Pros & Cons of DCTs & Virtual Clinical Trials

From "The Gift of Participation" by Ken Getz, Founder & Board Chair, CISCRP

They are known by different names: DCTs (decentralized clinical trials), remote, direct-to-patient, virtual, digital, site-less or simply patient centric clinical trials. All of these approaches share the common goal of making it easier to participate in research by reducing—or eliminating altogether—the number of study visits patients must make to conventional investigative sites or labs and allowing for more flexibility in carrying out study-related activities. Many of these approaches use smartphones, mobile devices, and wearable sensors to collect and evaluate patient data during the study.

Study volunteers often need to travel long distances to medical facilities, many need to stay overnight in a hotel, and take time off work to participate in a conventional clinical trial. Research from CISCRP shows that about one-fifth of study volunteers find clinical trial participation stressful and report the investigative site location and time-consuming study visits are among the least-liked aspects of the experience. Half of volunteers also feel that participation causes disruption to their daily routine. New, more convenient approaches are especially valuable for patients who may be too sick to travel or for those who rely on caregivers for support. Study volunteers who find it difficult to fit additional medical appointments into an already busy schedule or those who live far from the investigative site and wouldn’t otherwise be able to participate in the trial also benefit.

Not every clinical trial currently offers study volunteers an in-home or remote option, and it will take quite some time for a large number of trials to be done this way, but use of these approaches is expected to increase as pharmaceutical and biotechnology companies invest more widely in efforts to improve the clinical trial experience for patients. Research sponsors and regulators are working on initiatives that better take patient needs into account and could eventually allow patients to participate in clinical research wherever and whenever they want, whether it be their own primary-care doctor’s office, home, workplace, school, or anywhere else.

You shouldn’t feel forced to participate in a remote or at home study if you live near an investigative site and would prefer to have a face-to-face relationship with the study staff.

Pros:

  • You won’t need to travel and make frequent visits to an investigative site.
  • You’ll spend less time in a medical office.
  • You can participate in telemedicine visits at a time convenient for you, perhaps in the evening or on weekends.
  • You can contact someone on the research team 24 hours a day.
  • You may feel empowered by being able to participate whenever it is convenient to do so.

Cons:

  • You won’t have the same number of face-to-face interactions with study staff.
  • If you have a technical problem, you have to reach someone on your own to resolve the issue.
  • You’ll need to make sure you’re home to sign for clinical trial-related deliveries.
  • You may be asked to take your own vital signs or perform tests several times a day.
  • You may need to travel to a lab or medical facility for lab work or exams.
  • You may be asked to collect samples and arrange for them to be picked up.
  • You’ll likely need to send back all of the loaned devices and monitors at the end of the trial.
  • Not all wearable technologies have been validated, so you may need to repeat tests or travel to the research center for a special assessment.

When deciding whether a home-based or remote clinical trial is right for you, after you’ve learned as much as you can about the study visits and what activities you’ll need to perform on your own, discuss the pros and cons with your family, friends and primary care physician. It’s best to ask for input from people you know and trust and to involve your support network in your decision-making process.

For more information on decentralized clinical trials and making informed decisions about volunteering for clinical research, read “The Gift of Participation” by Ken Getz, Founder and Board Chair, CISCRP.

You can find the book here.

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.

Out of the Dark: A Journey Through Postpartum Depression, Part 2

Authored by Melissa E. Daley, Communications & Marketing Manager, CISCRP

& Emma Kane, Clinical Research Coordinator, Clinical Operations & Development, Sage Therapeutics

NOTE: CISCRP hosted a 2-part webinar series on postpartum depression (PPD), in collaboration with Sage Therapeutics, Inc. This is Part 2 of a 2-part article series, providing an overview of the second webinar, which focused on PPD, clinical research, and shared perspectives from a PPD survivor/patient ambassador, medical professionals, and a patient advocate. You can access Part 1 of this article series here (link to be created).

“The best-planned life can turn as quickly as a rainstorm on a summer’s day,” shares Chelsie, a PPD (postpartum depression) awareness ambassador and moreover, a survivor. Married to her high school sweetheart, she had accomplished many goals on her way to starting her family: she loved her job as an elementary school teacher, had purchased a home and was financially stable. “Everything fell into place according to plan. Teaching students was my passion, but motherhood was my calling,” explains Chelsie. Three years into their marriage, she and her husband were delighted to learn that Chelsie was expecting. Her pregnancy was typical until her 36th week, when she was diagnosed with pre-eclampsia and put on bedrest until her delivery.

Chelsie gave birth during a storm, “…amid tornado sirens and pounding rain,” she recalls. “Even more miraculous, the sun broke through and created a glorious rainbow just minutes after our son Weston was born.”

But almost immediately, Chelsie felt that something was wrong.

She did not feel an instant connection to Weston and she realized her maternal instinct was not taking over. Instead, Chelsie’s mind was swirling with questions about her baby’s welfare, as well as her own. Her anxiety escalated when her first attempts to nurse Weston were unsuccessful.

Chelsie did not know it yet, but she was beginning her journey with PPD.

A few days later Chelsie returned home with Weston. “I was physically weak and emotionally drained.” Everyday tasks were overwhelming. She was nervous, and her baby could feel her tension. “I was wracked with anxiety. I didn’t feel like I could even hold him,” says Chelsie.

