scientist and drug developer, the EveryLife Foundation
focuses on catalyzing and accelerating the development
of drugs for rare and orphan diseases. Before
founding EveryLife, Dr. Kakkis was Chief Medical
Officer of BioMarin Pharmaceuticals and is currently
the CEO of Ultragenyx Pharmaceuticals. IPPF President
Badri Rengarajan, MD, interviewed Executive
Director Julia Jenkins.
Q: What does the EveryLife Foundation do?
A: Our organization works to create scientific-based
public policy to improve clinical development and
the regulatory process in rare diseases. The Orphan
Drug Act was passed more than 30 years ago. Yet
drugs have only been developed and approved for
five percent of them (400 diseases among the 7,000
rare diseases). [Author note: In the US, a disease that
affects fewer than 200,000 people is considered
an orphan disease, and a disease that affects fewer
than 50,000 people is considered an ultra-orphan
Q: What is distinctive about what your organization
does relative to other rare disease organizations?
A: Our organization was created specifically to create
change and improve the drug development process.
We focus on policy issues that no other organization
Q: Do you have a focus on certain types of rare disease
or certain situations?
A: Our initiatives are beneficial for any rare disease.
Improvements in the drug development process will
benefit all rare diseases. However, our primary focus
is on ultra-orphan diseases, which make up 80
percent of rare diseases. Our motto is “no disease
too rare.” We also focus on diseases in which no
treatments have been developed (which are most of
them). It should be noted that 83% of diseases have
fewer than 6,000 patients, yet only 18% of orphan
drugs have been approved for ultra-orphan diseases.
We are trying to address this lopsidedness.
Q: Who are your constituents or “customers”?
A: We work with patient organizations and, in some
cases, parents and patients. We work mainly with
smaller patient organizations (so-called “kitchen table
organizations”). We also do some work with industry
and regulators, and academics. We hold workshops
to develop science behind drug development and
regulatory process for rare disease drugs.
Q: What are some significant programs of the EveryLife
A: CureTheProcess: We want to accelerate biotechnology
innovation and increase the predictability of
the FDA’s regulatory review process.
Rare Disease Legislative Advocates: We support
legislative advocacy for all rare disease groups with
events such as lobby day, Capitol Hill Briefings, and a
gala. We want to empower the individual to become
an advocate by providing informational meetings,
legislative resources, advocacy tools, and special
events that support organizations and advocates
working to promote rare disease legislation.