It’s Rare Disease Day and we at Patient Advocacy Strategies were puzzled and disappointed by the ‘postponement’ of this year’s FDA/NIH Rare Disease Day amidst the current political turmoil.
The undisputed fact remains that there are over 7,000 rare diseases that impact over 30 million Americans, and the vast majority are considered life-threatening and absent any viable treatment options.
Remarkably, this devastating and last-minute setback didn’t compromise the passion and mobilization of this community, as evidenced by the 1000 rare disease advocates who assembled in Washington this week, undeterred and as vocal as ever.
For Us, It’s Personal
Our team at Patient Advocacy Strategies is comprised of several rare advocates, who, in addition to demonstrated expertise in their respective skill set domains, also bring personal, lived experiences caring for a loved one or living with a rare condition themselves.
Over the past several weeks, our team has reflected deeply on the current state of healthcare. Despite the frustration, we’ve tried our best to collectively focus on what we can control.
What Should We Do?
We firmly believe that patient communities and drug developers in the rare disease space can control two key areas, despite conflicting external forces.
- Intensify advocacy efforts with unrelenting action to amplify the impact and enduring unmet needs of those navigating a rare diagnosis.
- Leverage the best possible life science innovation through the execution of best-in-class clinical trials that demonstrate efficacy and safety rigorously and swiftly to deliver viable treatment options for patients.
Observing the rare disease community converge in DC to raise the flag this week ensures the persistence of the first point, and the resilience of these leaders is truly inspiring.
Regarding clinical research, we are acutely aware of the inherent challenges in study recruitment and the barriers that exist for rare conditions.
Are You Experiencing Enrollment Challenges?
If you are a clinical leader dedicated to rare disease R&D and are encountering enrollment obstacles, Patient Advocacy Strategies can help you overcome these barriers. Our extensive and trusted network within the rare community, coupled with our proven experience deploying tactics to drive study awareness, accelerate enrollment, and ironclad ensure ironclad retention, will set your clinical strategy up for success and ensure every effort you make is on target. We enable you to take your best shot on goal.
In a time of uncertainty and often wavering hope, we will work together with you to take control of what we can to deliver innovation for our patients.
