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Three resonating patient-centric initiatives

Friday, July 1, 2016

Sponsor companies are piloting patient-centric initiatives at a furious pace. Although few initiatives designed to engage patients as clinical research partners have achieved widespread adoption, several are making substantial inroads. In this issue, CenterWatch profiles three major patient-centric initiatives that appear to be resonating across the clinical research enterprise.

Insights from these profiled initiatives may guide clinical research professionals in anticipating sustainable patient engagement approaches that appear poised to generate public and patient community ownership in the success of clinical research programs.

Training patient advocates

The move toward a patient-centric drug development model has created the need for patient advocates who can effectively work as equal partners with drug developers and regulatory authorities throughout the drug R&D processes. Patient advocates don’t always have a deep understanding of the various activities required to develop new medical treatments, yet they are increasingly asked for advice about complex issues such as protocol design, informed consent, marketing authorizations or health policies.

In response, the European Patients’ Academy on Therapeutic Innovation (EUPATI), a public-private partnership led by the European Patients’ Forum (EPF), was established in 2012 to provide training and educational resources designed to help patients and their advocates understand drug R&D and become more actively involved during each stage of the process, from preclinical research and development all the way through clinical trials and drug approvals.

“Patients have a unique perspective on their needs and desires in terms of treatments, symptoms, side effects, quality of life and an adequate balance between the benefits and risks of treatment and research,” said EUPATI director Jan Geissler. “A number of studies, for example in CML (chronic myeloid leukemia) and myeloma, have evidenced that there is a significant difference in perceptions between healthcare professionals and patients in terms of severity of symptoms and side effects. Research and development might just end up with the wrong outcomes if patients and their unfiltered needs are not involved in research design.”

The EUPATI initiative, supported by more than 30 European organizations, offers an intensive 14-month training course created to help patient advocates develop the knowledge and skills needed to engage confidently with researchers, drug sponsors and other stakeholders at the European and national levels. Other resources include the EUPATI Toolbox, which includes articles, videos, presentations and fact sheets about clinical research that patient advocates can download in seven European languages, and a comprehensive library of online resources about drug R&D geared toward both patients and the lay public.

“Patients can become better advocates in our discussions with, for example, regulatory authorities and sponsors. If we are at the negotiation table, we need to speak their language. Otherwise, we are not an interesting party for them,” said Marleen M. Kaatee, founder and president of PSC Patients Europe, a foundation focused on finding a cure for a rare liver disease called Primary Sclerosing Cholangitis (PSC) and improving the quality of life for PSC patients, and a 2015 graduate of the first EUPATI expert patient training course. “Patient advocates and ambassadors need to have the capacity and capability to understand how they can collaborate in medical research and development with all the other stakeholders.”

UPATI doesn’t provide information about disease-specific issues or therapies, but rather focuses on processes and issues related to drug R&D and the possible role of patient advocates and organizations in development of new medicines.

By the end of the year, about 100 patient advocates from 12 European countries are expected to have completed one of the two EUPATI expert patient training courses conducted since 2014. More than 200 patients and advocates applied for each session. The course, which is free for participants, requires students to complete six in-depth online modules and attend two four-day in-person training workshops in Barcelona, where participants meet other patient advocates and discuss what they have learned during the course. Participants must pass an exam at the end of each module in order to progress to the next section; those who successfully complete the course become EUPATI Fellows, a designation that is endorsed by the European Medicines Agency (EMA) and the Innovative Medicines Initiative (IMI).

EUPATI keeps a database of its patient experts and helps facilitate patient collaborations with academic and industry researchers, regulatory authorities and ethics committees. EUPATI-trained advocates have participated in patient involvement activities across the drug R&D spectrum, including preclinical research, phase I-III clinical trials, regulatory approval and post-approval activities.

Kaatee believes patient involvement in the drug R&D process should begin as early as possible, beginning with discussing patient priorities in research with stakeholders and how disease affects their daily lives.

