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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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.” 

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.

 

Medical Hero Story: Shauna Whisenton

Shauna Whisenton was once an individual living with sickle cell disease (SCD). Now she’s an advocate for better therapies, a cure, and better under-standing of SCD.

Whisenton, now 41, was born with SCD, a painful, inherited disorder where red blood cells are misshaped, restricting blood flow and oxygen to parts of the body.

“It was all going pretty well until I had my third son,” she says. “Then my health started to take a turn for the worse.”

While nursing, she was admitted to the hospital every few weeks and, despite best efforts, was not replenishing enough fluids for her body to function properly and had major organ complications.

Her doctor asked her to consider a bone marrow transplant clinical trial to cure SCD.

Whisenton didn’t believe she could be cured and some members of her family were unsure if she should undergo the treatment. For many in the minority com-munity, there is a fear that medical testing may exploit patients instead of helping them. However, clinical trial oversights ensure safety during participation.

After consulting an SCD patient advocate, she realized, “Although a cure is not guaran-teed, this could be an amazing opportunity. If that is not possible for me, researchers could learn something from my participation to save others.”

Finding a donor

The best chance for a donor match would be a family member. Whisenton lost her parents when she was a child and her sister wasn’t a match; but her 9-year-old son, Dorian, was a 50-percent match.

A successful transplant meant a better life for Whisenton and her family.

“I felt like my children had suffered enough watching my pain,” she says.

Whisenton’s son had marrow extracted from his pelvis bone. It was a one-day procedure for him but the start of a two-year ordeal for his mother.

 
Journey

Whisenton’s journey was tough. She was hospitalized, received anti-rejection medications, and had to undergo was an important part of procedure preparation and recovery. This included coordinated care to provide relief from the symptoms of her disease and the transplant, including pain and detoxing from opiates, but also the physical and mental stress from the procedure.

“It’s important to equip someone who’s received a curative therapy with tools to rebuild their lives during and after recovery,” she says.

Within nine months of receiving the bone marrow transplant, Whisenton was SCD-free and now only carries the trait.

Whisenton calls the date of her transplant her birthday.

 
Patient advocate
 

Whisenton has made it her life’s work to help those living with SCD. She’s the manager of sickle cell disease community engagement for the ASH Research Collaborative (ASH RC) Sickle Cell Disease Clinical Trials Net-work (CTN), established by the American Society of Hematology, the world’s largest professional society concerned with the causes and treatments of blood disorders.

Whisenton is carrying out the mission of the ASH RC CTN to improve the lives of individuals with SCD by expediting the development of new therapies through innovative clinical trial research. Whisenton ensures that the voice of the patient is heard at all stages of these clinical trials.

“SCD warriors, don’t lose hope. There are many treatments in the pipeline to help those living with this disease have a better quality of life. Our hope is that advances in research will bring more effective treatments to individuals living with SCD.” 

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.

Medical Hero Story: Rachel Patties, Congenital Generalized Lipodystrophy Advocate

Oklahoma Mom on a Mission Reflects on Daughter’s Rare Diagnosis

Rachel Petties, a mother of five, refuses to let her daughter’s rare genetic disorder define the lives of their family.

From the moment she first laid eyes on her daughter, Rachel Pet­ties knew something was wrong.

“Alani was all muscle,” she recalls. “She had no body fat whatsoever. Her head size seemed abnormal, and her belly was very large. She was stiff and cried all the time.”

It was the beginning of a diffi­cult journey.

“I ended up dragging my infant baby girl in and out of hospitals, doctor to doctor, specialist to spe­cialist,” Petties says. “You have no idea what loneliness is until you have to pack your child up, hop on a plane and trust someone to help you when you know no one else believes you.”

Receiving the diagnosis
In 2015, Alani was clinically diag­nosed with congenital general­ized lipodystrophy.

“My world crashed, and I’m still recovering from the loss,” recalls Petties, a single mother of five. “Receiving her diagnosis was a bit­tersweet moment – my suspicions were confirmed, but I was very sad because what was wrong with her was worse than I expected.”

Alani, now 4 years old, was enrolled in a long-term clinical trial program at the National Institutes of Health to evaluate her treatment and find improved ways of treating the disease through pre-screening and other medical advances. The hope is to prevent future complications by identifying and addressing problems early on.

“She’s a patient for life,” says Pet­ties. “I’m extremely encouraged. Alani has elite specialists study­ing her case. She’s monitored closely and watched by top profes­sionals in the field.

“We have to travel once a year to Bethesda, Maryland for a host of tests that usually last 2-3 days. It’s a very stressful time for us, but we know it’s necessary.” 

