp.org

Phyllis has a vague recollection of waking up in a hospital with tubes attached to her body, and a distinct memory of saying, “Take the tubes out!” At the age of two, she was diagnosed with type 1 diabetes, an autoimmune disease, typically diagnosed in childhood, but can manifest at any age. Diabetes has led Phyllis on a path from patient to advocate to clinical research participant.

“I have been an advocate since I was 12 years old. It started in junior high school, in a gym class when the teacher made me take off my medical alert bracelet, due to a ‘no jewelry in gym class’ rule,” says Phyllis. When she went to retrieve the medical bracelet from the gym locker after class, she discovered it had been stolen. This incident spurred Phyllis to write a letter to the town superintendent of schools, demanding its replacement and a change in the rule to allow medical-related items to be worn. By the time the letter had been delivered, the bracelet had been anonymously returned to the school’s lost & found box. Phyllis was allowed to wear the bracelet moving forward.

The most important thing to understand about type 1 diabetes, says Phyllis, is, “The patient or caregiver has to make so many decisions about the disease, every day with no break. With diabetes, every day is different.”

Type 1 diabetes develops quickly. The body’s immune system attacks and destroys beta cells in the pancreas that create insulin. The body cannot produce insulin without these beta cells. Insulin is a peptide hormone that helps your body metabolize fats, proteins and carbohydrates through glucose (a type of sugar) that is released into the bloodstream when you eat food. The glucose is then absorbed from the blood in the liver, fat and skeletal muscle cells. Type 2 diabetes develops more slowly, over time. The body produces insulin, but cannot use it effectively.

Decisions about how much medication to take are based on many variables including food, exercise, change in weather, change in personal schedule, and stress.

“That’s why education is so important,” says Phyllis. “If I am going to exercise, I have to plan ahead, at least a couple of hours before, as exercise impacts blood sugar. There are so many hidden things to know about diabetes that impact your decisions.”

Phyllis has participated in four clinical trials: two for rescue medications for severe hypoglycemia and one for a medical device. “As a longtime advocate, I felt that participating in a clinical trial was the ultimate form of advocacy,” Phyllis explains.

“The trials were very different from each other,” says Phyllis. “Two of the three were very easy. One involved two full days in clinic, and that was really hard, with nine hours of ongoing blood tests. Those were physically difficult days, but worth it. The other two clinical trials were less invasive.”

When considering the two-day, in-clinic trial, Phyllis and her husband reviewed the protocol together. “I wouldn’t participate without consulting him,” says Phyllis. He accompanied her to the two in-clinic days, to be with her during the nine hours of ongoing tests and to lend additional support.

When asked if she faced any concerns from family or friends about her clinical research participation, Phyllis says, “No, quite the opposite. People were really interested in the ‘why’ of what I was doing and what the outcomes were.” Phyllis didn’t seek any advice from patient advocacy organizations, because of her own experience as an advocate. She is a brand ambassador with Medtronic Diabetes to share her experience with their medical device, and also volunteers with JDRF (Juvenile Diabetes Research Foundation) and ADA (American Diabetes Association).

Phyllis advises individuals considering clinical research participation to “Ask all the questions you have when meeting with the nurse/study lead. No question is too silly. Read the protocol and informed consent, which can be confusing. Use a highlighter to mark items in the protocol or use Post-its to make notes. Keep asking questions throughout the course of the study. At times the research staff may not always be patient-centric, and if you’re not getting the answers to your questions, ask to speak with someone else on the study team. Be your own best advocate and keep pushing. Researchers are not always prepared to answer patients’ questions. If something doesn’t sit well with you, voice it.”

Phyllis’ advocacy work has also led her to CISCRP, where she is Senior Manager, Events & Community Engagement. CISCRP’s mission to inform and engage patients and the public about the importance of clinical research resonated with her. Phyllis leads CISCRP’s Aware for All free clinical research educational programs, which have pivoted from in-person, city-specific events to regional, virtual programs with the advent of the pandemic. You can learn more about AWARE for All here.

Her experience as a clinical trial participant has strengthened Phyllis’ commitment in sharing information about the importance of clinical trials to everyone. Phyllis is adamant and passionate about participating in clinical research again if the opportunity presents itself, reiterating, “Absolutely. Without clinical trials, new treatments can’t happen, and without clinical trial participants, clinical trials can’t happen.”

