The drug development process is at the epicenter of a seismic shift. Gone are the days when pharmaceutical companies would partner solely with scientists and physicians to develop new medicines, with patients only becoming part of the process upon enrolling in a clinical study. Today, the path from development to FDA approval looks vastly different – with the patient’s perspective and voice playing a critical role in regulatory decision making. And while one voice can add significant value, it’s the collective amplification of many voices that has proven to be most effective in accelerating research.

Patient groups are now recognized as an integral part of the drug development roadmap. However, that wasn’t always the case. When advocacy groups were originally founded, many focused solely on patient education and support. Today, these communities have evolved from that initial mission and are now highly engaged at all points along the drug development process from discovery through commercialization. Patient groups add tremendous value and influence throughout the process, from designing studies, to identifying study participants, and even providing input to regulatory agencies on which outcome measures mean the most to patients. They fund research, lobby legislative bodies and serve on government advisory boards.

Engaging with patient groups to understand the “patient voice” from the beginning of research can greatly benefit overall drug development. Patient input helps study sponsors understand the full scope of the unmet need. Currently, Horizon Pharma is working on a clinical trial which involves evaluating a medicine for the treatment of Friedreich’s Ataxia (FA), a rare, debilitating, life-shortening neuromuscular disorder. As part of the study, Horizon collaborated from the onset with the Friedreich’s Ataxia Research Alliance (FARA), a patient organization with deep ties to the patient population it serves. For clinical studies in the rare disease space, recruiting and retaining patients has historically been challenging. Unlike more prevalent conditions, like arthritis or heart disease where the disease’s natural history is well-defined and researched, rare disease studies must rely greatly on input from those directly affected, as well as the health care professionals who treat them. Because a rare disorder by definition affects fewer than 200,000 people, patient groups prove to be the key connector.

For the FA study, FARA offered crucial input on trial design to ensure that travel requirements, medicine administration and study eligibility criteria were realistic within the FA community. Moreover, FARA provided full access to its comprehensive patient registry, the only collection of demographic and clinical information for 2,000-plus FA patients around the world. The study for FA began recruitment in June of 2015 and completed enrollment in less than one year, an unusually fast pace for a rare disease trial. Partnering with FARA and its passionate network of patients, families and professionals greatly accelerated the research process, which may potentially bring a meaningful treatment to patients more quickly – and efficiently.

The FDA is also committed to patient-focused drug development (PFDD). According to the FDA-supported Clinical Trials Transformation Initiative (CTTI), a public-private partnership working to develop and drive adoption of practices that will increase the quality and efficiency of clinical trials, PFDD refers to the meaningful engagement of patients in the development of therapeutic products and the important roles patients play in improving the entire process, from study endpoint selection that reflects meaningful patient outcomes to recruitment and retention in clinical trials. Since the Prescription Drug User Fee Act (PDUFA) was reauthorized in 2012, the FDA has been working to incorporate the patient voice into its regulatory review process which in turn helps inform decision making. In 2015, cancer patients reaped the benefits of this policy change when the FDA approved a new melanoma therapy. Patients provided feedback on what it felt like to see their melanoma lesions shrink. Their input on the cosmetic and psychological advantages to the therapy proved vital in the FDA advisory committee’s decision on an “overall favorable benefit-risk profile of therapy for patients.”

Engaging with patient communities outside of the clinical trial arena is also key to creating a strong patient-centered approach to the drug development process. Participating in disease awareness events like walks and bike rides, attending educational forums or interacting during patient get-togethers helps pharmaceutical companies grasp, on a more intimate and personal level, the challenges patients and their families face on a day-to-day basis. Truly understanding the patient journey helps inform every step of the process – from designing a clinical trial to ultimately making a medicine available to those who need it most.

Focusing research and studies on the patient perspective, voice and experience is now at the forefront of bringing new, innovative therapies to market. At the end of the day, we’re all patients whose collective voices deserve to be heard.


*Dr. Jeffrey W. Sherman, MD, FACP, is the Chief Medical Officer at Horizon Pharma




PhRMA Patient-Focused Drug Development fact sheet –

Xtalks webinar: The Powerful and Evolving Role of Patient Advocacy Groups in Orphan Drug Development –

The Scientist: The Challenges of Rare-Disease Research –

Clinical Trials Transformation Initiative (CTTI) / Project: Patient Groups & Clinical Trials –