Research Bonds Strengthen

Efforts to collaborate around patient engagement in every phase of product development are illustrating the growing relationship between disease advocacy groups and industry

In the past, the term “patient centricity” was sometimes brushed off as something pharmaceutical companies would say to sound good. 

Not these days. 

“There has been much talk in the industry about putting the patient in the center, but for the first time this is really becoming a centerpiece for most pharmaceutical companies-the patient really comes first,” says Lynn O’Connor Vos, president and CEO of the Muscular Dystrophy Association (MDA). “It comes in as early as designing clinical trials. Many advocacy groups now come together with industry to say, ‘How early should we engage a patient in the discussion of a clinical trial?’ and ‘What kind of outcomes would be meaningful for you?’ We’re seeing in every phase of product development that there’s a role to engage patients, so I would say the role of patient advocacy is getting much stronger.”

Someone who has had a front row seat to this change is Allison Moore, founder and CEO of the Hereditary Neuropathy Foundation. She founded the group after learning of her sudden onset of Charcot-Marie-Tooth (CMT) disease, an inherited neurological disorder characterized by progressive loss of muscle tissue. “It’s very positive,” says Moore of the climate between drug manufacturers and patient advocacy groups. “More pharma companies are hiring advocacy directors to work with people like me. That’s a good sign.” 

For Moore, the dynamic is quite different then when she first started reaching out to pharma companies years ago. All but one were not very interested in working with her. That one was the French biopharma Pharnext. On a whim, Moore emailed the company’s then-CEO in 2013, before leaving for a family vacation to Europe. To her surprise, he got right back to her and after some itinerary changes, Moore ended her vacation in France, talking with the Pharnext about her disease. 

“I spent two days with these guys,” she recalls. “I knew a good amount already, but I learned so much … and they were excited to meet someone with CMT.” 

That meeting began a long-standing, mutually beneficial professional relationship.

Two-way street 

Patient advocates say the positive relationship that has been developing over the past few years between industry and advocacy groups is helpful to both parties, and, in turn, patients. In fact, this opportunity was a key reason Vos took the position with MDA. 

“What’s remarkable about the Muscular Dystrophy Association and the neuromuscular medicine category is there have been so few therapies available for patients,” she says. “That is what attracted me to the MDA-the opportunity to build a greater partnership with pharma and biotech to help make sure diseases are better understood and characterized so that we can bring better therapies to market for our patients. We are helping pharmaceutical companies facilitate clinical research studies and recruit patients by gathering clinical and other key data sets.”

MDA is able to do this through its MOVR (NeuroMuscular ObserVational Research) initiative, a data platform that will combine clinical, patient, and genetic data all in one location for better collaboration among care centers and experts. The goal of MOVR is to help pharma design better trials, to ultimately create more effective products for MDA’s patient community, Vos says. 

Making the connection  

Vos and Moore point out that, in many cases, their groups serve as a middleman between the patients and the pharma company. 

“Patient advocate organizations are the ones that can be the convener and connector to big pharma and patients to make sure the patients’ perspective is always being represented,” says Vos. 

Both MDA and HNF have held events and programs that bring patients to the forefront. The 2019 MDA Clinical and Scientific Conference is set for April 13. 

“This is the largest neuromuscular medical event that serves as the intersection of pharma, our care centers, our doctors, and our medical and scientific community,” says Vos. “We will be featuring technology companies, as well as pharma/biotech and other patient advocacy organizations. Our role is to be the convener and set the stage for a great dialogue, conversation, and sharing.”