Strategic Access: Bridging the Gap for Rare Disease Therapies

Today, on Rare Disease Day, we are reminded that for millions with a rare disease, diagnosis is just the beginning, a beginning that can take an average of 5 years to achieve. Getting diagnosed is not just a medical classification; it is a profound step in the journey towards access to a treatment which might be as difficult to receive as the diagnosis itself.

What if the treatment existed, but geography, economics, or bureaucracy stood in your way? This is the stark reality faced by millions, a reality that we must collectively address with urgency and determination.

Over 7,000 rare diseases affect more than 300 million people, yet less than 5% of rare diseases have approved FDA treatments. The journey from investigation to broader patient availability can span over a decade, and the access gap widens further depending on where you live, with only 10-15% of these approved treatments available to patients in Low or Middle-Income Countries (LMICs). This profound inequity fuels my passion to accelerate innovative access solutions – where you live should not limit your access to essential medicines.

At our 4^th Clinigen Insights Conference this week in Windsor, we convened leading pharma and biotech experts to tackle the critical issues shaping global patient access. We polled them on the biggest barriers to rare disease treatment distribution and patient access and 74% of respondents identified Managed Access programs as “the greatest opportunity to improve patient access to treatment, particularly for rare diseases”, whereas 48% thought “geographic accessibility” was the biggest barrier. It is clear, strategic planning and tangible solutions are crucial for the healthcare sector to effectively tackle access disparity.

The convergence of early access programs and successful market entry is now a strategic necessity, especially considering how managed access has evolved beyond logistics. This shift requires a more nuanced approach, using Managed Access programs to drive market success. Traditional strategies often fall short due to small patient populations, complex regulations, and high investment needs. Navigating this evolution, with the help of actionable data, strategic partnerships, and a global perspective, is essential for ensuring patients gain timely access.

Managed Access programs, including pre-approval access, named patient programs, and post-trial access, play a vital role in bridging the gap between clinical development and commercial availability. These programs not only provide patients with early access to potential treatments, but also generate valuable real-world data that can inform market access strategies and regulatory submissions. Crucially, patient engagement is vital to the success of these programs, and by actively including patient considerations in the ethical design and implementation of Managed Access programs, they can be tailored to meet the specific needs of the rare disease community.

Clinigen Insights proved to be an invaluable platform for exploring the operational challenges and future potential of access programs, emphasising the importance of real-world evidence for access policies and Health Technology Assessment (HTA) evaluations, and practically addressing the complexities of ethical pricing and access in global regulatory environments.

Looking ahead, I see managed access evolving into a globally interconnected system, using digital platforms to streamline access and collect real-world data, while fostering global collaborations to ensure improved access, especially in LMICs. We are moving towards an era of personalised access programs, where predictive analytics and digital platforms will revolutionise patient diagnosis and care because of their hyper targeting abilities.

At Clinigen, we believe that access to medicines should be global by design. That is why we partner to explore ethical access through every possible legal pathway – whether through clinical trials, standard commercial channels, or Managed Access programs for unlicensed treatments. With more than 35 years’ experience, our global Managed Access team accelerates access to innovative medicines for thousands of patients, supporting hundreds of active Managed Access programs around the world every year. I am deeply appreciative of the contribution of every Clinigen colleague globally, who work tirelessly to enhance patient access to essential medicines.

On Rare Disease Day, and every day, let us work towards a future where rare disease treatments are accessible to all who need them. In the end, access to treatments should not be rare.

Best,

Jerome