Finding the strength
Petties’ days are long and exh­austing. She must prepare special foods for Alani and is the only fam­ily member trained to administer crucial injections.

“It’s very isolating, and I’m afraid of what will happen if I’m ever physically unable to give her this medicine,” she says.

Restricted mostly to the ind­oors, Alani can be awake for 24 hours and sleep for 15 hours str­aight. Her home must be bleached daily to fight germs, and she must be monitored for exces­sive eating.

“Before gaining some control over this disorder through the clinical trial she’s cur­rently on, Alani would eat the paint off the walls, erasers, socks and soap;’

Petties says Although she’s cut back dramatically to focus on her kids, Petties is committed to getting the word out about lipodystrophy.

“I woke up every morning with the mentality to outrun this disease,” she says. “I ran state to state, city to city, advocating with every ounce of life in me. We marched in Washington and visited senators’ offices, petitioning for their help.

“Sometimes you’ll want to fight, other times you won’t care one bit. I had to teach myself that it’s important to be okay with all those emotions and just get through the day, because every day Alani wakes up is another day this disease didn’t win.”

Alani, who enjoys dancing and performing karaoke, knows she’s different and doesn’t care, accord­ing to her devoted mom. And although weary, Petties herself finds beauty in the darkness.

“I am rare,” she says. “I am a mother of a child who holds a title of 1 in 10 million. That by far is nothing to be sad about. She is incredible and unique, and she’s all mine.” 

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.

Medical Hero Story: Rachel Minnick, Passionate Clinical Trial Volunteer and Advocate

Remembering Rachel Minnick

Rachel Minnick dedicated much of her life as a medical hero. We honor and remember her legacy. 

Diagnosed with stage 2 breast cancer in 2013, the wife and mother of two fought back fiercely against the disease with double mastectomy, chemotherapy and radiation. She participated in a clinical trial. And she worked tirelessly to educate others about their treatment options and the clinical research process.

“Rachel was always pro research, pro clinical trials,” says her husband, Pete Minnick. “She always had that mindset ‘we’re on the cutting edge of medical breakthroughs and technology,’ and she wanted to be on that cutting edge.”

Her cancer was in remission from 2014 to early 2017, until she began experiencing pain in her back and neck. It was at this time that her doctors informed her that the cancer was back and had spread to her bones. “That was a huge blow to us,” Pete says, noting it was stage 4 cancer. ”She knew she wasn’t going to be cured.”

In early 2018, the cancer spread to Minnick’s Iungs and liver. She was actively looking for her next clinical trial when she passed away in April, at age 39, but her legacy lives on.

Meaningful work

Prior to her cancer spreading, Minnick took a position as a Senior Manager of Marketing and Patient Engagement Alliances at The Cen­ter for Information and Study on Clinical Research Participation (CISCRP), a non-profit dedicated to educating the public and patients about clinical research. 

She was passionate about her job for many reasons not only was there a focus on the patient com­munity but it also allowed her to work from her Philadelphia-area home which gave her the oppor­tunity to continue her medical care as well as spend time with the couple’s children, Emily and Sam, now ages 9 and 7.

Being a part of CISCRP was more than a job for her.

“She felt like she was helping the entire clinical trial community as well as doing something she liked,” says her husband. CISCRP gave her the opportu­nity to offer hope to other people who were in her same situation.

Personal experience

“Rachel had direct experience which gave her such an inside perspective,” says her former boss, CISCRP founder, Ken Getz. “It fed her compassion and helped her understand, even more deeply, what so many patients are going through.” Getz says Minnick’s leg­acy lives on through her work; the panels she moderated, the clinical trial awareness initiatives that she spearheaded, her collaborative projects, and the enduring educa­tional brochures, newsletters and other patient communications that she wrote during her time with CISCRP.

“Her passing was truly a shock to me,” says Pete Koerner, a phar­maceutical industry colleague, who worked with Rachel and the CISCRP team for two years.

He described Minnick as some­one who was always enthusiastic, passionate and dedicated to her family, her staff and her work.

“She was invested in clinical research,” says Koerner, explain­ing Minnick was proud to be the patient voice and wanted to advance the techniques and technologies in the clinical trials process.

“She felt like she was helping the entire clinical trial commu­nity.” Getz reflects on Minnick’s lasting impression, “Her compas­sion combined with her profes­sionalism and the passion that she brought to CISCRP has inspired so many people – those who reported to her, those who she mentored, and those with whom she col­laborated. That will stay with us forever. She truly helped to define our culture and evolve it in such meaningful ways.”

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.

