The Fierce Urgency of Now — The 2023 NEXT Report

July 13, 2023

While fundraising is a key part of patient advocacy and drug development, time may be more important than money for those diagnosed with a rare disease. Waiting for a cure, treatment, or therapy can seem insurmountable when comparing the symptoms and life expectancy of some conditions to the timeline of drug development.

Read an excerpt from Endword of the 2023 NEXT Report to learn how the intersection of technology and collaboration are playing a part in the future of drug development:

“Technology continues to transform rare disease patient advocacy and give rise to next-generation patient advocates.
In grammatical terms, these advocates have moved from direct objects to subjects. They are the ones taking action
rather than being passive participants in their own healthcare. They are connecting to other patients from around the
globe, building communities and collaborations, and conducting their own research from within their own homes.

Though there was a time when all a next-generation advocate who wanted to take a hands-on approach to drug
development could do was launch their own drug company—the John Crowleys of the world—the disaggregation of
research, discovery, drug development, and manufacturing has unshackled these processes from the pharmaceutical
industry and given rare disease advocates the ability to access the expertise and tools along the full continuum from
the lab to the patient.”

NEXT report provides an overview of developments across the rare disease landscape and highlights trends in research, diagnosis, development, and treatment, as well as the changing regulatory and financial environment. Read about:

  • How new genome sequencing technologies are cutting the cost and accelerating the speed of making a diagnosis
  • How patients are taking a hands-on approach to research to change the fundamental understanding of some rare diseases
  • What advances were made on the policy front in rare disease and the unfinished work that will be addressed in the coming year

Read the report

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