A week after returning home, Chelsie was rushed to the emergency room with a racing heart and unresolved pre-eclampsia. Recognizing that Chelsie needed sleep, her OB/GYN prescribed a sleeping pill. Chelsie and her mother hoped rest would help. Still, she felt that her mind would not rest. “The next night would prove to be the start of the worst time of my entire life. I cried until I couldn’t cry anymore, and what came next was an utter lack of emotion. I knew I wasn’t myself, and I spoke up,” Chelsie shares.

“With my mom, dad, and husband in the living room, I explained I felt no connection to Weston. I felt like a terrible person and mother,” says Chelsie. Her family consoled her as best they could and suggested she speak with a cousin who had suffered with PPD. Her cousin advised Chelsie to call her OB/GYN. Chelsie’s OB/GYN had never treated anyone with PPD, and recommended a psychiatric facility.

“I felt nothing,” Chelsie says, as she left her six-day old son in her mother’s care. After an evaluation at the hospital, she voluntarily checked herself into a psychiatric hospital where she was officially diagnosed with PPD. She was prescribed medications to help with anxiety, depression, and insomnia.

“The longer I stayed (in the hospital), the worse I got,” says Chelsie. She was transferred to a second hospital.

In the span of 30 days, Chelsie had given birth, been diagnosed with PPD and had been a patient in two psychiatric hospitals. But there were glimmers of hope. “Even when PPD broke me down, I was gently reminded of a higher purpose for my life,” recalls Chelsie. Her parents found the right help for Chelsie, and eventually, with an adjustment of medication and the assistance of a mental health professional, Chelsie began to recover.

“I opened up to my doctors and therapists and we built a relationship where I could tell them anything. They helped me find solutions for whatever I was dealing with,” says Chelsie. Her recovery was challenging, but with assistance from her family, she began to focus on the victories she was starting to have as a mother. Returning to teaching scaled Chelsie’s confidence as well. She joined a local PPD support group and met other mothers navigating the same issues.

“Power in community is so precious. The key to my recovery was not a simple one. I needed the right therapy, medication, and family and friends to support me. Recovery takes time. I love my son more than I can put into words. PPD is nothing to be ashamed of or embarrassed about,” says Chelsie.

Chelsie is active in a non-profit organization focused on serving and building community with moms along with providing them with resources for PPD, regardless of their ability to pay.

There is an unmet need in treating PPD. Dr. Bassem Maximos, OB/GYN, explains that “A lot of physicians don’t recognize it (PPD) and even if we do recognize it, it’s difficult to find resources to point our patients to get the treatment they need.”

 Standard-of-care options for the treatment of PPD can be outlined in three categories.

“It takes a long time to find that therapist that you can trust, work with, and open your heart and mind to allow them to help you in your journey,” says Dr. Maximos. “It’s an ongoing treatment,” he adds, “and also sometimes you need other supportive therapies with it.” He also explained that unfortunately, it can be challenging to find a therapist, depending upon the community where you live and the resources that are available. In some regions, there are not many mental health professionals, and the few that are in the area may have so many patients that they are not able to assist new ones.

While it may be challenging to find these groups, Dr. Maximos stresses the importance of supportive psychotherapy and mother-infant therapy groups. “We’ve found in our practice if we get moms to see other moms that have gone through the same experience she’s going through, or have experienced PPD, they are more willing to open up and talk about their experiences – it takes away the fear or stigma.”

Peer support can also be found through groups active on social media when it’s difficult to find professional support. The COVID-19 pandemic has scaled the importance of this channel, with the need for social distancing measures to limit the spread of the virus.

“We’re still not screening our moms enough,” says Dr. Maximos. “The American College of Obstetricians and Gynecologists has been putting a lot of effort into encouraging obstetricians and gynecologists to screen for postpartum depression during the pregnancy and afterwards by giving them screening tools.” But there is still progress to be made.

There are organizations working to escalate access to support mothers suffering with PPD.

Tonya Fulwider, Associate Director for Mental Health America of Ohio and Program Director for POEM (Perinatal Outreach & Encouragement for Moms), says “We work with frontline providers, OB/GYNs, pediatricians and home health professionals. We have a team of certified peer support specialists who work as care navigators. We serve as that single-entry point of care for any birthing person who is struggling with a mental or emotional health complication.” The healthcare provider can reach out to the POEM team and directly connect the patient to receive assistance from a care navigator or peer

support specialist. They can offer a menu of options to address their needs, “…recognizing that she is the expert in her life,” says Tonya.

Sage Therapeutics has conducted multiple clinical trials in PPD over the past several years, and is currently conducting a trial studying an investigational oral tablet to treat PPD. A full listing of clinical trials for women with PPD is available at clinicaltrials.gov.

If you’re considering participating in a clinical trial, Dr. Gus Alva, a psychiatrist focusing on neuropsychiatric conditions with over 27 years of clinical trial experience, advises “Always be informed.” He explains that in his practice, they provide patients with a consent form, a standard clinical trial document which explains the trial in detail and confirms a patient’s willingness to take part in the study, and have them  take it home with them. They encourage patients to discuss it with their primary care doctor or other professionals in the medical field. “The consenting process,” he adds, “entails pros, cons, alternatives, and being well-educated.”

There is a regulated process that takes place in not just PPD clinical research, but in all clinical trials.

