By Ed Miseta, Chief Editor, Clinical Leader
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As with most people I meet who are involved in patient centricity, Beverly Harrison is passionate about her work. Currently the principal at Harrison Consulting and recent VP of clinical development at the Multiple Myeloma Research Foundation (MMRF), Harrison has over 20 years in the industry, having spent time with both MDS Pharma Services and PharmaNet Development Group.
“In this industry, I believe we always have to ask ourselves what is best for the patient,” she says. “Always keeping the patient in mind will be the surest way for us to improve drug development and get past the old paradigms of working with patients, which have become large obstacles to overcome.”
Despite the years she has spent in the industry. Harrison admits she is still surprised by how difficult it is for many companies to get the patient integrated into the design of trials. Although there are risks involved in doing so, there are also risks in not getting them involved.
Awaiting The Opportunity
Harrison believes patients are anxious to contribute to the trial process, but in the past have simply not had the opportunity to do so. The Internet and social media has changed that. Working through patient advocacy groups, in her opinion, is the best and most efficient way for pharma to interact with patients, get their opinions and feedback, and get them more involved in trials.
“Patients are more than willing to provide information on what works for them and what doesn’t,” she says. “I was invited to a summit at a major pharma company where they were interested in learning how multiple myeloma patients look at data, and specifically filling out forms. This is a disease that primarily affects older people. We administered a survey to them and had a huge response rate. In overwhelming numbers these patients indicated they preferred to answer questions and submit data via electronic means (such as their personal computer). This is a question Pfizer had wrestled with for a long time, not knowing if it would work. For them it was a great insight, and that simple survey got the patient voice back to the sponsor so they could come up with a better way for patients to submit data.”
When pharma and biotech work with advocacy groups, more wins like this can be realized. Harrison notes she has seen an increase in these types of interactions, but they are still not widespread. However, these engagement efforts are what could help move patient participation in oncology trials from 3 percent to 5 or 10 percent, significantly reducing the time to reach conclusions on trials and getting safe and effective products into the hands of patients.
Consider Many Factors Of Involvement
With patient involvement still in the early stages, a lot of learning is not yet taking place. Harrison notes it is important to engage volunteers in providing insight into study feasibility, recruitment, protocol design, and more. If the patient voice is not involved in protocol design, protocols may not make a lot of sense. If that happens, patients are not going to participate, dooming the trial from the start.
There are many factors that should be considered. With many older patients she has worked with, travel can be a problem. For example, patients who live in the northeast most of the year may spend a few months in Florida during the months of January through March. “That situation would have to be considered if they were participating in a trial where their feedback was needed year-around,” she says. “The question then becomes how will you make it as easy as possible for those patients to supply their feedback?”
Other factors that should be considered include education, collaboration, and technology. Sponsors need to consider how to best leverage all the pieces to bring the most benefit to patients. Even once the pieces are in place, companies need to measure how they are performing on all of them. Harrison notes patients are much more savvy than they used to be, and will spend time looking for a study that brings the most benefit to them.
Harrison cites an example from the MMRF. She was performing work on an observational trial and was looking at both clinical data and genomic data. Each patient had to submit a tissue sample which was genomically sequenced and used to create the largest existing database on multiple myeloma patients. It was an open access database that could be accessed by researchers all over the world, which really appealed to patients. “These patients know that even though there have been great advances in the disease, it still does not have a cure,” she says. “They knew this database could help find a cure one subtype at a time. Once they were educated and understood the study, they were willing to participate. I actually had patients calling me and asking how they could participate.”
Friend One Day, Enemy The Next?
Another risk of getting involved with patients is that an advocacy group that is your friend today could be an adversary tomorrow if access to a medicine were denied for any reason. Harrison notes that is definitely a challenge for sponsors. A pharma executive once told her that it took a lot of courage for his company to move forward with an expanded access program to aid patients. Still, it is a risk that pharma needs to take. “The collaborations with advocacy groups will generally help with patient recruitment. It shows you are in touch with the patient and that you’re partners with them trying to find a solution,” adds Harrison. “It is certainly a touchy and delicate relationship, because collecting additional data can complicate your approval process, depending on how the data is collected, organized, and reviewed. This is why it takes courage on the part of the sponsor.”
Even when trying to help patients that have nowhere else to turn, pharma faces additional risks. Expanded use (getting approval from the FDA to allow patients to continue to have access to a drug after it has been submitted to FDA for approval) and compassionate use (one-off requests to use a drug after all other options have failed) are two such methods. While expanded use will allow pharma to collect additional data, there is also the risk of something going wrong. With compassionate use, you are also dealing with a lot of patients who have exhausted all other available treatments and willing to try an experimental medicine. Harrison still views this as a worthy risk since the FDA generally sees it as a positive and it is one way of rallying the patient community behind you.
The best way to handle these partnerships is still for both sides to sit down at the table and have a meeting of the minds. Pharma wants to help patients, but they also have a business interest (getting the drug approved and on the market). The question is how to best meet both goals. If done right, these interactions should always result in greater patient participation and retention in trials. “Everyone involved has to perform a risk/benefit analysis and ask if the partnership makes sense,” notes Harrison. “Hopefully the end result will be a win for both sides.”