Three Ways Patients Fuel Horizon’s Passion for Discovery

Patients are critical to clinical research – not just as clinical trial participants but as active contributors in the decision-making processes across the research continuum. Horizon has long put its patient-centric philosophy into effect, inviting patients to be involved in everything from trial design to providing ongoing feedback on therapies after approval.

But what does a people-first research and development (R&D) strategy look like in action?

Read how four Horizon leaders and their teams incorporate patient perspectives into their work – and how doing so helps move R&D forward in meaningful ways.

Partnering with advocacy groups

One of the most fundamental ways in which patients help inform research is by sharing their experiences living with a disease. Horizon seeks out opportunities to hear these experiences directly by partnering with patient advocacy groups, from large global associations to smaller organizations. Many of the groups Horizon works with advocate for people living with the rare, autoimmune and severe inflammatory diseases that Horizon is developing medicines for, including lupus, scleroderma, pulmonary fibrosis, myasthenia gravis and more.

Veronica Moore, associate director, patient advocacy, says working with patient groups can give Horizon additional insights that can help shape multiple stages of R&D.

“The information we gain is so central to our work, from clinical trial design to patient recruitment to clinical trial operations,” Moore says. “For instance, feedback from our advocacy partners led us to redesign materials for one of our trials in lupus, which helped us create documents that really resonated with patients and supported our recruitment efforts.”

These insights are anonymized, gathered, stored and analyzed, creating a dynamic library of first-person accounts. People with the rarest diseases often provide the most illuminating insights, since less may be known about their conditions.

“Because some of the conditions we’re developing therapies for are ‘silent’ diseases with long roads to diagnosis, patients often feel invisible,” Moore says. “It’s our duty to hear them, and to use that information to advance new treatments that might help them.”

Understanding patient needs

A main pillar of Horizon’s R&D approach is to weave the patient experience and needs into the design of clinical trials. Theresa Podrebarac, M.D., M.Sc., senior vice president, clinical development, says hearing how the symptoms of a disease affect a person’s day-to-day experience can key researchers into the right set of endpoints or outcomes to include in a trial to measure clinical benefit. One way Horizon gathers these insights is by attending seminars and focus groups hosted by the U.S. FDA and other patient-focused organizations.?

“A few years ago, the clinical development team attended a seminar on diffuse cutaneous systemic sclerosis [dcSSc] hosted by the FDA,” Podrebarac says. “We heard firsthand how debilitating pain can be for these patients and how it impacts their lives. With this important insight, our team updated our protocol to include pain in the list of endpoints in our clinical trial on a medicine for dcSSc.” ?

Sometimes the patient perspective even comes from within the company. Julia Taylor, a Horizon employee who lives with Sj?gren’s, provided details of her experience in order to help with the trial design for Horizon’s investigational treatment for Sj?gren’s.

“Some researchers may never meet a patient with the disease they’re working on a treatment for,” Taylor says. “I’m grateful to be able to share thoughts to support our scientists as they develop a potential therapy.?Our company is helping to change patients’ and caregivers’ lives for the better, and it’s been wonderful to be a part of that.”

Infusing the patient voice

Patient input is key across all stages of R&D, from preclinical to post-approval. Horizon’s health economics and outcomes research function (HEOR) generates evidence and measures the efficacy, effectiveness, value and other treatment outcomes (including patients’ quality of life) of investigational and on-market medicines to inform the R&D process. The team works to capture this through in-depth surveys and other metrics at multiple steps in the clinical development process.

“Our studies incorporate patient perspectives, preferences and priorities across the product lifecycle – or sometimes even before a product exists,” says Hari Patel, senior director, medical affairs and outcomes research. “The work we do helps us understand disease unmet needs, diagnosis and treatment journeys, quality of life and how existing therapies are working or not working for patients. These are topics that aren’t typically captured by clinical trial outcomes or physicians’ reports. Sometimes our research captures areas of disconnect between patients and physicians that are important for our researchers to understand as we develop new medicines.”?

Horizon also uses HEOR to monitor aspects of how its medicines work once on the market. For instance, the HEOR team captured data on the effects of Horizon's on-market medicine for Thyroid Eye Disease (TED) over several months and across numerous care sites to understand its effects on patient health, quality of life, treatment delivery and other aspects of care around the disease. These data give the R&D team valuable insights into how the current therapy works in real life – and how the next iterations could be made even better.??

“Publishing HEOR findings is always the goal,” Patel says. “Learnings from HEOR need to be shared in the public domain, to help the industry at large do even better work for patients.”

Horizon is constantly on the lookout for new ways to infuse patient voices into its R&D process. Listening and learning from patient experiences first-hand is an important way to make science stronger and potentially bring clinically meaningful therapies to people living with rare, autoimmune and severe inflammatory diseases. Learn more about our R&D work at Science Forward.