“There are several inclusion and exclusion criteria that are tied to all studies,” explains Dr. Alva. Inclusion and exclusion criteria are requirements that must be met in order to take part in the study. “Going in and getting screened would certainly be a good option.” He adds, “the nice thing about being involved in a clinical trial is that it’s not binding. There’s no penalty to withdrawing consent at any point. It’s a matter of trying to figure out whether this is a good fit or not, but not necessarily feeling that you are now bound to it.”

Individuals considering clinical research should consult the medical professionals involved in their care.

Dr. Maximos explains that before signing up for a clinical trial, “patients should speak to their physician, whether it’s their OB or psychiatrist.” Clinical research sites also conduct outreach efforts to community healthcare providers to raise awareness about clinical research participation opportunities.

“Be a question-asker,” advises Tonya Fulwider, saying that the care navigators she works with often encourage patients to write questions down as they come up so they can address them with their health care providers, whether it’s their OB/GYN, mental health professional or someone on the clinical trial team.

COVID-19 has impacted clinical trials across the board. Very strict guidelines have been put in place at all clinical trial sites, such as taking temperature readings, requiring masks, social distancing, and disinfecting of surfaces continually.

“Individuals that do come in know that we’ve placed into play a lot of different modalities to ensure that there is appropriate disinfecting, minimal touching of door handles or other objects, and that if they’re coming in, there’s a very specific purpose,” Dr. Alva explains. “We take every precaution possible to keep patients safe,” Dr. Maximos adds, including that his entire staff is now vaccinated in addition to the other precautions they’ve put into place over the course of the pandemic.

“Of course, COVID-19 has impacted everything about everyone’s lives, and it certainly has impacted mothers,” says Tonya. “Being able to continue to converse with them and talk about their concerns –   that’s really the most important thing.”

To learn more about postpartum depression, access the webinar series, “Postpartum Depression & Being Informed” here. You can also learn more about the Skylark study for PPD here, or access resources from Sage Therapeutics here.

ABOUT SAGE THERAPEUTICS

Sage Therapeutics is a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating disorders of the brain. We are pursuing new pathways with the goal of improving brain health, and our depression, neurology and neuropsychiatry franchise programs aim to change how brain disorders are thought about and treated. Our mission is to make medicines that matter so people can get better, sooner.

ABOUT CISCRP

CISCRP (Center for Information and Study on Clinical Research Participation) is a Boston-based, globally focused, non-profit 501(c)(3) organization providing public and patient education and advocacy.  CISCRP’s mission is to inform patients and the public about clinical research and the important role that it plays in advancing public health and to help stakeholders in drug development engage with patients and the public as clinical research partners (www.CISCRP.org)

Out of the Dark: A Journey Through Postpartum Depression, Part 1

Authored by Melissa E. Daley, Communications & Marketing Manager, CISCRP

& Emma Kane, Clinical Research Coordinator, Clinical Operations & Development, Sage Therapeutics

NOTE: CISCRP hosted a 2-part webinar series on postpartum depression (PPD), in collaboration with Sage Therapeutics, Inc. This is Part 1 of a 2-part article series, providing an overview of the first webinar, which focused on PPD, and shared perspectives from a PPD survivor, medical professionals, and a patient advocate. You can access Part 2 of this article series here (link to be created).

“I did not want to move. I did not want to breathe. And I did not want anyone to know,” explained Alexis, about her experience with postpartum depression (PPD). PPD is one of the most common medical complications during and after pregnancy[i]. PPD is more severe in scope than the “baby blues” that many mothers experience, which generally occur within the first few days of delivery and resolve without treatment in 2 weeks[ii].

Symptoms of PPD may vary, but can include:

  • Changes in mood, including sadness, emptiness, hopelessness, irritability, persistent doubt, feeling overly anxious and thoughts of suicide
  • Changes in the body including fatigue, difficulty sleeping or sleeping too much, aches and pains, and changes in appetite
  • Changes such as trouble concentrating, remembering details or making decisions
  • Social differences, such as withdrawing from family and friends, and distress or impaired ability to functional in social or work settings[iii]

“Clinically, we basically say that if someone has had an episode of depression during pregnancy or the postpartum year, we’re going to call it postpartum depression,” explains Dr. Constance Guille, Associate Professor, Department of Psychiatry & Behavior Sciences; Director of the Women’s Reproductive & Behavioral Health Program, MUSC. “Postpartum depression and baby blues are two very distinct entities.”

It’s estimated that about 50-80% of woman are affected by the “baby blues” after childbirth[iv].

“The symptoms are not severe, and they do not impact activities of functioning in any way,” explains Dr. Guille about the baby blues.

Alexis had an unexpectedly difficult delivery, but her daughter was healthy. From the outset, she had problems bonding with her baby. “I looked at her like I was holding an alien from another planet,” Alexis recalls. Initially, she blamed it on exhaustion, but it quickly became apparent to her that there was something more serious happening. With the support of her husband and parents, Alexis sought medical assistance. She ended up switching psychiatrists and medications many times, and nothing seemed to be working. Alexis voluntarily admitted herself to a psychiatric hospital where she remained for a month.

“At one point, I was sitting and waiting to see the doctor, when I overheard the social worker telling the counselor that I was faking it,” says Alexis. “I slid even further into the abyss. If they did not believe me, who would?”

After she left the hospital, her depression deepened.

“I struggled with thoughts of not wanting to be alive anymore,” says Alexis.

