Recently, Rare Disease Report sat down with Mariah Kelly, BSN, RN, Advocacy Lead, and Patient Affairs Liaison at Pfizer to talk about her company’s commitment to rare diseases, including a most recent program: Gaucher Personal Support (GPS). This specialized GPS program is designed as a one-stop resource for patients who are prescribed Elelyso™ (taliglucerase alfa) for injection. Elelyso is Pfizer’s enzyme replacement therapy for people with Gaucher disease.
RDR: What is the Gaucher Personal Support Program?
Ms. Kelly: The GPS program started in May 2012 following the Food and Drug Administration’s approval of Pfizer’s Elelyso. Prior to Elelyso’s launch, we at Pfizer held a series of advisory boards where we brought Gaucher patients together and asked them what type of support services they need. What’s working? What’s not working? How can we bring this program to the community where it is meaningful?
We listened to what they had to say, then built and fine-tuned the GPS program based on the patients input. We eventually launched GPS on May 1st, 2012. So GPS is truly a response to the community’s needs. It is a program built from the ground up for patients.
How does the Gaucher Personal Support Program help patients?
Gaucher Personal Support does just about everything. It provides reimbursement support. It helps with the paperwork needed by insurance companies.
GPS ships the drug and coordinates the infusion and has even been known to help coordinate rides to the infusion centers. If a patient wants to be infused at home, GPS works with the patient in setting up at-home infusions. For patients on commercial insurance, GPS offers co-pays and deductibles with a maximum of $10,000 per year per patient. So it is a very comprehensive program designed by patients for patients with Gaucher disease.
Who Runs the Program?
GPS is run by Centric Health Services – which is a specialty pharmacy in St. Louis Missouri that specializes in ultra-orphan drugs. Centric has case managers, nurses, pharmacists, and pharmacy technicians who run the GPS program 24/7.
How else is Pfizer committed to the rare disease and Gaucher community?
Pfizer understands the devastating impact that some rare diseases have on patients and caregivers, and we are dedicated to addressing these serious, unmet medical needs by discovering, developing, and delivering innovative orphan medicines. Pfizer is very proud to be part of the rare disease community and has 22 medications on the market for rare diseases approved worldwide. We are committed to working with the community to raise awareness, aid in diagnosis, and ensure access to treatments. Pfizer’s Rare Disease Research Unit (RDRU) in Cambridge, Massachusetts is adopting an innovative and collaborative approach to the development of new medicines whereby it looks to develop strategic partnerships with academic and commercial enterprises to create novel therapeutics across the spectrum of rare diseases.
In addition, Pfizer partners with many other rare disease organizations, including Jewish community organizations. Pfizer is currently collaborating with NORD to create an online physician guide about Gaucher disease for healthcare professionals.