To search for medical conditions in a specific location, visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

 

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Why Clinical Trial Diversity Is Key To Increasing Access To Routine Care And Innovative Treatment Options

By: Luther T. Clark, MD, Deputy Chief Patient Officer, Merck

The COVID-19 pandemic has shone a light on longstanding health care disparities and amplified the importance of clinical trial diversity, especially participation of those patients and communities disproportionately impacted by the disease being studied. Since clinical trials function as the gatekeeper to bringing new medicines safely to patients and communities, it has become increasingly important that diverse communities be represented in our research efforts.

Clinical trial participation provides access to possible new and innovative treatment options for patients, an especially important benefit for those who have conditions with limited treatment options – such as some forms of advanced cancer.  It is important to note that, while not all clinical trial participants will receive the investigational treatment being evaluated in the clinical trial, all patients receive high quality standard-of-care, which is the standard treatment that is used for the health condition.

During the COVID-19 pandemic many patients have delayed or avoided routine medical care for underlying conditions – a hidden harm that has further amplified its already enormous health toll. While pandemic associated medical care delays and avoidance may be understandable, safely returning to essential care is imperative for improving patient outcomes and reducing disparities as well as increasing patient access and ability to participate in clinical research. 

Pandemic Associated Medical Care Delays and Avoidance
During the COVID-19 pandemic, delays and/or avoidance of medical care for both routine and serious conditions have been widely reported. According to one recent analysis (1) an estimated 41% of adults in the U.S. delayed or avoided medical care because of concerns about COVID-19 – including both routine care (31.5%) and urgent or emergency care (12.0%).  Avoidance of urgent or emergency care was more prevalent among individuals with underlying medical conditions, Black adults, Hispanic adults, young adults, persons with disabilities and unpaid adult caregivers. When patients delay or avoid medical care, they increase both their morbidity and mortality risks. For example, 

  • Vaccines play an important role in helping to protect people from preventable diseases, but data show concerning decreases in vaccination rates since the onset of the COVID-19 pandemic. A recent international poll revealed that 73% experienced disruptions in vaccine demand.
  • Similarly, while routine cancer screenings have contributed to important cancer survival gains, screening rates have dropped during the pandemic. In the U.S. alone, approximately 285,000 breast cancer screenings, 95,000 colon cancer screenings and 40,000 cervical exams were missed between March 15 and June 16, 2020.

Cancer is a particularly powerful example of how longstanding inequities in care coupled with the pandemic’s impact on reduced access/utilization of routine health care can converge and create an even more devastating impact on patients, families and communities. Cancer incidence is known to be disproportionately higher in under-represented minorities compared to other groups, and access — to timely diagnosis, quality care and to clinical trials of promising therapies — is suboptimal among people of color. Furthermore, cancer and its treatment predispose to many other health outcome disparities, as demonstrated by the disproportionate impact of COVID-19 on morbidity and mortality rates among people with cancer.   

Social Determinants of Health (SDOH)
Social and economic factors, referred to as social determinants of health (i.e., education, economic stability, neighborhood, health and health care access, social and community context) not only contribute to healthcare disparities but may also negatively impact the decision and the ability of patients to participate in clinical research (2-4).  By recognizing and understanding the SDOH, we can help accelerate return to medical care, overcome barriers to minority participation in research, and ultimately improve patient outcomes. Effective and meaningful community engagement, collaborations, and partnerships are critically important for addressing all of the SDOH, and especially helpful for increasing awareness, education and building trust.

Bridging the Digital Divide
Digital technologies have many potential benefits for improving healthcare, including the potential to improve healthcare quality, patient safety and reduce disparities (5).  However, inequities in access to virtual technologies do exist and may lead to or exacerbate disparities. Often referred to as the “digital divide”, there is a considerable difference between those patients/communities that have access to digital technologies and the ability to understand and use them effectively (digital literacy) and those who do not.  For example, access and utilization of digital health care technologies are known to be significantly lower among older Black and Hispanic patients than their white or Asian counterparts.  In addition to differences in access and digital literacy, structural inequities (i.e., lack of broadband internet availability) impact disproportionately some groups and communities – racial and ethnic minorities, rural communities and individuals of lower socioeconomic status (6). Without recognition and action, the digital divide will only widen.

