Patient-driven access to approved rare disease treatments is crucial to Pharma. Patients are the most authentic and passionate in advocating for access. Improved access benefits patients and Pharma.

1. Pharma: Why Patient-
Driven Access is
Crucial

2. Worldwide orphan
drug sales
continue to
increase from
2.5% of total drug
sales in 2000, to a
projection of 22%
in 2020.

3. Rapid growth in
the orphan drug
market
contributed to
barriers in
accessing rare
disease
treatments.

4. Insurance
coverage is one of
the barriers
impeding patient
access to
approved rare
disease
treatments that
we will address.

5. Patients are the most authentic
and passionate in advocating for
improved access to rare disease
treatments.

6. Improved access to rare disease
treatments benefits both patients
and Pharma.

7. Pat Furlong is the Founding President and
CEO of Parent Project Muscular
Dystrophy (PPMD), the largest nonprofit
organization in the United States solely
focused on Duchenne muscular
dystrophy (Duchenne). Its mission is to
improve the treatment, quality of life,
and long-term outlook for all individuals
affected by Duchenne through research,
advocacy, education, and compassion.
John Williamson is a Senior Patient
Advocate at the US Hereditary
Angioedema Association. John advocates
on behalf of HAEA patients on a many
issues, including clinical trial design and
legislative reform. John is also an HAEA
patient.
Khrystal K Davis is a core member of
Families for Acceleration of SMA
Treatments (FAST). Khrystal regularly
advocates for patients with SMA and other
rare diseases. Her son, Hunter, was
diagnosed with SMA as a newborn in 2011.
Khrystal wrote “Hunt for a Cure: An
Unexpected Adventure to Save a Life,”
which chronicles the adventure to obtain
lifesaving treatments for Hunter.
Meet the Panelists
PF JW KD