By Chris Peetz
Patients, caregivers, and their voices are the foundation of our industry. They are the driving forces behind every life sciences organization, whether that means discovering new scientific mechanisms at the lab bench, making clinical study participation less onerous, developing oncology therapies that will make cancer a manageable illness, or engineering technologies capable of curing a child’s life-threatening rare disease. Patients are what our industry was founded on, and patients will always be at the heart of why we do what we do.
Then why do patients feel that pharmaceutical companies don’t truly understand their needs?
In a recent survey by consulting organization Accenture, the majority of patients feel that pharma companies don’t understand their emotional, financial, and other needs related to their condition.
So, where do patients go to receive the help and support they need in navigating their conditions?
According to the same survey, most patients have greater trust in and better experiences with patient organizations, such as patient advocacy groups (PAGs). Whether by phone, social media, or attending in-person events, patients utilize PAGs to seek everything from therapy information to disease education. Through earlier, more personalized, and more frequent interactions when compared to their pharma counterparts, PAGs help patients feel more educationally and emotionally supported.
We are an industry that claims it is “patient-centric.” We often use “mission-driven” as a phrase to describe ourselves and our passion. We embrace and share patient stories to help demonstrate the unmet need that exists. And yet we must strive to improve how we serve the very patients who are the heart of our industry.
As an industry that prides itself on connection, we can’t exist in a vacuum. Effective patient engagement starts with forging strong relationships with the leaders of PAGs, bridging our strengths to give patients the full appropriate support that they need — and deserve.
PATIENT ADVOCACY ENGAGEMENT IS OFTEN RARE
As the CEO of a company developing treatments for rare diseases, I have worked with different PAGs, learning about patient needs and incorporating them into research and development programs.
For rare disease organizations, there isn’t much of a choice. Because there are so few impacted by these devastating and life-threatening diseases, each voice is that much more important, and each person involved is that much more vital in raising awareness. Without large averages or population-based metrics to observe, we must understand what the disease means on an individual level. When developing something that can be so impactful for a small number of people, it becomes necessary to engage with patients personally to more deeply understand their stories and what they go through on a daily basis.
But PAG engagement doesn’t — and shouldn’t — apply to the rare disease space exclusively. Nearly every life sciences organization was built on a patient’s unmet need. And nearly every life sciences organization would benefit from more direct patient involvement. PAGs have created a hub between industry, patients, regulators, and other stakeholders, helping to facilitate and manage these direct connections.
A PARTNER IN ENROLLMENT AND BEYOND
For one, PAGs help to drive patient interest in clinical research and studies — ultimately enabling faster trial enrollment, which translates into faster study completion. While this is especially crucial for the rare disease communities, clinical trial enrollment is becoming increasingly more difficult across all sectors and disease areas. One-size-fits-all approaches are becoming outdated, and the advent of precision medicine and targeted biomarkers is creating patient population subsets at an exponential rate. PAGs help raise awareness of clinical trials to the patient community, expediting the enrollment process in these smaller communities.
"We are an industry that claims it is ‘patient-centric.’ … And yet we must strive to improve how we serve the very patients who are the heart of our industry."
But PAGs are truly a partner, not just a mechanism for expediting clinical trial enrollment. I’ve worked very closely with the Alagille Syndrome Alliance, a PAG created by parents to raise awareness of this deadly disease to the research community. While initially only involving academics and scientists, their work eventually snowballed and extended to industry. Twenty-five years later, the Alagille Syndrome Alliance now organizes a biannual scientific conference that unites all key stakeholders in a single room: Academics speak toward new discoveries, industry provides drug development updates, and families share their personal stories to draw attention to what’s needed.
This open, united forum of communication is the most effective way to move medicines forward, regardless of who your patient population is. It makes our work that much stronger, because it helps ensure a patient’s own experiences and needs are more directly translated to the therapies we’re developing.
PROVIDING PATIENTS WITH A VOICE
I think most would agree that every life sciences industry company would benefit from having more direct patient connection. Yet, due to privacy and data protection laws, including those governing the confidentiality of patient information in clinical trials, there are defined boundaries and consent requirements that must be observed between patients and the organizations developing treatments. The PAG hub, under certain conditions, can provide a conduit to communicate patients’ needs and share their experiences while remaining compliant with these laws and applicable regulations.
This interface for the patient community is immensely helpful when designing clinical trials and determining a study’s endpoints. In certain situations, we’ve requested PAG feedback on draft study protocols such as, “What does this look like from a patient perspective? Are there too many visits? Are the tests run during these visits lengthy and time-consuming? Is it realistic to have patients fill out this many questionnaires?” Answers to these questions help inform a study’s design before it even begins — taking the patient’s needs into account early on in the drug development process.
Advocacy groups serve as an interface on the regulatory side as well. Whether it’s participating in an FDA meeting or providing a strong, passionate voice in Washington, PAGs help raise awareness of the unmet need that exists on a direct, personal level. When someone is impacted by a disease — a patient themselves or even a family member — and they share their story on Capitol Hill and say, “You have to pay attention and learn about this disease,” people respond. And that’s when patients feel properly supported.
UPHOLDING OUR PROMISE TO PATIENTS
When I read the data that came out of the Accenture study, I was disappointed. But I also was not surprised. Having worked with advocacy groups so closely, I’ve witnessed the support they have provided for their respective patient communities. And I’ve also witnessed the impacts of partnering with these same PAGs, ensuring that the patient voice is communicated to its industry counterparts.
If we are who we say we are — an industry driven to improve human health and to impact and save lives — then we must do everything we can to uphold this commitment. Our industry must do better in serving and addressing the patient need, because, frankly, the metrics show that we’re not good enough. And becoming more involved with and appropriately supporting PAGs is the first step in this process.
CHRIS PEETZ is president and CEO of Mirum Pharmaceuticals, a clinical-stage pharma developing therapies for rare liver diseases.