Summary and Conclusions
It is important for the medical and public health community, policymakers, employers and health advocates around the world to come together to encourage people within our communities to get the care they need.

  • As we join the multi-sector effort to combat COVID-19, we must also focus on helping people in our communities get the routine care they need to protect their long-term health.
  • It’s vital to support individuals and patients in returning to care because delays or cancellations are associated with significant health risks.
  • While the COVID-19 pandemic has caused unprecedented disruption to the healthcare system, it has also led us to identify opportunities to strengthen it for the future. We are committed to working with stakeholders to enact solutions that will improve patient care and help protect public health over the long term.

 

This article was also featured in our Patient Diversity Campaign. See Campaign Here.

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References:

  1. Czeisler MÉ, Marynak K, Clarke KE, et al. Delay or Avoidance of Medical Care Because of COVID-19–Related Concerns — United States, June 2020. MMWR Morb Mortal Wkly Rep 2020;69:1250–1257. DOI: http://dx. doi.org/10.15585/mmwr.mm6936a4
  2. Clark LT, Watkins L, Pina IL, Elmer M, Akinboboye O, Gorham M, Jamerson B, McCullough C, Pierre C, Polis AB, Puckrein G, Regnante JM. Increasing Diversity in Clinical Trials: Overcoming Critical Barriers. Curr Probl Cardiol 2019; 44:148-172
  3. Asare M, Flannery M, Kamen C. Social Determinants of Health: A Framework for Studying Cancer Health Disparities and Minority Participation in Research. Oncol Nurs Forum. 2017 January 02; 44(1): 20–23.  https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5583708/pdf/nihms893015.pdf
  4. Weinstein JN, Geller A, Negussie Y, Baciu A. Communities in Action: Pathways to Health Equity. Report of the National Academies of Sciences Engineering Medicine (2017). https://www.ncbi.nlm.nih.gov/books/NBK425848/pdf/Book-shelf_NBK425848.pdf
  5. Lopez L, Green AR, Tan-McGrory A, King R, Betancourt JR. Bridging the digital divide in health care: the role of health information technology in addressing racial and ethnic disparities. Jt Comm J Qual Patient Saf 2011 Oct;37(10):437-45. doi: 10.1016/s1553-7250(11)37055-9.
  6. Campos-Castillo C, Anthony D. Racial and ethnic differences in self-reported telehealth use during the COVID-19 pandemic: a secondary analysis of a US survey of internet users from late March. Journal of the American Medical Informatics Association, 28(1), 2021, 119–125. doi: 10.1093/jamia/ocaa221

Results From The 2021 CISCRP Perceptions & Insights Study Coming Soon

By: Jessica Cronin | jcronin@ciscrp.org

CISCRP is excited to announce that the full results of the 2021 global Perceptions & Insights Study will be available this fall! This latest study contains insights from nearly 12,000 individuals around the world on various aspects of clinical research — including the impact of the COVID-19 pandemic on awareness and perceptions, as well as thoughts on and experiences with clinical trials that require fewer visits (decentralized trials). These results uncover significant new findings on patient engagement preferences and ways to increase access, particularly among underrepresented communities.  

Our 2021 study captured the experiences of over 5,500 trial participants across diverse therapeutic areas, which was nearly 2,000 more participants than in previous years!  

Key findings from the 2021 Perceptions & Insights Study include:

  • Most respondents (75%) noted that the COVID-19 pandemic increased their overall awareness of clinical research, particularly among those from ethnically and racially underrepresented communities.
  • Individuals from ethnically and racially underrepresented communities were significantly more likely to cite that knowing pharmaceutical companies employed diverse staff would help to increase their trust in pharmaceutical companies.
  • While most (82%) said that they were willing to participate in clinical research, about one third of individuals (36%) cited being more willing to participate as a result of the COVID-19 pandemic.
  • Of those who participated in a clinical trial during the pandemic, top changes reported included a shift from in-person visits to virtual clinic visits (over the telephone or computer), and an increase in the use of telemedicine and home trial drug delivery. Only 20% reported that their clinical trial was temporarily stopped. Consistent with prior studies, most clinical trial participants (92%) noted that their trial met or exceeded their expectations, with a significant percentage (95%) willing to participate in another trial in the future — a strong indicator of overall satisfaction.