The stigma around experiencing PPD is real. However, there are healthcare providers who are working to change attitudes and perceptions around postpartum depression and individuals suffering with it. One approach adopted by medical professionals is to routinely ask about a patient’s mental wellness, both before and after delivery.

“You see a patient during her pregnancy about 20 times,” says Dr. Jason James, OBGYN, Private Practice in Miami. “For me to ask how she’s doing, how is she coping, is she feeling down or depressed, is she feeling anxious or guilty, all the kind of non-judgmental questions – if I ask it all the time and get lots of “No’s”, it normalizes the situation.” Routinely checking in with a patient about her mental health can reinforce her comfort level about transparently sharing any changes she may be experiencing.

“The more that people can share their stories, the more that other people can look at that person and say, ‘Oh gosh, I’m just like them!’, the better off we’ll be,” says Dr. Guille.

Providing a robust familial and social support system to postpartum mothers is key to assisting them in their recovery.  “Having a baby is emotionally, psychologically, physically and financially depleting,” says Dr. Guille.  Finding time to relax, getting enough sleep, and ensuring proper nutrition is essential.

Heather Dopp is a patient advocate and Mom Ambassador with 2020 Mom, a national organization with a mission to close gaps in maternal mental health care. She is a mother to two children. Her advocacy work is spurred by her own experience as a survivor of perinatal anxiety, depression, and suicidal ideation during her second pregnancy. Heather’s advocacy has taken her from the halls of the Utah State Capitol to the United States Capitol, with the purpose of improving awareness and increasing resources available to mothers facing maternal mental illness within the United States.

“From a non-clinical point of view, I have an acronym we like to use with maternal mental health. It’s SUNSHINE, and it’s a great way to remember the basic things you can help with for the mother struggling, so she can manage everything she is going through and hopefully try to thrive,” says Heather.

“For many women that I have talked to across the United States, we don’t know about postpartum and perinatal mental disorders in the first place. When it happens to us, we’re already experiencing ‘mom guilt’, and we don’t want to own up to it, because maybe we’re the only ones… we haven’t heard our friends talk about it, and we haven’t heard our providers mention anything about it.” says Heather.

Other patient barriers to discussing symptoms include:

  • Mistrust and fear of being judged
  • Lack of insurance coverage
  • Time constraints
  • Access to childcare during postpartum visits
  • Lack of awareness regarding impact on own health and infant health
  • Lack of information regarding where to seek treatmentv

Alexis changed physicians and treatments many times and took the time to do research and learn everything she could about PPD given there were very few medical professionals specializing in PPD in her location. Eventually, Alexis found a counselor and psychiatrist who supported her. They developed a treatment plan to best address her symptoms and over the next year, she was able to begin the healing process.

“I began to truly laugh and smile again. Life became brighter, more colorful and the darkness began to drift away,” Alexis recounts. Her daughter has no recollection of Alexis’ struggle with PPD, and they are very close.

Alexis says that PPD has taught her five important lessons:

  1. Never give up; postpartum depression doesn’t last forever.
  2. Don’t be embarrassed. Reach out and find a support system.
  3. Self-care is essential and it doesn’t have to cost money. It can be as simple as reading a book or taking a bubble bath.
  4. Do your best not to feel guilty.
  5. Tell your story, when the time is right, because it can help you heal, and also help others.

“I did not give up. I made it out of the darkness. And through the pain, I found a strength and courage that was always there, but needed some prompting to come out,” says Alexis.

To learn more about postpartum depression, access the webinar series, “Postpartum Depression & Being Informed” here. You can also access resources from Sage Therapeutics here and from 2020Mom here. Your healthcare provider should always be your primary source of information about diseases or disorders and treatment options. Please contact your healthcare provider with any questions pertaining to a medical condition.

ABOUT SAGE THERAPEUTICS

Sage Therapeutics is a biopharmaceutical company committed to developing novel therapies with the potential to transform the lives of people with debilitating disorders of the brain. We are pursuing new pathways with the goal of improving brain health, and our depression, neurology and neuropsychiatry franchise programs aim to change how brain disorders are thought about and treated. Our mission is to make medicines that matter so people can get better, sooner.

ABOUT CISCRP

CISCRP (Center for Information and Study on Clinical Research Participation) is a Boston-based, globally focused, non-profit 501(c)(3) organization providing public and patient education and advocacy.  CISCRP’s mission is to inform patients and the public about clinical research and the important role that it plays in advancing public health and to help stakeholders in drug development engage with patients and the public as clinical research partners (www.CISCRP.org)

Sources:

[1] Ko JY et al. MMWR Morb Mortal Wkly Rep. 2017;66(6):153-158.;  Martin JA et al. National Vital Statistics Reports; vol 68 no 13. Hyattsville, MD: National Center for Health Statistics. 2019.;  DeSisto CL et al. Prev Chronic Dis. 2014;11:E10.; Centers for Disease Control and Prevention. Updated June 12, 2018. Accessed May 5, 2021 https://www.cdc.gov/reproductivehealth/maternalinfanthealth/diabetes-during-pregnancy.htm; Centers for Disease Control and Prevention. Updated February 28, 2019. Accessed May 5, 2021 https://www.cdc.gov/reproductivehealth/maternalinfanthealth/pregnancy-complications-data.htm; Roberts JM et al. American College of Obstetricians and Gynecologists Task Force on Hypertension in Pregnancy. Obstet Gynecol. 2013;122(5):1122-1131.; Callaghan WM et al. Am J Obstet Gynecol. 2010;202(4):353.e1-e6.