“We can be involved in the various phases of the clinical trial, preferably starting with phase zero. When researchers want to start researching something, they can first check with us and see if it’s really something that we want. We can work from there. Especially when you are dealing with a disease without a cure, like PSC, the patients might have another priority,” said Kaatee.

As a EUPATI Fellow, Kaatee has assisted researchers with grant applications for PSC-related projects and advised industry and regulatory stakeholders about inclusion/exclusion criteria that could impact the willingness of PSC patients to participate in a clinical trial. For example, her foundation conducted a survey of PSC patients that found in some European countries, potential participants would not join a clinical trial if a liver biopsy was required. This is critical information when designing a global clinical trial for a rare disease. Kaatee has offered patient perspectives in meetings with Dutch health authorities, the Dutch Clinical Trials Foundation and pharmaceutical companies. Among many other efforts, she participated in European Medicines Agency patient-focused drug development workshops and worked with researchers and patient groups in Europe, the U.S. and Japan.

“I figured out very early on that if I wanted to be at that negotiation table, if I wanted to be an equal partner, I had to have the knowledge. What I see now is that I can really have impact,” said Kaatee. “It’s a great time to be a patient advocate because you can really make a change. But it’s give and take. You need to take responsibility for being an educated patient.”

As patient-centered drug development evolves, Kaatee believes researchers and drug sponsors need to develop a better understanding of how to incorporate patient perspectives into drug R&D and ways to work with patient advocates.

“The researchers also need to be trained as to how to incorporate the patient view and work with patients. There are huge differences among the different patient advocates about experience, training and willingness. For some stakeholders, it’s still just, ‘Let’s invite the patients.’ That is fine. But then they really see the possibilities. It has to grow,” she said.

Easing burdens of participation

New approaches are emerging designed to take into account real-life patient needs and make study participation more convenient. Sponsors and CROs, for example, have used home nursing services and telemedicine to allow patients to participate from their own homes. Companies also are piloting electronic consent forms and wearable devices that can improve study volunteer experiences and make it easier to collect patient-reported data.

The Philadelphia-based Clincierge recently won a 2016 Clinical Informatics News best practices award in recognition for its service, which provides personalized support for study volunteers and their families. The service reduces the unexpected costs and burden of coordinating travel and overnight stays required for some patients to participate in a clinical trial. Employees are located in some 30 countries, called Clincierges or “clinical concierges,” and also provide personal assistance to clinical trial participants, manage study visits and answer questions in the patient’s own language.

Clincierge CEO Scott Gray said offering concierge-type services to study participants can lessen anxieties about trial-related travel and logistical issues that could deter patients from enrolling and completing studies. Ultimately, he said the company’s goal is to reduce overall clinical testing time and costs by making clinical trials more patient-centric.

“For the most part, there is a lack of social interaction in how clinical trials are operated. It’s not a hospitality-themed discussion between the site and the patient,” said Gray. “For true patient centricity, there need to be methods of connecting with patients and removing as much anxiety about participation as possible.”

Trial logistics, including the distance to the investigative site, the frequency of study visits and out-of-pocket costs for travel and meals, remain a significant barrier to patient participation in clinical trials. This is particularly true in rare disease studies where there are typically only a small number of sites in each country able to perform the complex study and patients must be recruited across multiple countries and wide geographic areas. In some cases, patients must travel to another country for study treatment where they don’t speak the language.

“Trial participation can be stressful for patients on many levels,” said T.J. Sharpe, a stage IV melanoma survivor and patient advocate from Florida who relocated for four months in order to participate in a clinical trial. “Any service that can limit the stress to the patient and help get those patients to sites removes one significant impediment to making that match happen.”

Clincierge addresses these challenges by booking flights and hotels for study participants, arranging transport to and from study sites and obtaining health care coverage for medical visas when required. The company also issues participants debit cards for travel purchases in order to reduce out-of-pocket expenses. In-country coordinators or clinical concierges address any special patient needs and communicate with study volunteers and their families throughout the clinical trial process. To provide these services, Clincierge leverages the travel and hospitality experience of its parent company, Gray Consulting International, which for the past 22 years has specialized in providing meeting and event planning services for pharmaceutical and life sciences companies.