Parents & Children Share Perceptions & Experiences with Clinical Research: Survey

During the month of April 2020, CISCRP conducted an online US-based survey among 500 parents and their children. The goal of this survey was to gain insight into general perceptions of pediatric trials, preferred channels of communication, key information parents and their children would want about pediatric clinical trials, as well as past or current experiences participating in pediatric clinical trials.

The findings of this Pediatric Perceptions and Insights Study illustrate what matters most to patients and their parents and offer ways to best support and inform prospective pediatric volunteers and their guardians before, during, and after the clinical research process. You can review the report here.

Understanding and Awareness of Clinical Research High among Parents, but Children were Unsure

Parents generally self-report high levels of understanding about clinical research, and generally high willingness to have their child participate in a clinical research study. Awareness, understanding, and willingness to have their child participate were greatest among parents whose children have previously participated in clinical research.

Among children, levels of awareness of clinical research vary by child’s age, as older children were more likely to have heard of clinical research compared to younger children. Overall, few report that they understand clinical research “Very Well.” Though many were not sure whether they would want to participate, 50% report that they would be willing. The top motivation to participate was altruistic as children wanted to advance science through their participation.

Parents and Children Alike Report Burdensome Participation Experiences Despite Good Communication with the Study Team

During participation, parents report highly burdensome experiences and high levels of disruption to their daily routine. Top burdens included traveling to the study clinic and having their child complete lab work (like blood draws and urine tests). However, the majority of parents said that they received updates or study results once their child finished participation.

Results also indicate that children generally received adequate information about their participation. 92% remembered getting information about the clinical trial before they joined, and 85% found this information ‘Kind of’ or ‘Very Easy’ to understand. Despite these expectation-setting measures, children reported some study requirements as difficult to complete, most notably taking the study medication and undergoing blood draws. However, though children report burdensome study experiences, most indicate that the study exceeded their expectations and they would be willing to participate again.

HCPs Key for Parents to Decide to Have Their Child Participate

A consistent theme throughout the survey findings was the critically important role that healthcare professionals play at several points along the journey toward participation. For example, parents discuss clinical research with their child’s doctors often and cite their child’s doctors as the top way they learn about participation opportunities. Children would also most prefer to learn about clinical research through their doctor. Doctor’s recommendations were ultimately the top reason that parents decide to have their child participate.

You can view other CISCRP reports on clinical research perceptions and experiences here.

 

 

Engaging Underserved Communities in Clinical Research

by Stephanie Loomer, CISCRP Staff

Originally published in Clinical Leader. See link below for the complete article.

The importance of clinical trial research in developing treatments and advancing healthcare is widely acknowledged. In 2019 alone, 46,391 study volunteers contributed to clinical trials that resulted in the approval of 48 novel drugs. Of the demographic subpopulations represented, 9% of study volunteers were Black/African American, 9% were Asian, and 18% were Hispanic, highlighting a lack of representation of underserved populations.1 Recently, the FDA recommended broadening eligibility criteria to enhance diversity in clinical trials and therefore better reflect the patients who will be using a drug once it is approved.2

Pharmaceutical companies have begun working to address these disparities in clinical trials. For example, companies such as Sanofi and Eli Lilly have partnered with patient organizations and medical associations to better connect with underserved communities.3 When identifying study sites, both companies include geographic locations with diverse populations in their searches.  Furthermore, Eli Lilly requires that at least two sites be in diverse locations for larger research studies.3 These are a few of the ways that companies can engage a more inclusive group of study volunteers and better understand how the drug will impact the broader target patient population upon approval.

To read the article in its entirety, please visit Clinical Leader.

CISCRP’s Clinical Trial Results Service: A Tool for Driving Patient Engagement

Patient engagement is no longer just a buzzword in the clinical research industry. It’s a movement that demands attention and concrete steps to take action. One very practical method is to enhance communication with clinical trial participants through the delivery of lay language clinical trial result summaries. Developing these capabilities and leveraging lay summaries can be a powerful tool to add to an organization’s patient engagement toolbox.

CISCRP’s Communicating Trials Results service directly supports the development of lay language summary programs. Our dedicated team of lay language experts, medical writers, and senior editors, work directly with some of the top pharmaceutical and industry companies in the world to assist them in disseminating plain language trial results to their clinical trial participants.

To understand why these communication programs can be such an effective tool for patient engagement, we have identified both ethical and strategic reasons for adding lay language summaries to the clinical research process.

