The single word that describes this week’s Rare Disease/Orphan Product meeting is “collaboration.” Speaker after speaker used this word to support the idea that the challenges ahead for the rare disease community can be addressed only through collaboration among patients, patient groups, government agencies, academia, industry and the medical community.

The challenges identified include:

• the need to fundamentally change the medical research system.
• the need to establish more certainty in the product development process to reassure the venture community that investments in orphan product development should be sustained.
• the need to assure that NIH and FDA both have adequate financial underpinning to support rare disease research and orphan product regulatory oversight.
• the need to assure access to products and services needed by patients, and to assure adequate reimbursement.

This was the second “summit” meeting sponsored by NORD, FDA, NIH, EURORDIS, Duke, and the Drug Information Association. The meeting is unique because it brings together all stakeholders concerned about rare diseases and orphan products.

I hope that subsequent meetings will continue to serve as a unique forum where stakeholders can come together and collaborate. The challenges are mighty but the opportunities to improve the lives of patients with rare diseases are vast.