Advocacy Groups in Patient-Focused Drug Development
Juliane K. Mills
Juliane K. Mills
Director Therapeutic Expertise, Center for Rare Diseases

For years, clinical research was missing a key piece – the patient voice. Fortunately, today’s drug developers realize that patients are experts in their disease and have turned their attention to patient-focused drug development (PFDD). It’s important that patients’ unique experiences, perspectives, needs, and priorities are not only understood, but incorporated into the drug development and evaluation process. When drug developers put patients first, they can ultimately run more cost-effective, efficient, and successful studies. Our Juliane Mills and Kendall Davis discuss how advocacy groups can help:

Patient advocacy groups are organizations formed to provide education, advocacy, and other support services to the millions of people around the world that suffer from a physical or medical condition. In the rare disease space, they’re most often created by the parents of children with a rare disease, and there’s typically only one group in existence.

The overarching goal of each group is the same: help the people and families who are directly affected by medical conditions. There are several different ways advocacy organizations can do that.

  • Some advocacy groups are considered support organizations. These groups help build support networks of families with similar diagnoses, as well as act as bridges between care provider networks with experience in diagnosing and treating a condition or disease. Support organizations also provide the disease community access to the latest research and scientists.
  • Advocacy groups also provide support through their focus on funding for research and communication with lawmakers. These groups will advocate to government officials, drug developers, and the general public to raise awareness of a disease and try to increase the funding available for research.
  • Of course, a disease does not just affect the patient, but also the people closest to that patient. Some advocacy groups are established for the caregivers of patients and they often operate as a support network for family members with loved ones battling a disease. These groups may provide educational materials, meetings, counseling, sibling support, and other family services. Many patient advocacy groups provide a combination of these core functions.

Advocacy groups exist for both common and rare diseases, but for the rare disease community, these organizations often are the only source of support and information. Rare disease advocacy groups help connect individuals around the world and build a support network, create patient databases/registries, and begin collecting vital information on their diseases. These groups not only help rare disease patients navigate the complex landscape of their disease, but they also focus on educating treating clinicians and can connect patients to doctors that may be more familiar with their rare condition.

Advocacy groups play a key role in developing patient-focused drug development. By forming strategic, collaborative partnerships with advocacy groups, drug developers can gain access to qualitative data that can help them better understand a specific disease and its patient population. These data include the language used to talk about a disease, symptoms that are most important or bothersome, resources for treatment and coping, as well as emotional context for each step along the patient journey. With this information and the in-depth knowledge advocacy groups provide, sponsors can better design clinical trials that work for patients in their everyday lives and focus on developing new treatments that will have the greatest impact on patients’ quality of life.

In addition to deep data and trial design, these groups can also help sponsors connect with patients and healthcare providers for participation in research trials. Advocates are key opinion leaders (KOL) who speak the language of the patient, and often have already developed relationships with patients and their caregivers. They are seen as a trusted source of information for patients, and they can help sponsors spread the word about trials that truly meet patient needs. Many advocacy groups are also well-connected with healthcare professionals that work specifically with the condition they advocate for, and they can help connect sponsors with the right doctors for a trial.

It’s also not uncommon for advocacy groups to collaborate with sponsors on identifying clinical endpoints that are patient-centered. For many patients, measurements like blood pressure aren’t their main focus. They’re more concerned with how a treatment affects their day-to-day. Can they still take a walk? Spend time with family? Go to school? By measuring how treatment options work for patients in their daily lives, we can better understand the benefit and value these options provide.

When done right, these partnerships can be mutually beneficial at every stage of clinical research. During the pre-clinical phase, advocacy groups can fund and support research, provide registry and natural history data, assist in defining what is most important to the community, as well as assist in eligibility and site selection. Throughout the clinical process, these groups can assist in protocol and feasibility, study awareness, recruitment, retention, and education. Advocacy groups can also offer immense support to regulators throughout the regulatory review process by providing the day-to-day context of life with a disease and the need for treatments.

It’s clearer than ever that patient-focused drug development is the future of healthcare. By working with patient advocacy groups, we can connect with patients early in the drug development process and make them active partners throughout the entire clinical research life cycle.

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