From The Editor | July 25, 2017

5 Best Practices For Engaging With Patient Advocacy Groups

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader

5 Best Practices For Engaging With Patient Advocacy Groups

Patient recruitment has always been a challenge for companies conducting clinical trials. But when you are developing treatments in the rare disease space, finding patients to take part in your trials becomes an even greater hurdle to overcome.

One of the ways pharma and biotech companies can reach patients and learn about the challenges faced by those individuals is patient advocacy groups. But knowing how to properly engage with these groups can determine whether they will treat you as a trusted partner or a necessary evil. So, how do you know what they hope to gain from the relationship?

Fibrocell is one such company involved in the rare disease space. According to CEO John Maslowski, Fibrocell is a gene therapy company focused on skin diseases, primarily those caused by genetic disorders that impact children. Cells from a patient are genetically modified and reintroduced to the patient to enact change in a disease and alleviate the symptom.

“We are currently working on a blistering genetic disorder called Recessive Dystrophic Epidermolysis Bullosa (RDEB),” says Maslowski. “It has a high mortality rate in patients by the time they reach their twenties. That product is currently in a Phase 1/2 clinical trial and dosing has begun. We are also working on a treatment for localized scleroderma, a disease that affects the surface of the skin with lesions. We are hoping to find a treatment that will reduce disfigurement and restore the patient’s mobility. The second program is still in the preclinical phase. Both have been granted Orphan Drug Designation and Rare Pediatric Disease Designation [by the U.S. Food and Drug Administration].”

If you are hoping to work with advocacy groups in order to gain insights from patients, the relationship you have with those groups will be critical to the success of the effort. Maslowski believes there are several tips companies should keep in mind when trying to engage these groups.

1. Focus On Building Trust

For the RDEB indication, there are two main advocacy groups that Fibrocell works with. Maslowski notes his team took the initiative to reach out to both of those groups. “We are a small biotech company,” he says. “We are not a well-known company like Merck or GSK.”

If you work for a Big Pharma company, most advocacy groups are aware of your company and the products you are working on. For a small biotech company, you may not be on their radar. “Our visibility is lower than it is for the bigger companies,” says Maslowski. “That being the case, we could not sit back and wait for patient groups to come to us. In those instances, you need to feel comfortable reaching out to the groups and pursuing a relationship with them.”

In approaching the groups, Maslowski likes to reference his company’s solutions and explain why they might work well for the impacted patients. He believes these relationships work best when you can build a level of trust right up front.

“Advocacy groups do not always trust the pharma companies that approach them,” states Maslowski. “The individuals in these groups are used to working with physicians and researchers in academia. Those are also the folks who make up their scientific advisory boards. When someone approaches them from the pharmaceutical industry, it’s normal for them to think you might be trying to profit from their patients.”

For that reason, Maslowski likes to take the time to explain what Fibrocell is attempting to accomplish, what its challenges are, and what it is hoping to learn from the advocacy group. One of the primary focus areas is patient needs. The advocacy group is in the best position to understand the challenges and needs of patients, that can make for a constructive learning experience for the pharma company.

“We can create a treatment that will help patients with their wounds,” notes Maslowski. “But what you might learn from the advocacy group is that patients have a greater concern over the pain or itching that might occur as a result of the wound. If that is what is really impacting their life, it should also impact your product profile. With rare diseases there are no set clinical models already in place. Therefore, advocacy groups can help you understand what those models should look like and what will be most important to the patient and their quality of life.”

2. Understand Each Other’s Focus

Maslowski has found that different advocacy groups can each have a different focus, even if they operate in the same therapeutic space. For example, some will place a focus on patient care. They will focus on raising money to purchase supplies for patients or to pay for hospital stays for patients or travel expenses to get to those places of care. Others will simply raise money for research into the disease. There may even be some groups that are a hybrid of those two models.