[1] 1. National Institute of Mental Health. Accessed May 5, 2021. https://www.nimh.nih.gov/health/publications/postpartum-depression-facts/index.shtml; American College of Obstetricians and Gynecologists website. Frequently Asked Questions: Postpartum Depression. Accessed May 5, 2021. https://www.acog.org/patient-resources/faqs/labor-delivery-and-postpartum-care/postpartum-depression; Thurgood S et al. Am J Clin Med. 2009;6(2):17-22)

[1] American Psychiatric Association: Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition. Arlington, VA: American Psychiatric Association, 2013.; National Institute of Mental Health. Accessed May 5, 2021 https://www.nimh.nih.gov/health/publications/postpartum-depression-facts/index.shtml)

[1] Thurgood S et al. Am J Clin Med. 2009;6(2):17-22)

v Prevatt BS et al. Matern Child Health J. 2018;22(1):120-129.; Byatt N et al. Arch Womens Ment Health. 2013;16:429-432.; Santora K et al. NIHCM Issue Brief. 2010. Accessed May 5, 2021 https://www.nihcm.org/pdf/FINAL_MaternalDepression6-7.pdf; Goodman JH. Birth. 2009;36(1):60-69.

From the Editor

Written by Brandis Pickard 

Dear Readers, 

We’re pleased to share with you our summer (yes, summer!) edition of the CISCRP newsletter. It’s hard to believe that just a few months ago, most of us were living in some form of quarantine, and COVID-19 vaccinations had just become available for groups deemed most at risk. As CISCRP brings you our second newsletter of 2021, there’s anticipation in the air that as more of us become fully vaccinated, we can celebrate a “normal” summer. While we embrace the chance to resume the activities we love, we know the pandemic will leave a lasting impact and that we have an opportunity to inform our approach to clinical research based on what we have learned over the past months. 

One of the things we have come to appreciate most is the importance of human connection. Whether it’s the sting of not visiting our loved ones or the challenge to have our voices heard when we aren’t in the same room as our colleagueswe have all encountered and conquered new obstacles over the past year. This has been especially true when it comes to healthcare. For those with limited access to technology or information, the burden has been particularly heavy. As we noted in our winter newsletter, and what we all need to remember as we move forward: the pandemic has underscored existing racial and economic disparities that we must acknowledge to remedy.  

In this issue, we highlight how two supporters of our Patient Diversity Campaign plan to identify and address diversity challenges in clinical trial participation. Not surprisingly, communication is a key factor in their approaches. Honest communication—making that human connection—is critical to improving health literacy and education. It’s something that Melvin Mann, this issue’s Medical Hero, touches on when reflecting about clinical trial participationPlease follow the link to read Melvin’s inspiring story about fighting chronic myelogenous leukemia. 

When thinking about transparency in health communication, a good place to start is with the development of the COVID-19 vaccines. In this newsletter, you’ll find an article that highlights how CISCRP is answering important questions about the COVID-19 vaccines—not only how they were created, but which populations were included in the clinical trials and what is being done to monitor safety. 

We’ve also included some thoughtful insights from patients about the difficulties of clinical trial participation during the pandemic, and the solutions identified—some of which will likely change the way future trials are conducted.  

Finally, were happy to share our own successes in maintaining our human connections and engaging the community in important conversations about clinical research. Read on to learn about our first regional AWARE for All  webinar, our Medical Heroes  Appreci-a-thon, and our Third Annual Plain Language Summary of Trial Results User Group Meeting. 

As always, thank you for your ongoing support in initiatives like these, and in all that we do. We wish you a happy and healthy summer. 

Regards, 

Brandis Pickard 
Senior Manager, Editorial 

 

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Medical Hero Spotlight: Melvin Mann & Chronic Myelogenous Leukemia (CML)

Melvin Mann with his wife, Cecelia, and their daughter, Dr. Patrice Mann

Written by Melissa Daley

“CML (chronic myelogenous leukemia) is supposed to be a disease that strikes people in their sixties, and I was in my thirties,” recalls Melvin Mann. A family man and Army major, Melvin was stunned to learn he had CML at the age of 37.

His symptoms had started out as back pain and fatigue, which he had been treating with medication and physical therapy, to no avail. In a visit with his physician at a local military clinic, an MRI was ordered requiring Melvin to drive several hours to another military base that had the MRI equipment. It would be a month before Melvin learned the results of the full-body scan.

It was January of 1995. Melvin received the diagnosis of CML, which was confirmed by a second opinion he sought. Melvin was advised by his doctor that without a successful bone marrow transplant, his life expectancy was about three years. The best bone marrow matches are with a patient’s relatives, but if they are not a match, a donor must be of the same ethnicity. No relatives matched, nor did any registered donors. At that time, there were a limited number of African Americans on the bone marrow donor registry. Melvin began the treatment prescribed by his physician, which were daily injections of interferon.

Melvin thought about his wife, Cecelia, and his daughter, Patrice, who was just five years old and decided to take action in the search for a donor. Working with marrow donor organizations, and the Department of Defense (DOD) Marrow Foundation, marrow drives were launched within the military. Melvin participated in these outreach efforts, which scaled to include college campuses, malls and churches, radio and television interviews, as well as military bases. His experience as an Army recruiter and public relations officer and degree in public relations propelled him to forge ahead, even when he didn’t find a match. Melvin medically retired from the Army in 1995 and continued his outreach efforts with bone marrow drives, as his health would allow.