Meet Participant Expectations
Every other year, CISCRP conducts a global survey to better understand patient and the public’s perceptions about the clinical research enterprise. Most recently in 2017, we surveyed over 12,000 people and when asked specifically about receiving trial results summaries, 72% of the respondents reported that they do want a summary of their results, 91% think receiving these results is really important, and 53% reported that they have never received a summary of results. This data supports the idea that participants want to know what was learned from their clinical trial participation and find it to be an important component of the overall experience.

Demonstrate Appreciation 
Lay language summaries, along with frequent communication, signal to patients that they are valued contributors to the clinical research process. There is an ethical obligation to show patients that they are partners in the quest to improve the future of public health.

Reinforce Meaningful Experience
Distributing lay language summaries and other communications through the investigative site staff offers a chance for the site to stay in touch with and engaged with the patient during and after the trial. These important touch points help to reinforce site-patient rapport and provide much-needed and appreciated closure.

Build Public Trust and Research Literacy
Providing lay language summaries is a chance to show a commitment to scientific transparency and increased public visibility. This will help to build public trust in the clinical research enterprise and promote a greater public awareness and understanding of the research process.

Improve Recruitment and Retention
Again citing data from the 2017 CISCRP Perceptions and Insights Study, it was demonstrated that when participants received a summary of their trial results, this correlated to them reporting that the study greatly exceeded their expectations and they were more likely to share the experience with friends and family. In a time when patient recruitment is challenging and low enrollment often delays the start of potentially life-saving clinical trials, it is critical for participants to have a good experience and be willing to share this with others, who in turn may look to also join clinical trials.

Returning trial results is a way to show study volunteers that they are valued and engaged partners in the clinical research process. It’s an essential and practical tool for patient engagement.

To learn more about CISCRP’s Communicating Trial Results service and how to implement your own lay language summaries program, contact Jill McNair, Senior Director of Patient Engagement or Jay Matthews, Business Development Executive. 

By: Rachel Minnick

After the Trial: Using Lay Language Summaries to Close the Loop with Study Volunteers

A growing number of pharmaceutical, biotechnology and medical device companies are closing the loop with their study volunteers by providing clinical trial results summaries. Also referred to as lay language summaries or plain language summaries, the advent of these documents follow research initiatives showing that most clinical trial participants want to know what the research communities learned from their participation, yet most never hear from the sponsor or site staff at all after the trial has concluded.

To accommodate this void and fulfill other objectives including patient engagement and an ethical obligation to return trial results, research sites are increasingly turning to lay language trial results summaries to provide a digestible summary of the study, trial and outcomes. Read on to learn what exactly a lay language summary is, why more and more sites are using them and how you can promptly and easily deliver one to your study participants following your next clinical research trial.

What are lay language summaries?

Lay language summaries are an important emerging practice by sponsors, regulators and other healthcare authorities in effort to increase data sharing and transparency. Written in easy-to-understand lay (aka as plain) language for a general audience, a lay language summary is a non-promotional regulatory document that addresses and thanks volunteers directly and includes information such as:

  • What is happening with the trial now?
  • Why was the research needed?
  • What treatments did the patients take?
  • What happened during the trial?
  • What were the results of the trial?
  • What medical problems did have?
  • How has this trial helped patients and researchers?
  • Where can I learn more about this trial?

The summaries are often written by a third-party organization, guided by a high-quality, patient-focused editorial process and disseminated to volunteers via their investigative site. To accommodate different learning styles, summaries can be developed in multiple formats such as print, electronic and audio.

Why do you need lay language summaries?

There are many reasons to utilize lay language summaries, including fulfilling an ethical obligation to return trial results as well as to demonstrate to volunteers that they are respected as true partners in the clinical research process. While results summaries are not currently required by the FDA, studies consistently show that most volunteers want to know what was learned from their contribution:

  • Participants want to know what was learned from their participation in a trial: 84% of clinical trial participants said it is ‘very important’ or ‘somewhat important’ to receive the results of their trial.1
  • Patients need lay language summaries to comprehend the technical results: 91% of clinical trial participants understood the results of their trial well after reviewing a lay language summary when prior to review, basic facts about the trial were understood by less than 11%.2
  • Lack of communication may negatively impact the clinical research enterprise: 91% of clinical trial participants want to be informed about research findings from their trial and would reportedly not participate in future clinical trials if left uninformed.3

While results are posted to scientific and medical journals and websites such as ClinicalTrials.gov, the writing is not intended for patients or other lay audiences. Rather, the expectation is only that these results would be understandable by an “educated reader of the medical literature,” as stated by Deborah Zarin, Director of ClinicalTrials.gov. Beyond that, other reasons sponsors are planning and implementing summary programs include supporting their patient engagement and retention objectives, nurturing relationships with investigative site personnel, anticipating regulatory pressure and reform, and finally, informing discussion between study volunteers and their healthcare providers.