“We get our Clincierges through our established network of global meeting and hospitality professionals. They are very customer-service focused. It has been described as a white-glove service,” said Gray. “It’s treating people like people rather than subjects or guinea pigs.”

In one case, a biopharmaceutical company contracted Clincierge to provide patient support services for a clinical trial in a rare disease area that was being conducted in 18 countries. The study enrolled 120 patients required to travel long distances to study sites every 21 days for up to 33 visits. A few of the patients lived in Poland and needed to fly to London for treatment, but none of these Polish volunteers spoke English.

In order to support the logistics of this global trial, Clincierge employed 17 clinical concierges who lived in the participating countries and collectively spoke 20 languages. These coordinators made all of the travel and hotel arrangements, which included transporting patients to airports or study sites. Clincierge covered major travel and hotel costs up front, spending more than $900,000, and reimbursed patients for minor expenses, such as meals, quickly through an electronic payment system. If Clincierge hadn’t handled the travel planning and logistics, patients would have been required to spend an average of $8,400 up front for trial-related costs and wait for reimbursement following their visits. Similarly, Clincierge saved patients and caregivers each an average of 32 hours that it would have taken them to book travel, housing and other trial logistics on their own. The model also relieves clinical research site staff from spending time on arranging patient logistics for a clinical study.

Gray said Clincierge’s support of this global trial helped maintain a drop-out rate of less than 5% over the 22 months the study has been ongoing. Moreover, he said the cost of incorporating Clincierge services has averaged less than 1% to overall clinical trial costs.

The company, which began assisting its first patient at the beginning of 2014, has since supported more than 600 patients in 32 clinical trials. Clincierge coordinators offer services in 23 languages across the globe.

Gray said the model can be applied to studies other than those for rare diseases. Clincierge already has supported patients in neurologic and sleep studies. The company also is working on proposals to transport and accompany Alzheimer’s patients to study visits and on an initiative that could increase compliance in a juvenile diabetes study in urban U.S. cities by using a car service to transport young patients to investigative sites.

Communicating trial results to study volunteers

Among the initiatives meant to strengthen the important role patients play in the research process are those that communicate clinical trial results to study volunteers in everyday language. About 30 of the top 50 biopharmaceutical companies, including Pfizer and Eli Lilly, have collaborated with The Center for Information and Study on Clinical Research Participation (CISCRP) to share lay-language research findings with clinical trial participants and show their appreciation for the study volunteers making scientific advances possible.

CISCRP’s Communicating Trial Results program, which began in 2011, was designed to help study participants learn about the results of their trial, build a stronger connection with volunteers after their participation ended and convey that study volunteers are respected by the research community as partners in the development of new medicines.
“Transparency at every stage of the research process leads to greater patient engagement,” said Jill McNair, senior director of patient engagement at CISCRP. “There are so many opportunities throughout the clinical research process to engage the patient. The more we can do it, the better.”

CISCRP’s lay-language summaries, which have been translated into more than 90 languages and distributed in 40 countries, are aligned with Pharmaceutical Research and Manufacturers Association (PhRMA) and European Federation of Pharmaceutical Industries Association (EFPIA) transparency commitments. New clinical trial regulations in the European Union (EU) also will require drug sponsors to provide both a technical and lay-person summary of results for interventional trials, which will be posted on an EU-wide public registry within one year of the trial’s conclusion.

CISCRP research has found that most study volunteers (90%) want to find out about their specific clinical trial results, yet most never hear from the research sponsor or study staff after their clinical trial ends. As a result, many volunteers have reported that they feel unappreciated by researchers and question whether their participation contributed to medical science.

“Our data shows that when patients are in a clinical trial, they form a very close bond with their study staff. When the trial is over, this bond is broken abruptly. They might get a handshake and a thank-you, and then they are out the door and they are never seen again,” said McNair. “What we have seen in our research is that if patients are given the results of their trial in plain language that they can understand, if they receive follow up, their experience is enhanced and the bond is reinforced. These volunteers are more likely to participate in the future and they will stay engaged as partners and ambassadors in the clinical research enterprise.”