At a marrow drive organized by his aunt in April of 1996, Melvin had a life-changing meeting with a local businessman who had seen Melvin’s TV promotions for the event. This individual had been gravely ill with hairy cell leukemia and had been treated at MD Anderson Cancer Center in Houston, Texas, with excellent results. He urged Melvin to contact them.

Thus began Melvin’s experience with clinical trial participation. It had been 16 months since his diagnosis of CML, and none of the medical professionals involved in his care had broached the topic of clinical trials. The bone marrow drives he was involved with were finding donors for many patients, but not for him. Melvin decided that participating in a clinical trial was his next step towards fighting the illness.

Over the next two years, Melvin traveled between his home in Atlanta and Houston, trying different combinations of vetted and clinical trial medications through MD Anderson. At the outset of the Imatinib clinical trial, he had to stay in Houston for three months. Over time, the interferon and other clinical trial medications stopped working. A lifelong competitive runner, his body weakened and he suffered intense fatigue. He was, however, eight months past his original prognosis of surviving three years, when he had the opportunity to participate in an Imatinib clinical trial. The Phase I trial tested for the drug’s safety in humans, dosage level and evidence of efficacy.

The Imatinib clinical trial had three locations: MD Anderson, Oregon Healthy & Science University in Portland and UCLA Medical Center in California. Melvin’s medical care continued to be seated in Houston.

“It was for quality of life – not a cure. I was hoping for a better outcome,” Melvin explained. He also thought about how his participation would help other cancer patients.

The process was rigorous. In the first week, Melvin had blood drawn up to 10 times per day. He was required to keep a detailed record of side effects, the time he took the medication, his energy level, diet and other daily details. He experienced severe nausea and was prescribed medication to alleviate it. Melvin’s effort, perseverance and grit paid off. The drug was a game-changer for him. In June of 1999, nine months after starting the clinical trial, Melvin participated in fundraising events for the Leukemia & Lymphoma Society by running a marathon in Alaska and a few months after that, cycling 111 miles in Tucson, Arizona.

Melvin has been taking Imatinib ever since. The medication was approved for use in CML in May 2001 by the United States FDA (Food and Drug Administration). Melvin participated in another clinical trial to see if he could come off the medication, but he could not. Over the past 26 years, Melvin has participated in three clinical trials for different combinations of medications.

“Trials have changed over the years,” said Melvin. “The last (Imatinib) trial I was in was in 2017. I had to travel every three months to the trial site, although some testing was done at home. The COVID-19 pandemic has increased that whatever you can do at home or with a local doctor, in terms of aspirations and blood draws.”

For some patients, particularly members of the Black and African American communities, the specter of the unethical Tuskegee Syphilis Study still influences opinions about the safety of clinical research, despite vast advances in oversight and regulation made in subsequent years by the United States government to ensure the safe and ethical treatment of all patients. It is important that patients are asked if they would like to participate in a clinical trial.

Melvin advises patients and the public that “It’s vital to know that clinical trials are an option. There can be benefits from a clinical trial. You are getting more attention because the studies often require you to be watched more and seen by staff more frequently. In oncology clinical trials, you’re not getting a placebo, you’re at least getting the standard of care treatment. It’s important to weigh your options. Can you get the support you need from family or a caregiver? Ask questions of the study staff. There are risks involved, so ask about them. You have the choice to stop being in a clinical trial, whenever you decide.”

Melvin is now the world’s longest living Imatinib and tyrosine kinase inhibitor CML survivor. Melvin had a MBA, but in 2000 he decided to change his career focus. He returned to higher education and earned an BA in English Literature, M. Ed. In Secondary English and a teaching license, all within the span of three and a half years. He and Cecelia continue to volunteer for various cancer and bone marrow donation organizations. Melvin continues to participate in marathon and half-marathon events, and sometimes 10Ks with his wife and daughter, Dr. Patrice Mann, a psychiatry specialist, graduate of Harvard College and Emory Medical School.

“I recommend clinical trials, at least to ask about them. It’s also important for doctors to ask patients if they want to participate in clinical research,” said Melvin.

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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The Role of Clinical Trials in COVID-19 Vaccine Development

Written by Behtash Bahador, MS 

As COVID-19 vaccinations are rolled out, some people still have questions and concerns about the safety of the vaccines. How were they made so fast? How do we know they work and are safe? Were they studied in a diverse group of people? 

To help address these questions, CISCRP developed an infographic: “The Facts About COVID-19 Vaccine Clinical Trials.” The overall goal of this piece is to provide engaging educational content to people who may still be deciding if they or their family will get vaccinated.  

To complete the project, we brought together a team with years of experience sharing clear, unbiased, nonpromotional messaging about clinical trials. To create the infographic, we reviewed news and information from trustworthy sources.  These included national and global health organizations, universities, and peer-reviewed publications. This helped us learn what people still want to understand about the COVID-19 vaccine trials, and what else they may need to know.  

Then, the hard work began: we had to decide which topics and messages would be covered in the limited space of an infographic, and how. In the end, the main take-aways we aimed to provide are:  

  1. Teamwork and years of knowledge and experience allowed the vaccines to be made quickly. 
  2. Trials have shown the available vaccines are safe and effective. 
  3. The vaccines will continue to be studied and monitored for safety.  
  4. The COVID-19 vaccine trials included more diverse participants than many other trials. 
  5. There was diversity among the many professionals involved in vaccine development.  