Regardless of the type of advocacy group, Maslowski believes it is important to understand each group’s focus. Since each will have different goals, you need to understand those goals before you engage the group. If you are trying to gain their trust, the worst thing you can do is make a presentation without fully understanding the mission of the group. Those different angles may also require you to speak to them differently.   

While it is important to understand the intentions of advocacy groups, it is also important for them to understand yours. Maslowski recommends clearly explaining your intentions to them so both sides clearly understand what each wants to get out of the relationship. Are you looking to gain patient insights? Understand the patient condition? Recruit patients? If you tell the group one thing and then do another, you will have destroyed the trust you are trying so hard to create.

Once you understand a group’s focus, you can also better understand how to help them. Fibrocell will support groups by attending their events and providing support via financial donations. The company will also make personnel available to speak at advocacy group conferences in order to provide updates on research to patients and caregivers.

3. A Different Patient Recruitment Process

With a commercialized product, the focus for pharma tends to be on creating a large sales force, which will then focus on finding doctors who have access to patients in need. That is not how it works in the rare disease space. For example, in the U.S. there are only 16 centers that treat patients with RDEB. That makes the patient recruitment effort very different.

“We work with Stanford University on this disease,” says Maslowski. “A location like that already has a registry of RDEB patients. That means much of our recruitment effort will involve working with Stanford and their patient registry. But as we expand our trials, we will need access to more patients, and that is where the advocacy groups can help out. That type of outreach effort can be far more effective than providing leaflets in a physician’s office.”

Advocacy groups will generally make use of social media, and they also conduct a lot of outreach to patients, especially via email. Those outlets can help to spread the word about what a pharma company is trying to accomplish, provide information on treatment options, and share updates on ongoing or upcoming clinical trials. Patients or caregivers interested in getting more information on a treatment or clinical trial would then have the option of reaching out directly to the company.

4. Get The Right People Involved

When a large pharma company decides to connect with a patient advocacy group, it might send a slew of patient centricity officers to the initial meeting. Many will even have personnel who are dedicated to building relationships with these groups. Again, the scenario is a bit different in the rare disease space.

Fibrocell is a small company with only 25 employees. None are specifically dedicated to working with patients or patient groups. When it comes time to interact with an advocacy group, there could be a variety of employees who show up, starting with the CEO.

“I will sometimes work directly with advocacy groups,” says Maslowski. “By engaging myself or other top personnel in our company, it gives us more visibility and the groups will realize we are dedicated to developing a relationship with them. If they have a need to reach us, they know they can get to me, our head of scientific affairs, our chief medical advisor, or our head of clinical operations.”

Maslowski also believes this approach demonstrates to these groups the focus Fibrocell places on patient advocacy. If someone at any of these groups knows they have direct access to the CEO of the company, it gives them confidence that the company is dedicated to support the group as well as the needs of the patients.       

5. Do Not Choose Favorites

If there are different advocacy groups to work with, Maslowski recommends doing your best to not show favoritism to any. He notes that can sometimes to be difficult to do, especially if you find yourself having better interactions with one over another. Even though both groups are in business to help the same patients, there can be some competition that exists between them.

“We do see divides,” says Maslowski. “If there are two groups working to help patients, we find some families will follow one or the other. When that happens, we need to show that we are supporting both groups equally. Not only is that fair and important to patients, but it keeps you from possibly distancing yourself from half of the patient population.”

You might wonder how one patient group would know how you’re interacting with another. But according to Maslowski, these groups are well aware of each other’s activities. He notes dealing with a rare disease is akin to attending a small college. If something happens, everyone seems to know about it the next day. Although patients align with different advocacy groups, they do talk and interact with each other. Directors and physicians meet at conferences and talk to each other. And, of course, members of each group will attend each other’s events and will be aware of what activities you have ongoing.

“If you give one group $10,000 to sponsor an event and don’t respond to the other one, it will not take long for that second group to find out,” he adds. “And their next question for you will be, ‘Why weren’t we sponsored?’ You want to have a good working relationship with both groups, and the best way to ensure that happens is to not favor one over another.”