Since 2013, CISCRP has conducted the Perceptions & Insights Study every other year to monitor trends and identify opportunities to better inform and engage the public and patients as partners in the clinical research enterprise.

To review these findings and more from the 2021 Perceptions & Insights Study, CISCRP will be hosting a webinar in October, as well as posting reports on the CISCRP website. If you would like to register for the webinar – please go to here.

 

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PLSP: The Results Of The DESTINY-Breast Cancer01 Clinical Trial Published In Future Oncology

Written by: Joan Chambers | jchambers@ciscrp.org

CISCRP and Oxford PharmaGenesis collaborated with Daiichi Sankyo, AstraZeneca, and Dr. Shanu Modi of the Memorial Sloan Kettering Cancer Center in New York to write a plain language summary publication (PLSP) of the results of the DESTINY-Breast01 clinical trial.

The demand from the public, patient, and health care communities for plain language information on results of clinical trials is extremely high. For more than ten years, CISCRP has been translating scientific clinical trial results information into plain, easy-to-understand language for patients and the public around the world to be communicated in print and digital formats.

This PLSP provides important information about HER2-positive breast cancer to patients, their family members or caregivers, and patient advocates. In a simple format, the PLSP highlights and addresses: what HER2 positive breast cancer is, why the clinical trial was conducted, the most common adverse events, and the overall trial results.

Over 253 women aged 18 and older participated in the trial. This PLSP answers the main questions researchers had in the DESTINY-Breast01 trial:

  • Did the participants’ tumors shrink or disappear after receiving the T-DXd treatment?
  • For how long did the participants’ tumors shrink or disappear before growing again?
  • For how long did the participants live with their cancer before it got worse?
  • What were the most common adverse events during treatment with T-DXd?

The PLSP team worked to ensure the PLSP was easy to read by adding creative visuals, tables, and answers to key questions about the DESTINY-Breast01 clinical trial. The PLSP was reviewed by an editorial panel of patients, patient advocates, members of the public, and healthcare professionals to evaluate and confirm that a “patient-first” approach was taken. The panel reviewed the writing, design, and layout to help patients, family members, and caregivers understand the trial results.

Read the full published PLSP on Future Oncology here.

CISCRP collaborates with industry organizations to create documents from scientific manuscripts that are accessible for patients, patient advocacy groups, and the public. Visit Health Communication Services for more information. 

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AWARE for All Fall Updates

Written by: Hope Ventricelli | hventricelli@ciscrp.org

At the beginning of 2020, the CISCRP Events team had their work cut out. We had to adapt our live educational event (AWARE for All) model to a completely virtual program while remaining accessible to the patient and public communities. And, we had to do so while affording CISCRP the opportunity to engage the public in a new and meaningful way.

AWARE for All-Southwest will be a live webinar stream via the virtual platform on October 21. With it, we aim to continue spreading awareness about the essential role that trial participants play in clinical research and how the research community can engage them as partners in the clinical research process.

Unlike our previous AWARE webinars, AWARE for All-Southwest will be condensed to just one hour and focus heavily on the panel discussion—a favorite segment among audience members. This discussion between trial participants, researchers, and industry stakeholders will give attendees a well-rounded view of the clinical trials process from varying perspectives.

Diversity and inclusion is at the heart of all the AWARE For All events. The importance of representation in healthcare is pivotal to comprehensive treatments, and the need is often misunderstood by the public.

“People can experience differences in response to a treatment as well as different side effects,” explains Dr. Lok, who participated in the recent Midwest panel. “This is why clinical trials need people from all communities representing all stages of a disease to find an effective treatment.”

As part of these efforts, CISCRP collaborated with trial volunteers and researchers to share their unique experiences with clinical research to help the audience better understand the importance of participation. As Desiree De-Luca-Johnson, a Breast Cancer Trial patient, shared at a recent event, “I felt safer in a clinical trial because I had a better relationship with my research oncologist and oncology nurse in Boston. I was able to feel safe for a year—it gave me some time to breathe and research my options.”

