Disruptors In Clinical Trials: How Can Pharma Assimilate Empowered Patients?

By Ed Miseta, Chief Editor, Clinical Leader

Innovation is shaking up the world of clinical trials and creating new challenges for clinical professionals. A panel of thought leaders at the CROWN Congress in Philadelphia addressed this topic and shared their insights. Part one of this article focused on how technology is disrupting clinical trials. In this piece, the topic shifted to empowered patients and the role they will play in driving change in clinical trials.

Participants in the panel, moderated by Elizabeth Mascherino, Advisor, Clinical Innovation at Eli Lilly, included:

• Al Altomari, President and CEO, Agile Therapeutics
• Andy Lawton, Global Head, Clinical data management at Boehringer Ingelheim
• Paulo Moreira, VP of Global Clinical Operations  - External Innovation at EMD Serono
• Mark Travers, Global Head of Monitoring Excellence and Interim Head of North America Clinical Operations, Merck

More Power In Groups

There is no question that patients have a greater knowledge of their diseases than ever before. Moreira notes he has seen patients evolving over the years, but despite that evolution, the awareness of trials amongst patients still seems to be incredibly low. Still, the rise in the use of the Internet by patients has been incredible, and the information available has changed the way patients look at their own health.

“One critical change we are seeing is patients recognizing that by getting together and forming groups they have a voice and can become empowered,” he notes. “This movement started with AIDS patients in the 80s and more recently we see it happening in the breast cancer community as well. This empowerment movement is something the industry will have to be able to deal with.”

But Moreira also points to what he calls the exposed patient. These are patients who seem to have no concerns about posting their medical records online and commenting on their health status almost daily. Their goal seems to be to use their own personal data for the betterment of others. This has also led to some patients and groups trying to raise money for their own clinical trials.

“They are no longer waiting for us,” he says. “They get some information, they get some funds, and they get the ball rolling. The ALS community is one example. Those patients do not have time to wait for us and have chosen to act on their own. It would be a mistake for the industry to try and ignore them. We need to work together with them so they can help us to develop better drugs.”

Their Job Is NOT Clinical Trials

So what exactly is an empowered patient? “To me, the empowered patient is someone who is connected, not just to the Internet, but to other patients via a patient advocacy group, blogs, or discussion groups,” says Travers. “They are knowledgeable about their disease and current treatments, and are aware of current and future research. Oftentimes they are also lobbying the government or their local representatives to get access to treatments. But what we all have to realize is that these individuals also have a life, a family, and a job. And their job is not participating in clinical trials.”

Travers notes many impaired patients do not live near a major hospital. In some cases, they do not even live in or near a major city. They want to participate and be involved and connected, but they want to do it on their own terms and not have to travel far. As a result, they are interested in participating remotely and in using new technologies, including smart phones, apps, and wearable technologies. 

In order to meet the needs of these patients, pharma has to be prepared to work with them when the patients decide the time is right. “That may mean around the clock facilities for clinical trials,” says Travers. “There is a great untapped potential here for investigators willing to work outside regular business hours. As an industry conducting clinical trials, we really need to change and start moving in the same direction as consumers. You can now order a pizza, buy a book on Amazon, or check your savings account balance 24/7. This is the new model that consumers have come to expect. If we are not willing to bend to meet their needs, is it any wonder they refuse to participate in clinical trials, or choose to withdraw from them?”

Will Empowered Patients Lead To Fearful Sponsors?

With patients becoming more knowledgeable and empowered, is pharma left feeling inspired or fearful? A few years ago the mother of a young patient started a Twitter campaign that became one of the largest the industry had ever seen. It attacked a company that would not provide the child with access to an experimental drug. Celebrities got involved and eventually it toppled the company’s CEO. Something like that can leave an industry feeling befuddled and fearful.

Altomari addressed the topic best. “We do not know how to deal with that, and as a result it will probably happen again” he states. “Patient groups are mobilizing and we are just starting to see them getting it together. They are funding trials and talking to politicians and even the FDA. Patient groups will soon be telling us what is going on in universities. We need to be talking to them, forming relationships with them, and learning from them. They are an amazing resource and we need to work with them in a much more collaborative way. If we don’t do it, or don’t do it well, we will find ourselves back in the headlines.”

Creating better patient centric efforts within pharma will also be important. At most sponsor companies, Moreira notes an effort is underway to place an increased focus patients. “We are all trying to build a culture that is trusting and puts the patient at the center of everything we do,” he says. “But we also realize we can’t allow scientific merit takes a back seat. The secret is to find that intersection where patient centricity and scientific merit merge, since companies will not be successful having one without the other.”

EMD Serono is currently using patient advisory boards to provide feedback on clinical trial design. This has led to discussions with patients on topics such as the number of required visits, the number of assessments within each visit, and the appropriate time between visits. “This has enabled us to provide services to patients that others have told us are important to them,” he says. “It can be something significant, such as periodic updates on their progress over the course of a trial, as well as courtesies such as a parking space in front of the building, valet parking, or significant trial milestones.”

At the end of the trial, Moreira also recommends thanking each patient and providing feedback on the trial. Constant engagement is important to patients, and they now have networks where their voices can be heard. They are not afraid to talk about your trial and how you are treating them.

“It is important for us to figure out ways that will help trial leaders ease into the implementation of new technologies and innovations,” he adds. “I will occasionally work with our trial leaders to implement at least one new thing on their trial. This allows us to gain great insights into what is and isn’t working. Sponsors cannot afford to fall into a rut of always assuming that something new is not going to work. Some things will and some things won’t. Even something that didn’t work in that particular instance might have other applications. We must all try to keep an open mind when it comes to new ideas.”