GOALS

• Ensure that new drug applications for rare disease therapies are supported by robust evidence of safety and efficacy derived from real-world patient data
• Navigate the complex regulatory landscape for rare disease drug development and approval by staying informed about evolving guidelines and leveraging patient insights to support submissions
• Advocate for patient-centric drug development and regulatory policies that accelerate access to innovative treatments for rare diseases

Frustrations

• Facing difficulties in accessing and interpreting real-world data from patient communities in a way that meets stringent regulatory requirements for evidence generation
• Dealing with lengthy and often opaque regulatory review processes for rare disease therapies, which can delay patient access to potentially life-saving treatments
• Struggling to bridge the communication gap between patients, regulators, and pharmaceutical companies to ensure that patient perspectives are adequately considered in the drug development and approval process

GOALS

• Empower patients with rare diseases and their families by providing them with access to reliable information, resources, and support networks through online platforms
• Raise awareness about the challenges faced by the rare disease community and advocate for increased funding, research, and policy changes that improve their lives
• Facilitate meaningful connections and collaborations between patients, researchers, and pharmaceutical companies to accelerate the development of effective treatments and improve patient care

Frustrations

• Encountering difficulties in navigating the overwhelming amount of information available online and identifying credible sources of support and resources for specific rare diseases
• Feeling frustrated by the lack of awareness and understanding surrounding rare diseases among the general public, which can lead to stigma, isolation, and inadequate support
• Struggling to have their voices heard and their needs met by healthcare providers, researchers, and policymakers who may not fully grasp the unique challenges of living with a rare disease