How Patient Centricity Can Lead to Better Outcomes

Over 25M Americans are living with a rare disease, yet only ~5% of them have an FDA approved therapy to help improve their life. We can and should do better.

As defined in the U.S., a rare disease is a condition that affects fewer than 200,000 people. Although coined as “rare,” when looked at collectively, rare diseases affect between 25 and 30 million Americans. Yet, people living with rare diseases still struggle to receive the treatment options they deserve; more than 95% of people living with rare diseases lack an FDA-approved treatment for their condition.

Patient centricity is certainly not a new concept in the pharmaceutical industry. And while it has always been integral when focusing on rare diseases, the past year has given us a much deeper understanding of why meaningful connections with the patient community are so important.

At Harmony, we understand that every patient experience is unique, and that’s why keeping patients at the heart of all we do is the mission that grounds our overall perspective. As a rare disease company focused on rare central nervous system and neurological disorders, patient centricity has always been at our core, but as the world continues to adjust to the dynamic challenges of this pandemic, it is a mission-critical time for us, and the pharmaceutical industry at large, to take heed, listen more closely to patients, and ensure their voices are the focal point in every stage of our work.

Listening to people living with rare diseases is a critical piece of expanding rare disease treatment options. Research and development (R&D) must be informed by what we are hearing from patients to ensure we deliver based on their needs and are keeping their interests at the forefront. We always ask: What do our patients need? What are they saying, and how can we solve it? Engaging directly with people living with rare diseases is one of the best ways to get these answers and can help us build a clinical development program that will have meaningful differentiation.

Because of rare disease R&D challenges – such as smaller patient population sizes and a lack of existing knowledge or understanding of disease states – many companies are daunted by the prospect of addressing patient needs in a nuanced space. However, we know that the pharmaceutical industry is stronger together. We recently joined The Rare Disease Company Coalition (RDCC) as a founding member where we have come together with other like-minded companies to educate key policy stakeholders about the distinct considerations life science companies must navigate when developing and delivering rare disease treatments to people in need. Our goal is to help inform policies that allow for cost-effective, timely development and commercialization of treatments for rare disease patients. We do this by working together, and by listening to the voices of people living with rare diseases to inform every stage of effective drug development – and luckily, connecting with these individuals is becoming easier than ever before.

If there is any silver lining to the past year, it is that COVID-19 accelerated some already-growing trends that make connecting with patients more streamlined and effective. For example, Harmony furthered our support with Patient Advocacy Groups (PAGs) by digitizing programming and using technology to connect with communities in new ways – and that’s just the beginning. The pharmaceutical industry must continue to seek out new methods to connect with people living with rare diseases, whether through policymakers, PAGs, or even grant programs to support patient-focused organizations.

The voices of people living with, or affected by, rare diseases should guide the actions that we, as pharmaceutical companies, take on the road to advancing treatment options. With a patient-centric approach, the pharmaceutical industry can be an agent of change for the millions of people living with rare diseases every day, and that change can start now.