Why Are More Patients Not Sitting At Your Decision Table?

By Ed Miseta, Chief Editor, Clinical Leader

Although patients are learning more about clinical research and getting more involved in clinical trials, they oftentimes are still not invited to meetings at the trial design level. This occurs, despite the fact that both sponsors and patients have valuable information that could be shared with each other.

The whole topic of patient centricity is really about bringing people together,” says Paul Wicks, VP of Innovation for patient network PatientsLikeMe. “Although engaging patients early on in the design process is still in the early stages, listening to patients as if they were partners is going to be a significant paradigm shift for the industry.”

The trial design process involves answering a lot of questions, such as what should go into a trial, what should be measured, what logistical difficulties will be faced, and how many patients should be recruited. There are a lot of decision-makers involved in answering those questions, including executives, scientists, clinicians, and representatives from legal. Ironically, the only stakeholders who generally seem to be missing from that table are the people who are going to benefit from the medication, who will decide whether or not to take part in the study, and will then decide whether or not to stick with it.

Why has pharma not gotten patients more involved in the trial design process? And why is the adoption of this paradigm not moving ahead at a faster pace? Wicks believes there are three primary reasons.

WHY CHANGE WHAT YOU’RE DOING?
“I believe the first reason is inertia,” says Wicks. “When something has always been done a certain way, it is very easy for people to keep doing it the same way. I think there is also a belief that some of those internal stakeholders speak on behalf of the patients, particularly the physicians. But while physicians are clearly experts in the disease and the treatments, they are not experts in having to live with the disease. A clinician may have views on whether or not a pathway, molecule, or side-effect might be disruptive, but they are not going to have insight into how a woman with a job and a child in school will be able to fit weekly clinical visits for an MRI scan into her schedule.”

Asking executives, regulators, or scientists to try and understand a trial from the patient perspective might be an impossible task. However, with the digital technologies currently available, it is easier to get patients to answer those questions themselves. “Imagine a cell phone company trying to design a new device for young people and doing so without talking to the users,” notes Wicks. “Or perhaps they just talk to the parents of the users. Parents might be able to discuss all of the features they would like to see on the device, but their opinion would be a poor proxy for the ultimate user. While that model is a nonstarter in the consumer world, in clinical trials it is simply how we have always done things.”

LEGAL, SAFETY, AND REGULATORY ISSUES
Another reason sponsors might not interact with patients relates to legal, safety, and regulatory issues. These are definitely real issues, according to Wicks. If a patient showed up and started talking about the side effects of a drug, that information would have to be reported to the drug safety department and reports would have to be filed in a timely manner. There are also issues around companies potentially paying patients to take part in surveys or sitting on advisory committees. Sponsors have to be careful not to cross any lines, particularly if the drug is not yet on the market.

Wicks notes there are also issues around patient confidentiality. If a sponsor speaks to 200 patients about a trial they are designing, there is a possibility that confidential information shared by patients might leak out somewhere. That possibility has to be properly managed from a risk point of view.

“There are some real concerns here,” says Wicks, “but fear of those issues might also be used by some companies as an excuse to not talk to patients. This is a real challenge to some companies that want to better engage with patients but have legal and regulatory personnel that are always quick to say no. From their viewpoint, engaging with patients is simply not worth the risk.”

Wicks disagrees with that point of view. He notes sponsors need to take a closer look at the legal, regulatory, and safety risks that exist from not engaging with patients. There is certainly risk of launching a product no one wants. There is also risk in designing a trial that no one signs up for, or giving trial patients a drug that they refuse to take. To navigate this dilemma, he states each company will have to develop its own playbook to ensure all employees are taking the appropriate precautions. Every company will likely have a unique approach, as one size will not fit all.

WHAT IS THE APPROPRIATE METHODOLOGY?
Once companies get past the internal inertia and legal/regulatory blockades, they will need to develop an appropriate methodology for interacting with patients. This can also be a challenge for many sponsors. If you are preparing to move into a Phase 2 trial and want more patient involvement, what exactly do you need to do?

There are a multitude of ways to elicit feedback from patients, including market research surveys, ad boards, social media listings, and more. But Wicks notes there is a playbook to be followed for each of those options. You would need to know who to commission them from, how much they cost, how effective they have been in the past, and more. Even when you decide on the best option to reach your targeted patient population, you will have to decide whether to speak to one or 50 or 500. Other questions you will need to answer are:

• How many times should you meet with them?
• How much information should you share?
• Should you have them sign a contract?
• Should you pay them and, if so, how much?
• Should you hire someone to be your advisor?

These are all unknowns that have to be considered before engaging. When considering these questions for the first time, they can be confusing and complicated, especially when undertaking them in a large organization. Without the support of management, this project will be an uphill struggle.

CHOOSE THE RIGHT ADVOCATE
Once you overcome these resistance challenges and formulate a plan of action, finding the right person to head up your patient advocacy efforts is not going to be easy. The people who seem to have the best skills at interacting with patient advocacy groups typically lie on the commercial or marketing side of the house. They tend to be nonscientific, do not get involved with research, and are generally not invited to trial design meetings. The M.D.s and Ph.D.s who do get invited to those meetings are used to reading scientific journals and performing scientific tasks. Drop in someone from patient advocacy who has spent most of their time providing grants to nonprofits and charities, and you often end up with three sides that do not even seem to be speaking the same language.

“The challenge for sponsors is figuring out how to reconcile these disparate world views,” says Wicks. “We are basically trying to create a patient-engagement science, three words that do not generally go together. We need to take the opinion of one patient and turn it into a replicable method that can be run by another sponsor, CRO, or university with similar results each time. For many sponsors, that is a brave new world.”

A PLAN FOR SUCCESS
To get patients more involved in the design of clinical trials, there will clearly be many challenges to overcome. In the long term, Wicks would like to see two things occur to make the vision more of a reality. The first is patient-centric decision making being made earlier on in the drug-development process. “This should not just occur at the clinical trial level,” he says. “Patients need to be brought onboard even at the point of deciding what molecules to select from the portfolio and push forward into human trials. Patients should have a voice in deciding where the real unmet needs are.”

Second, he would like patient participation to move up what is called Arnstein’s Ladder of Citizen Participation. The ladder shows who has the power to make important decisions. It goes from nonparticipation at the lowest level, to tokenism, and then citizen control at the highest level. Wicks would like to see more instances of patients being consulted and actually given responsibilities during the course of a trial. As an example, he cites a recent trial for breast cancer where 40 or 50 participants were added to the advisory board for the trial and given responsibility for making translations of the recruitment material.

“From the patient perspective, there is a huge difference between someone from a drug company asking you for help versus the company recruiting and training you and giving you some amount of responsibility for the success of the study,” says Wicks. “As an industry, we need to move from consulting people to actually working with them as partners. At the extreme end of this it could involve drug companies actually employing patients in advisory capacities. I believe this will be necessary to overcome the three barriers. There will no longer be that negative inertia because this effort will be done internally. We will have created a playbook that takes us through the minefield, which will give all stakeholders more confidence in the methods.”