Many research sponsors have piloted trial results programs with studies that are either completed or nearly finished, yet McNair that said ideally the process should begin during study initiation. This ensures that the results communications process is integrated into study planning and budgeting and involves study staff at the beginning of the trial. With this scenario, during the informed consent process, study staff initially informs study volunteers that clinical trial results will be made available after the study ends. During their last visit, volunteers receive a thank-you note and a reminder that the lay-summary results will be mailed to them. Most sponsors that provide trial results summaries through the CISCRP program also have post-trial updates sent every six months since study volunteers may need to wait years for a summary while results are being analyzed.

Many drug sponsors initially have concerns about legal and regulatory issues related to sharing lay-language trial results with study volunteers. A barrier to wider adoption has been the risk that lay summaries may be perceived as promotional or biased, despite the intentions of the sponsor company. The Food and Drug Administration (FDA) Amendments Act of 2007 provisionally requires sponsors to post clinical trial results written in non-technical, understandable language for patients on ClinicalTrials.gov. However, no final ruling has been made on the provision. The decision has been delayed, in large part, until the government can demonstrate that the summaries can be consistently produced without being promotional or misleading. Another common concern among sponsor companies relates to protecting patient privacy when distributing the lay summaries.

The approach CISCRP has developed to address these issues begins with the technical results summary prepared by the sponsor for government registries, such as ClinicalTrials.gov or the EudraCT system. CISCRP, in its role as an independent, nonprofit organization, organizes an objective editorial panel that includes medical and consumer health communications experts, patient advocates and specialists who translate the technical findings into everyday language following a template developed through extensive user testing. The summaries, which are validated at a plain-language reading level, include brief descriptions of the trial’s design, objectives and findings. Sponsor researchers and staff members provide a final review for accuracy. CISCRP then has the summaries printed and shipped to study staff, who mail the reports to study volunteers.

CISCRP’s method is designed with multiple checks and balances that ensure the results translations are non-promotional and scientifically accurate. It also separates the sponsor from patients through the involvement of the investigative site and a patient-focused third party (i.e., CISCRP) with no vested interest in the study outcome. The program was recognized in 2013 with a Human Research Protection Best Practice award by the Health Improvement Institute.

CISCRP recommends that investigative sites mail study volunteers a printed, hard copy of the clinical trial summary results as a tangible show of appreciation. CISCRP also provides summary results in electronic and audio formats to accommodate different learning styles. CISCRP is working with a number of sponsors to provide plain language summaries though dedicated web portals.

Questions have been raised about whether lay-language trial result summaries should be reviewed by ethics boards. An institutional review board or ethics committee approvals all communications received by the study volunteers while they are actively participating in the study. McNair said it has been CISCRP’s experienced that most ethics committees don’t expect to review the lay-language summaries once the trial has ended since they reflect publicly available information being provided to people who are no longer in a clinical trial. Many ethics committees, however, indicate they appreciate receiving a copy of the communication for their records. McNair said ethics committee policies and regulatory guidelines will need to confirm and clarify their position going forward.

CISCRP anticipates that efforts to communicate trial results to study volunteers will become standard practice within a few years. As the patient centricity movement continues to evolve, the organization expects that sponsor companies will combine trial results programs with broader, enterprise-wide initiatives to engage study volunteers and restore trust in the clinical research process.

CenterWatch will continue to monitor patient centric initiatives to identify and differentiate those that resonate as sustainable initiatives to broadly support clinical research programs and those that are more appropriate for select clinical research studies.  


Karyn Korieth has been covering the clinical trials industry for CenterWatch since 2003. Her 30-year journalism career includes work in local news, the healthcare industry and national magazines. Karyn holds a Master’s of Science degree from the Columbia University Graduate School of Journalism. Email karyn.korieth@centerwatch.com.

This article was reprinted from Volume 23, Issue 07, of The CenterWatch Monthly, an industry leading publication providing hard-hitting, authoritative business and financial coverage of the clinical research space. Subscribe >>

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