At the end of this summer, the events team was hoping to be in person for the final program of the season. CISCRP selected Atlanta for our first face-to-face event since 2019 due to the robust clinical research community, demographic diversity, and already established connection with the city. However, due to the Delta variant and public safety concerns, we have decided to remain virtual and proceed with an Atlanta-based event. Keeping virtual fatigue in mind we look forward to developing a fresh take on the webinar for  AWARE for All – Atlanta with local involvement and an exciting new agenda.

This flagship CISCRP initiative is a longstanding, internationally recognized program with a goal to address health disparities and low levels of clinical research literacy. Since 2003, CISCRP has been developing elements of the AWARE for All program to keep up with current health concerns and community needs while maintaining the need for diversity in research. Be sure to follow us for the latest updates on both programs and local opportunities to be involved.

To learn more about supporting as a sponsor, participating as a speaker or exhibitor, please email Hope Ventricelli, Manager of Events and Community Engagement, at hventricelli@ciscp.org.

 

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CISCRP’s Newly Launched Educational Brochure Initiative

Written by: Makenzie Michel | mmichel@ciscrp.org

At CISCRP’s Health Communications Services team, we develop educational materials to engage and empower patients and the public, including communities that are typically underrepresented in clinical trials. As an independent, non-profit organization, our unbiased deliverables are non-promotional. We offer many of these materials (which can be found in our online Education Center) free of charge to the public and patients.

Our materials are written in easy-to-understand language—sometimes referred to as “lay” or “plain” language—to ensure that we are communicating complex issues in simple terms. We also write our content to be engaging and culturally appropriate, and to address the key concerns of the audiences we hope to reach. Using strategic graphic design, we reinforce key messages through careful use of layout, colors, icons, images, charts, and other graphics.

Since being founded in 2003, we have developed brochures and other materials about the critical role of clinical research and trial participants in public health. One of our long-standing goals has been to reach communities that have been underrepresented in clinical trials. During our brochure reevaluation this year, we have collaborated with key stakeholders and community members to get significant input from these people we hope to reach. With the increased awareness and greater emphasis on social and health equity, we are focusing on connecting with underrepresented communities to make sure we appropriately address the concerns and barriers they face.

We are continuously partnering with medical writers, subject matter experts, and community members. We believe that this method of co-development will allow for the most educational and highest quality content. An editorial panel of patients, public, and professional representatives reviews each deliverable and provides their feedback before we finalize any publication. Additionally, we utilize Feedback Forums to acquire more detailed information about certain topics. In these forums, we speak directly with members of the communities we hope to reach and/or those who have expertise in communicating and working with those communities. They help to ensure the materials are effective, easy-to-understand, unbiased, and culturally appropriate. As always, all CISCRP educational materials are reviewed and approved by an IRB (Institutional Review Board), and are available in multiple languages.

Research professionals, sponsors, and other members of the clinical research community are able to license and co-brand these updated materials. Our brochures can be used by research centers, academic institutions, pharmaceutical companies, health care professionals, and advocacy groups to provide education about clinical research and clinical trial participation.

Keep an eye out for upcoming newly launched educational brochures!  

Visit CISCRP’s Education Center for more information about the newest brochures, to learn about the topics we are working on, and to share ideas that help close other gaps in clinical research.

As we continue to update our portfolio of educational materials, please reach out to Joan Chambers at jchambers@ciscrp.org to discuss purchasing and licensing opportunities. You can also visit CISCRP’s online store to purchase these deliverables.

 

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Medical Hero Story: T.J. Sharpe, Melanoma Advocate

Why One Cancer Survivor Wants All Patients to Consider Clinical Trials

When melanoma unex­pectedly returned after a successful surgery twelve years prior, T.J. Sharpe was both a husband and a father. Second, third and fourth opinions later, Sharpe was finally offered a life-chang­ing option.

“I wanted a chance to see my children grow up and be the husband and father I could be,” says Sharpe. With the first few doctors, Sharpe’s predicted life expectancy was under two years, which he was determined to extend. “I wanted the best chance at a long-term response.”

