By Rebecca Aune, NORD Director of Education Programs
The rare disease community knows hard truths all too well. Effective treatments are scarce across the thousands of known rare diseases. With small and dispersed patient populations, it can be difficult to even find others with the same condition, let alone advance the research. It can take years for scientists to fully understand the complexities of how a rare disease manifests and progresses. Rare diseases don’t discriminate – they affect people from all walks of life. Yet many of those affected are not researchers or clinicians. They may not be familiar with the research process, even though they are true experts when it comes to their own condition.
For those of us in rare disease patient advocacy, the statistics are sobering. Drug development is slow, complicated and incredibly costly. It takes an average of 12 years for an experimental drug to move from pre-clinical research through review by the U.S. Food and Drug Administration, and only about 14% of drugs that enter clinical trials make it across the finish line. The cost of developing a single drug ranges from $314 million to an astounding $2.8 billion. Currently, only 5% of rare diseases have approved therapies.
But what if this reality could change? What if the knowledge and lived experiences of patients and families could be used to accelerate and enhance the drug development process? What if their voices could lead to more treatments, faster?
A Collaborative Approach to Change for the Rare Disease Community
At the National Organization for Rare Disorders (NORD®), alongside our member organizations, our partners at the FDA and Critical Path Institute, and pharmaceutical companies working to develop new drugs, we wrestle with these questions every day. The need for rare disease treatments is urgent, and no one understands these conditions better than the patients themselves. They can and should always be partners throughout this process. For that to happen, however, patients and families need tools and knowledge to actively take part in drug development.
That’s why we’ve been working for two years to create a suite of free, accessible resources for the rare disease community. We believe these tools can empower patients and caregivers to contribute time, data, knowledge and lived experiences to help shape the most effective drug development programs for their specific conditions. As NORD’s Director of Education Programs, I knew these resources needed to be easy to understand, engaging, and user-friendly to equip our community to step into the crucial role for which only they have the expertise.
Empowering Patient Organizations to Drive Research
Patients, caregivers, and patient organizations play a vital and growing role in drug development, especially in rare diseases. Many patient organizations, especially those new to research, are eager to learn how they can be effective partners in advancing treatments. NORD is here to support them in this critically important work. In fact, several recent breakthroughs in rare disease drug development have been driven by patient organizations, many of which are NORD members.
To create a valuable learning experience for the rare disease patient community, we partnered with the FDA, Critical Path Institute, and nine NORD member organizations. The voices and stories of patients and advocates guided every step of our curriculum development.
Introducing the Rare Disease Drug Development Series
The result of this collaboration is the Rare Disease Drug Development Series: What Patients and Advocates Need to Know. This comprehensive, self-paced online course is available for free in both English and Spanish. It’s designed specifically for rare disease patient organization leaders and advocates and covers the research process in detail, from pre-clinical research to navigating FDA review and clinical trials.
Participants can work through the material at their own pace and earn up to three certificates of completion, as the course is divided into three manageable parts:
- Part 1: Foundations of Drug Development and Pre-Clinical Research
- Part 2: Navigating Drug Review and Access
- Part 3: The Basics of Clinical Trials
The course features:
- Short, animated explanatory videos
- Printable one-pagers and infographics
- Glossaries to help with new terminology
- Quizzes to track progress in learning
- Video interviews with patient organization leaders, representatives from pharmaceutical companies and health policy experts
Your Role in Advancing Drug Development
NORD is committed to patient-focused drug development (PFDD), ensuring that patients’ needs, perspectives, and experiences are part of every step in the drug development process. We also know how busy the rare disease community is, so we made sure the course is mobile-friendly. Whether you’re doing dishes, waiting in a doctor’s office, or in the school pickup line, you can easily engage with the course on the go.
We invite you to join us in this important effort! Register for the Rare Disease Drug Development Series today at learn.rarediseases.org and empower yourself with the knowledge needed to advance the development of new therapies for rare diseases. The course is free, self-paced, and designed to fit into your busy life.
Help us spread the word! Share the course with your networks: colleagues, fellow patients, and anyone across the rare disease community who could benefit. Use the Rare Disease Drug Development Series Toolkit to share the course on social media, via email, or through a newsletter.
Together, we can transform the future of rare disease treatments.
Alone we are rare. Together we are strong.