As with all of our health communications, we received feedback from an editorial panel of patients, advocates, health professionals and members of the public. This was an essential step to make sure the infographic is clear, is not missing important information, and is not misleading in any way. We hope that the end result is an informative, easy-to-understand snapshot of some key elements of the vaccine development and rollout. 

The full infographic can be viewed on this page in CISCRP’s Education Center. The page also includes the sources used to create the infographic and additional links to useful educational content.  

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AWARE for All Northeast

Written by Hope Ventricelli | Manager of Events and Community Engagement

For the first time, CISCRP brought its AWARE for All program to regional areas across the United States while remaining virtual. The AWARE for All program was created to raise awareness about the clinical research process and to provide education among diverse communities. The webinar for the first edition of this series, AWARE for All — Northeast, went live through the virtual platform ON24 on April 15th. For those who wish to watch on-demand or continue their learning, a recording of this webinar is available through the Informational Exhibit Center (IEC) found on the CISCRP website. The IEC features educational content from organizations across the Northeast, spanning multiple states and therapeutic areas. The IEC is designed to be easily accessible and to address the lack of knowledge about the clinical research and health resources in the audience’s “own backyard.”  

The CISCRP Events Team brought in more than 40 exhibiting organizations for the series’ opening event, from clinical research sites to community clinics and local health centers. Each shared free educational resources with the audience in the IEC. While several groups offer information about the trials they offer, most exhibitors simply aim to provide general education or medical information about specific health conditions. This helps to include those not as familiar with research and to make them feel comfortable learning more. Attendees cited their desire to learn more about research as their main reason for registering. Many also shared that they have a friend or loved one with a health problem.  

The IEC had an impressive number of viewers. More than 1,800 new and returning community members visited the virtual educational booths and the new Health and Wellness Pavilion, which features health tips and exercises.  

Through post-event surveys, audience members shared that their biggest takeaway was that they better understood the clinical research process and were grateful to have their questions answered in real time. As to their favorite part of the program, most attendees referenced the panel discussion. 

Through the panel discussion, AWARE for All — Northeast focused on key health issues such as oncology, Parkinson’s Disease, asthma, and the importance of diversity among trial participants. Outreach to minority health groups helped to bring a diverse audience from the local population to hear more from the speakers themselves. The lively discussion, which was moderated by CISCRP’s own Behtash Bahador, included several study participants as well as local researchers and industry stakeholders. These experts shared their individual experiences and answered questions the audience most wanted to know. 

Each virtual AWARE for All program aims to bring the research community, patients, and the public together to support a discussion in understanding clinical research and, most importantly, the role of study participants. 

Including the Northeast edition, CISCRP’s AWARE for All program will virtually host in 5 different regions through 2021. Future programs include Northwest on May 20th, Midwest on July 22nd, Southwest on October 21st, and Southeast on November 18th.  

To learn more about exhibiting at AWARE for All, contact hventricelli@ciscrp.org. 

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Supporter Spotlight: Patient Diversity Initiatives

CISCRP is developing a series of articles about initiatives from pharmaceutical companies to address racial inequities and health care disparities with a focus on clinical trial diversity.   

For our first Supporter Spotlight piece of the series, we feature two companies—Merck and Pfizer—our 2021 “Patient Diversity Campaign” editorial content providers.  

The COVID-19 pandemic has heightened awareness about the importance of clinical research. The pandemic has also placed a spotlight on racial inequalities within our U.S. health care system. Over this past year, we’ve seen many companies increase transparency by publishing clinical trial results summaries, hosting advisory boards, supporting mass media awareness-building campaigns, and collaborating with local communities.  

We met with colleagues from Merck and Pfizer to learn how they’re understanding critical barriers to participation and increasing health literacy among patients and the public.  

Here are three key insights from these two industry leaders that showcase their commitment to improving diversity and inclusion: 

Develop solutions that resonate among different stakeholders.  

“There are multiple barriers to diversity in clinical trials and multiple stakeholders who have the responsibility to address them,” shared Luther Clark, Deputy Chief Patient Officer at Merck. “We are strongly committed to conducting research that is safe and effective in all patients.” To demonstrate this commitment, Merck executes their clinical trials with a focus on meaningful patient and community engagement, addressing the social determinants of health, logistical and financial barriers, and minority investigator shortages.

One way Merck works to remove barriers and ensure equitable access to clinical trials is through heat map technology. This allows them to identify patients with the greatest disease disparity and need for medication. Adrelia Allen, Director of Clinical Trial Patient Diversity at Merck, shared the importance of this technology and of working closely with sites and community leaders. “[Prioritizing diversity and inclusion] is a part of the fabric of our corporate culture. It’s embedded into our clinical trial operations,” said Allen. 

At Pfizer, effective communication with community members is a priority. “Clinical trial accessibility starts with clinical trial awareness,” shared David Leventhal, Senior Director of Clinical Trial Experience at Pfizer. “There’s a difference between reading about clinical trials on the internet and really understanding a clinical trial as a care option.”  

Both Pfizer and Merck support CISCRP’s Perceptions & Insights bi-annual study to understand public and patient perceptions around trial participation and barriers. Both companies are writing full-page editorials about their commitment to achieving health equity in CISCRP’s Patient Diversity 2021 campaign.  