Seizing an opportunity
Sharpe was on his fourth oncol­ogist when he was offered his first clinical trial. It was for a new treatment in the form of a pill, and Sharpe was the first patient in the trial. Because it was so new, he ran into bureau­cratic barriers. “I was the first patient to try it, so there were a few stakeholder companies and pharmaceutical companies that I had to wait for over a month to get the contract approved.”

After contacting the stakehold­ers himself to push the paperwork through, Sharpe started the trial. “When you have a family and you are facing mortality, I wasn’t going to miss the chance to see these kids grow up because I was missing part of a signature.”

An incredible recovery
After months on the pill treat­ment, Sharpe’s tumors weren’t responding. But with determi­nation came plan B, and Sharpe started on his second trial. After twelve weeks, he saw a 46 percent reduction in his tumors. Four years later, Sharpe’s only signs of cancer are small spots that have stabi­lized for over two years. Today, he remains in the trial to continue monitoring his response and over­all system.

The results of the clinical trial have so far doubled his life expec­tancy, an accomplishment Sharpe does not take lightly. “Clinical trials should be considered as an option for care in every single case,” he says. For Sharpe, the norm should be to hear your stan­dard care options, but in conjunc­tion with the clinical trial options.

At that point, let the patient and their doctor make the most informed decision. “When it comes down to it, we are all patients at some point, so we should know what all of our options are before making decisions.” 

 

Article from 2017 Clinical Trials Supplement, USA Today. Read full Supplement here >

Medical Hero Story: Jackie Zimmerman, MS Advocate

Jackie Zimmerman is an accomplished entrepreneur, marketing professional, former roller derby league president, and non-profit founder with a “can-do” energy that is clear when you speak with her. She is also a patient advocate for people with multiple sclerosis, also called MS. She herself received the diagnosis during college at the age of 21. In the face of this news, Jackie decided to share her story with others. She later became a participant in clinical research. 

“It was a total surprise  no one in my family has it,” Jackie said. “My first question was…am I going to die? I didn’t know anything about MS. And all the patient-facing documentation was targeted at older individuals.” 

Jackie found this surprising, particularly when she learned that most patients are diagnosed in their twenties. To help process this news, she turned to blogging. 

“Initially, I started a newsletter and then it became a blog. This was before social media, so blogs were how you connected. Blogging got me on the scene of patient advocacy. There are lots of advocates now, but then, there weren’t as many,” Jackie explained. 

Jackie was diagnosed with relapsing remitting MSWith this form of MS, symptoms may “flare up” and then seem to be gone for a period of time. Jackie’s first symptoms were during a flare. At that time, the standard of care was given through an injection. 

“Which was really scary at 21  who likes needles?” Jackie said. “My doctor didn’t provide any guidance. He told me to pick whatever medication I wanted. Today, there are many different options and methods of delivery.” 

Another complication in the treatment of MS is that in terms of symptoms, reactions to medications, and outcomes, no two cases are alike. 

Jackie first participated in an MS clinical trial in 2008. However, she was unable to complete it due to unrelated treatment she was receiving forinflammatory bowel disease, also called IBD. Over a decade would pass before Jackie would join another clinical trial. 

During that time, Jackie founded Girls with Guts, a non-profit to support women living with IBDShe became a more active patient advocate. This led to public speaking engagements and work with healthcare and pharmaceutical companies looking to connect with patients. 

There were 2 factors that steered her back to clinical research as a healthcare option for MS. 

“I asked my neurologist questions, because I wanted to keep abreast of things, particularly because I write about them as an advocate,” Jackie said. “Being part of a patient advisory board for a bio-pharma company was also a huge driver. I understand about clinical research participation and what it means. The only factor this second time was a consideration about the time commitment  it’s about a 25-minute drive away. The trial does offer transportation if you need, which I think is awesome, but I don’t need it.” 

When asked about other influences in her decision-making, Jackie commented, “I’m married, so my husband was involved. He said it’s my choice, but I considered him in the process, and all the things you think about when you’re involved in a partnership. I know how to talk with my friends and family about clinical trials to allay their fears. I know how to position it so as not to scare anybody.” 

Jackie did not consult any patient advocacy organizations for guidance before participating. 

“I’m in the minority here because I know a lot more about clinical trials than the average patient. I knew what questions to ask because I had been on a patient panel and participated in an FDA patient advocacy training. I’ve spent a lot of time learning about trials.” 

