It has been 40 years since Congress passed the Orphan Drug Act incentivizing drug manufacturers to pursue the development of drug treatments for rare diseases, 15 years since the first Rare Disease Day and over 10 years since the FDA implemented the use of patient- focused drug development (PFDD) meetings to help address the need for systematic collection of direct patient input.

Over time there have been undeniable advances: now there are more than 1,080 orphan therapies for over 500 rare diseases; drug development pipelines are shifting focus towards rare disease; there’s increased awareness around rare diseases; advocates are making concerted efforts to motivate drug developers to ensure clinical research is patient-centered.

However, a recent survey found that only half of companies conducting rare disease trials work with patient advocacy groups.

Patients are at the heart of healthcare. They are the experts who understand what it is like to live with illnesses and what is needed to improve their experience and care. We believe engaging with patients is not only the right thing to do, but also, it’s imperative to the success of our clients’ business.

Engaging with patients:

• Deepens understanding of their complex healthcare experiences, especially unmet needs and expectations
• Ensures that internal and external stakeholders consider the patient perspective as decisions are made throughout a medicine’s lifecycle—from clinical trial design to adherence programs
• Reveals opportunities to support and educate them and enables co-creation of meaningful solutions to their challenges
• Generates empathy for our most important customers and connects our work to a higher purpose

It starts with a conversation

With 142 pages in the first 3 of FDA’s 4 Patient-Focused Drug Development guidance documents, patient engagement could understandably seem overwhelming. Yet, engaging with caregivers and patients does not have to be complicated or arduous. It can start like most relationships do, with a genuine desire to get to know someone better.

Patient organizations play an important role in providing drug developers and marketers with relevant insights into the patient community. Patient organizations can also enable direct dialogue and information exchange between patients and biopharmaceutical companies.

Think about being RARE in your work to make a positive first impression

• Respectful Embrace the autonomy of patient organizations and their commitment to supporting their community. Shared goals focused on benefiting the patients are the foundation of the partnership. This may mean setting your own agenda aside.
• Authentic Clinical trials and new therapies provide a lifeline of hope for patients and families affected by rare disease. Yet for all the progress, successful drug development in rare disease is…rare. Delays or failures in clinical research are often devastating for patients and their families. Honest and transparent communication ensures the community has both hope and realistic expectations.
• Reciprocal Approach patient advocacy groups with a desire to learn and support, not with your hand out. Collaboration—without an agenda—fosters trust, ensures goal alignment, and demonstrates your commitment to the community. Eventually, when you need help recruiting for clinical trials, for example, advocacy groups will help you because you support them.
• Empathetic It’s only human to be emotionally affected by patient stories, and often these reactions to patient challenges compel teams to take action. However, it is essential to know that it is NOT about your feelings (sympathy), it is about patients’ feelings (empathy). Rather than be sympathetic, the goal is to enable patients to share their fears, sorrows, hopes, and optimism.

A challenge to drug developers and marketers

My challenge to our industry this World Rare Disease Day—and beyond—is to embrace patients as the experts they are. Partnering with patient advocacy organizations and engaging with patients benefits those affected by rare diseases and companies alike. Maintaining the highest level of ethics and transparency is vital to successful patient engagement initiatives and guidelines are available to guide interactions.

The Patient Advocacy and Engagement team at Spectrum Science is also here to support your efforts. Reach out if you’d like to learn more.

This article was orginally published on LinkedIn by Kristin LaBounty Phillips, SVP, Patient Engagement & Advocacy. Kristin has a passion for rare diseases and working with patient groups to gather insights, share their authentic stories and cocreate solutions that ultimately lead to a better patient experience.