When the Orphan Disease Patient speaks, listen.
Patients and their oftentimes sophisticated advocates continue to voice their needs and wants; which mostly center around the obvious - research for treatments and cures.
“In an environment where scientists are many and trial participants are scarce, patient groups are starting to organize their own clinical trial networks and offer them to scientists because they realize their registries, their tissue banks, their biobanks, and their experience are the key resources.”
Patient groups can be crucial to solving three primary challenges faced when developing orphan drugs:
Provide patient reported data, tissue samples, and natural histories;
Provide access to, and education for, constituents regarding clinical trial enrollment opportunities; and
Provide federally mandated patient guidance and input to clinical trial design
Patient engagement has been called the “blockbuster drug” of the 21st century. No longer content to be bystanders in their own medical journey, today’s patients, particularly those with rare diseases, demand an interactive experience with the healthcare system and a voice in the development of management strategies as well as new treatments.
Whitepaper written by Dan Donovan & Tony Howell