Jackie shared some wisdom based on her experience with clinical trial participation. 

“Oh man, there’s so much advice,” Jackie replied, laughing. “I have so many angles. Mostly, don’t be afraid of them. It’s not a last resort for MS in terms of treatment. It’s a way to assist your community and get access to medications, years in advance. You’re so heavily monitored that the potential for anything going wrong is quite low.” 

Jackie would consider participating in future clinical trials. 

“Yes  as long as it fits into my life and it’s not an inconvenience or as long as the potential benefits outweigh the potential inconvenience. I know I have a lot of privilege  I can work from home and make my own schedule. If you’re looking at trials that may not fit into your life, know that there are pharma companies working around participant schedules with office and home visits. If it’s not feasible right now, it doesn’t mean that it will never be feasible.” 

Note: COVID-19 has led many sponsors to offer virtual clinical trials and the use of digital devices to monitor patient progress. CISCRP recently conducted a survey on the impact of COVID-19 on clinical research. Access the reporthere.

To search for medical conditions in a specific location visit our Search Clinical Trials page.

To stay informed about clinical trials, visit our Resources page.

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Medical Hero Story: Jillian McNulty, Cystic Fibrosis Advocate

Born with cystic fibrosis, Jillian McNulty has spent her entire life fighting to stay healthy.

It was only during a clinical trial, when she tried a new medicine called Orkambi, that she met with success. Since then, she’s been fighting for other cystic fibrosis (CF) patients to have access to the drug too.

One of four children, McNulty’s oldest brother had cystic fibrosis and died when he was five-and-a-half. Her prognosis wasn’t good either. When she was born, doctors told her parents she wouldn’t live past her fifth birthday. She’s now 43 and jokes that she has geriatric CF, and is grateful to be alive.

Last hope
Over the years, McNulty has suffered from recurring pneumonia, and sometimes spent 8-9 months out of the year in the hospital.

“I didn’t have a great quality of life,” she recalls, noting her condition was at its worst in 2012 after her other brother, who had special needs, died. She was able to run a marathon before her lung function dropped from the high 50s (percent) to the 30s. She became dependent on IVs and antibiotics.

Fortunately, McNulty qualified for the Orkambi clinical trial, which required her to have lung functioning in the 40s and to not be hospitalized for four or more weeks.

She got emotional when she got the news that she qualified. “I can remember I cried my eyes out because to me this was my last chance, my last hope. Things started to turn after that.”

Though she struggled for the first four months of the trial, at six months her hospitalizations started to decrease. But things took a turn in 2016, when she contracted swine flu and influenza A at the same time. Her lung function was down to 11 percent. Things were grim, but McNulty pushed on.

Changing lives
“Orkambi brought me back from the brink,” McNulty says, explaining it took three months to recover.

She says the drug, which is manufactured by Vertex, works on the underlying cause of CF, a life-threatening disease that affects approximately 75,000 people in North America, Europe, and Australia.

“It’s not a cure but it tweaks the channels so our bodies respond better,” says McNulty, whose lung function is now at 42 percent.

These days, she spends only six weeks a year hospitalized. But now she’s struggling with end-stage kidney disease, another chronic condition she’s had for years, and needs a kidney transplant. But before that, she needs a lung transplant.

Still she’s optimistic about her future and the future of other CF patients.

“It’s all changing. People with CF are going to live so much longer,” says McNulty. “That’s incredible.”

The CF advocate
For the past 11 years, McNulty has campaigned for CF patients in her native Ireland, including lobbying the Irish government to make sure Orkambi and other medications were made available for CF patients. Her campaign was successful and she obtained “pipeline” approval for 10 years of promised accessibility to the drug.

“My friends were dying and I couldn’t stand by and say nothing,” says McNulty, who’s won three awards for her advocacy. “I needed people to see what Orkambi had done for me.”

She’s also been instrumental in campaigns related to the hospitalization of CF patients, helping ensure they get single-unit hospital rooms to prevent them from being exposed to other sick patients while their immune systems were so compromised.

Her advocacy continues on, and she urges patients with CF and other conditions to participate in clinical trials: “It’s worth the chance, it’s worth the risk. It has the potential to transform your life in ways you can’t imagine.” 

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