New Global Genes Toolkit Series, From Molecules to Medicine, Helps Patients and Advocates Strengthen Their Understanding of Rare Disease Drug Development

Series describes how therapies are developed, the importance of
engaging in clinical research and trials, and case studies in effective
patient engagement

ALISO VIEJO, Calif.–(BUSINESS WIRE)–Rare disease patients now have new resources to help them better
understand and engage in clinical research and drug development, thanks
to Global Genes, a leading rare disease patient advocacy organization.

According to the National Institutes of Health (NIH), there are more
than 7,000 identified rare diseases that affect more than 30 million
people in the U.S. While the Orphan Drug Act has successfully provided
critical incentives for research and development for rare disease
treatments to enter the market, 95 percent of rare diseases continue to
lack any or adequate treatment.

“Patients continue to seek clear information about the length of time,
investment required, and research steps required to get to a
successfully marketed therapy for their rare disease,” says Nicole
Boice, Global Genes Founder and CEO. “It’s essential to our mission to
help inform and empower rare disease advocates, so they can learn from
compelling case studies and better prepare and participate in clinical
research and along the entire drug development pathway. Ultimately, this
engagement is central to success.”

The three-part From Molecules to Medicine series: How
Drugs & Therapies Are Developed
, Clinical Research and How
Patients Can Share their Voices Throughout the Drug Development Process
,
aligns with the Drug
Development Roadmap
release by Global Genes in the fall. That
Roadmap is an illustrated primer for any rare disease patient who wants
to learn about the process, and come away with greater knowledge on
where and how to participate.

“Rare disease advocates are more empowered than ever today to
meaningfully engage in research and drug development, and our From
Molecules to Medicine
Toolkit series clearly outlines key
information and relevant best practices from the rare community that can
serve as a catalyst for that engagement,” said Kym Kilbourne, VP,
Patient Advocacy, Global Genes.

In this age of rapidly advancing scientific discovery, it is fundamental
to provide rare disease advocates with information about participating
in drug development. Likewise, it is vital to ensure that public
policies continue to support patient engagement in drug development,
offer incentives to innovators to continue to advance treatments that
address unmet medical needs, and support access to those approved
treatments. The rare patient is at the center and needs to be equipped
through informational tools like the new From Molecules to Medicine
Toolkit series, to provide meaningful input and context in those
important dialogues.

The From Molecules to Medicine Toolkit series is available
free online to read
and download
. In addition to the expansive library of other critical
topics, Global Genes Toolkits are available to patients and their
organizations for educational purposes, including patient meetings and
conferences. To view the entire series or download any of the resources,
visit www.globalgenes.org/Toolkits,
or, for larger quantities in print, join us at Global Genes RAREhouse.

Global
Genes
is a leading rare disease patient advocacy organization. The
group’s mission is to eliminate the challenges of rare disease, by
providing patients with educational tools, building awareness, providing
critical connections to people and resources, and through investment in
technologies that will positively impact affected patients and families.
Recognized worldwide by the Blue Denim Genes Ribbon™, Global Genes
unites experts, advocates and patients of all ages to stand together in
hope for treatments and cures for the estimated 7,000 rare and genetic
diseases that impact approximately 30 million Americans and over 350
million people worldwide.

Contacts

The ACE Agency
Ashley Eckenweiler, 949-285-9239
ashley@theaceagency.com