Raise Your Hand to Support People with Rare Diseases

Raise Your Hand to Support People with Rare Diseases

03.07.12 | By Preet Bilinski

We continue the conversation of rare diseases as a global health challenge with Lundbeck's Dr. Christopher Silber. Offering effective therapies is a fundamental component of the commitment to help improve the lives of people with challenging, unmet medical needs. At Lundbeck the focus is on making a difference for patients, no matter how complex the condition.

What compelled you to join the fight against rare diseases?

Lundbeck's mission is to bring forward therapies that improve the lives of those suffering from psychiatric and neurological conditions. Some of these conditions are quite common, while other disorders only affect a very small number of people. The common thread for our efforts is our commitment to addressing unmet medical needs.

Lundbeck in the US has a deep commitment to supporting people with rare diseases. Our active engagement in these patient communities is an ongoing reminder of the importance of our work to support those with rare diseases. Over the years, we have come to appreciate that each rare disease might affect a small population but in the aggregate, about one in every 10 Americans is living from one of these uncommon conditions. We believe it is vitally important that we not only improve coverage and access, but also create an innovation-rich environment that pulls through important new medicines where the need is great.

In your mind, what are the greatest challenges to continued progress?

Through our extensive work in this area, we have come to understand the challenges faced by the rare disease community, where a simple diagnosis can take years and treatment options are often limited. One of the greatest challenges is attracting more companies to orphan drug development so that more therapeutic solutions are available to those in need. While great strides have been made in the number of drug approvals in recent years, significant challenges remain in bringing new therapies to the millions of Americans that need them.

Among these challenges are the logistics of the clinical trial process because rare disease patient populations are small and difficult to identify - like finding a needle in a haystack. Patients are often spread out, making it difficult to bring them to a central location to participate in the studies, all of which can delay enrollment in efficacy and safety trials. And there may be insufficient numbers of patients in this country to reach the necessary enrollment numbers for studies, further complicating the clinical trials.

Additionally, the approximately 7,000 rare diseases represent an exceptionally diverse collection of disorders, requiring biomarker development and reliable outcome measures to facilitate clinical research and evaluation of potential therapies. These are just some of the practical issues facing companies dedicated to treating orphan diseases, but we remain committed to working with stakeholders in the rare disease community to advance research and innovation and ensure patient access to newly approved medicines.

What is your hope for the future?

Lundbeck recognizes the tremendous need for scientific research to address many more of the rare conditions for which there are not yet approved therapies. We are continuing our efforts to collaborate with academic institutions and companies with promising compounds in development with the goal of having an even greater impact on the lives of patients, families and caregivers. Moving forward, we hope to see the medical community continue to focus resources on addressing the needs of patients with challenging diseases for which there is significant unmet need - regardless of whether they affect millions or far fewer numbers of people.

Follow Preet on Twitter @PreetAtPhRMA.

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