Learn about what matters most to patients and their families.  

Pfizer incorporates patient insights into their protocol designs and engages with patients and local advocacy groups. “We’ve learned that being a part of an alumni network is important to our participants, so we’ve expanded that around providing individual results, data return, and an opportunity to share stories,” said Leventhal. They also partner with local communities and work with advisory boards to ensure clear, consistent messaging.  

At Merck, organizing advisory panels, partnering with advocacy groups, and hosting forums helps gather direct input from patients. In addition, Merck is committed to improving health literacy around the world to advance health equity, impact population health outcomes, and drive efficiencies in health care systems. 

To develop best practices to increase diversity, Merck and Pfizer also work with major consortia, such as CISCRP’s AWARE Industry Consortium, PhRMA, the Diversity & Inclusion in Clinical Trials (DICT) Collaborative, and the Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard. 

Maintain relationships with local communities.  

“With educational awareness programs, we’re showing that we’re there to support communities,” said Allen. Merck has supported external grass-roots programs, such as CISCRP’s AWARE for All events and the Lazarex Cancer Center’s equitable access initiative. 

Clark added, “The first step is recognizing and overcoming mistrust, fear, and lack of comfort with the clinical trial process. Ultimately, it’s the patient who will make the decision about participation, but they’re influenced by people close to them—their families, friends, and the greater community.” 

Pfizer supports CISCRP’s AWARE for All programs and developed an FAQ on pfizerclinicaltrials.com to increase transparency and address concerns among underserved communities. They develop their educational materials in partnership with academic centers and advocacy groups to build trust, cultivate strong collaborations, and ensure best practices in health literacy. Pfizer also partners with large health systems to avoid multiple data collection mechanisms and support interoperability.  

Taking the time to understand participation barriers and meet the needs of participants is an investment in the future. 

“It’s not a sprint; it’s a marathon,” said Allen. “We’re willing to put forth the effort and resources to improve patient outcomes and achieve health equity for all.” To learn more about Merck’s commitment to diversity in clinical trials, click here. 

Tune in to the next article of the series where we’ll be profiling other companies!  

Author: Ellyn Getz, MPH, Associate Director, Business Development 

 
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Endpoint Selection in Plain Language Summaries: Insights from CISCRP’s Third Annual User Group Meeting

Written by Kim Edwards

On February 19, 2021, CISCRP hosted our Third Annual Plain Language Summary of Trial Results Virtual User Group Meeting. This meeting provides an opportunity for sponsors to participate in facilitated discussions about evolving or challenging aspects of developing plain language trial results summaries (referred to here as “PLS”). This year, topics included adverse events/reactions reporting, translations, dissemination, posting deadlines, and endpoint selection.  

In particular, the discussion about endpoint selection was spirited.  The group addressed the ongoing debate about whether it is appropriate to include secondary endpoints in PLS, and how this decision should be guided and documented. The biggest challenge with including secondary endpoints is striking a balance between providing the reader with relevant results of interest while also avoiding “cherry-picking” that leads to perception of bias. To avoid this perception, some sponsors advocate for clear guidelines across all plain language summaries within their organization. For example, some companies have created a policy to include only the results of the primary endpoint, regardless of the nature of the trial. Other sponsors suggest that this is not necessarily a patient-focused approach, as there may be secondary endpoints that are of particular interest to the trial participants.  

To get a snapshot of the current practices among our User Group participants, CISCRP asked the following poll question: Does your organization have a documented endpoint selection process and/or policy for plain language summaries? Interestingly, the responses, presented in the graph below, were quite evenly distributed.  

These results highlight the lack of a standard approach to endpoint selection. Later this month, we expect the release of a paper from industry stakeholders that offers suggestions on endpoint selection. In the meantime, we offer these three considerations: 

  1. Audience – Do your trials include secondary endpoints that participant readers will care about and want to learn about? 
  2. Cherry-picking – Can your organization implement secondary endpoint selection criteria across all trials and/or require protocol authors to indicate participant-relevant secondary endpoints prior to data collection? 
  3. Feasibility – Will a standard approach be practical for those in your organization who must operationalize it?  

We are grateful to the participating sponsors for their insights, collaboration, and dedication to providing participants with meaningful plain language trial results.  

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CISCRP’s Third Annual Medical Heroes Appreci-a-thon

Written by Justine Holleran

Throughout the month of March, CISCRP hosted its 3rd annual Medical Heroes Appreci-a-thon to bring together a community of almost 350 “racers” to express their appreciation for all “Medical Heroes”—medical and healthcare professionals, researchers, and clinical trial participants. 

This month-long virtual fitness challenge encouraged racers to go the distance to recognize Medical Heroes by tracking any form of exercise—cycling, running, yoga, yard work, pet-walking, and more. Any physical activity was translated into “miles,” and racers and their teams could virtually track their progress through a trail of U.S. national parks. 

Racers were able to stay active, strike up friendly competitions, and participate in fun weekly challenges all while learning about clinical research and reading stories about the Medical Heroes that brought them all together for this event. 

Appreci-a-thon attracted 346 racers and 90 teams. Our athletes collectively covered 29,647 miles of the course. Racers participated from almost all 50 U.S. states, Russia, England, Japan, Belgium, Germany, and more! With the support of our sponsors and racers, we were able to exceed our fundraising goal for this program. All funds support important CISCRP initiatives such as our educational events, appreciation programs, and more.