How do you develop and deliver lay language summaries?

An accurate lay language summary is to be written at a validated 6th-8th grade reading level; when translating highly technical results into scientifically accurate lay language however, this is easier said than done. Most research sites and sponsors choose to work with an independent third-party to write their lay language summaries which includes the following considerations:

  • Inclusion of non-promotional and unbiased plain language
  • Use of a high-quality, patient-focused editorial process
  • Adherence to regulatory compliant methods, including global mandates
  • Use of proper patient protections
  • Adherence to sponsor company specifications and operating practices

The timing of when to distribute the summary to study volunteers is up to the sponsor’s overall strategy – but often site staff will inform volunteers about the summary which can be expected a few months after the conclusion of the study. Available in multiple formats such as print, electronic and audio, a lay language summary is available to volunteers online and often provided in-person.

How do I get started developing lay language summaries for my patients?

If you’re interested in returning trial results and demonstrating to your volunteers that they’re regarded as true partners in the clinical research process, consider lay language summaries. At CISCRP, we’ve partnered with 30 of the top pharmaceutical companies to create and deliver lay language summaries that are non-promotional, unbiased and written in a way that participants will understand and appreciate. To give back to the participants who gave you their time, schedule a free 30-minute consultation with us today.

Do you need other clinical research assistance, education or information? Contact CISCRP today.

Sources

  1. 2017 CISCRP P&I Study, n=12,427.
  2. Getz et al. 2012. Expert Rev. Clin. Pharmacol. 5(2): 149-156.
  3. Sood et al. 2009. Mayo Clinic Proceedings. 84(3): 243-247.

MT Pharmacy: Empty Shelves Demonstrate the Need for Clinical Trials

To View the MT Pharmacy Video, click HERE.

 

Last month, CISCRP collaborated with Sanofi, Inc. to host our flagship clinical research education program, AWARE for All, at Rutger’s University in Newark, New Jersey.  As part of the program, a special pop up pharmacy, named the “MT Pharmacy,” was set up 3 days prior to the AWARE event in an effort to engage with the Newark community.  The purpose of the MT Pharmacy was to show that without clinical trials, pharmacy shelves would be empty, and would not carry the necessary medicines and therapies that are used to treat diseases and ailments for millions of people around the world.  It was also a chance to share information about clinical research and to invite the community to attend AWARE for All.

This special pharmacy project was developed with the assistance of Langland, who initially proposed the concept and idea during CISCRP’s Inspiring Hope Ideathon event held in September of 2016. The idea stood out to CISCRP as a well-suited complement to our AWARE for All events, as both the MT Pharmacy and AWARE for All aim to educate the public about the importance of clinical trials.  We piloted the idea of opening the MT Pharmacy a few days before our AWARE for All Newark event in an effort to reach more members of the local community to create greater awareness of clinical research and encourage them to attend the live AWERE for All event. 

The location for the pharmacy was strategically chosen to be central to downtown Newark and in an area where there is high foot traffic. The temporary storefront was set up to attract passersby’s with the promise of free health screenings and other giveaways. Upon entering the pharmacy, individuals were met with brightly lit walls of empty shelves and barren displays. At first sight individuals appeared curious or even confused. Onsite representatives explained that the empty pharmacy was a demonstration of what our stores might look like without the medicines and treatments that are developed in clinical trials.

The pharmacy was open for 3 days and within that time, hundreds of people were in and out of the store learning about the impact of an empty pharmacy and learning about the AWARE for All live educational event that was being held at Rutger’s University later in the week.  During their visit, people were encouraged to fill out and personalize empty pill boxes with a medicine or treatment that they wished was available. In total 465 boxes were filled out and placed on the empty shelves.

Charlotte Huyton from Langland shared her experience at the pharmacy explaining, “Curiosity drew people into the store; once they entered and chatted with the pharmacist and the team they filled out boxes and shared their story! Many were enthused to hear that a community event was coming to their area and that there was an opportunity to learn more.”  Nearly 120 of the MT Pharmacy visitors also attended our AWARE for All Newark event.

The MT Pharmacy provided a creative platform for creating awareness of clinical research and for building up momentum about our AWARE event within the local Newark community. CISCRP plans to apply initiatives and programs such as this one for future AWARE events. A video of the pharmacy event, including interviews, visitor reactions, and footage of the three days is available to view HERE and will also be made available on www.everyonesinvited.org. This video will be free for the public to view and share.

 By: Leslie Perez