Walking the Walk in Specialty Pharmacy: How Advocacy Creates Tighter Bonds With Patients

CF Progress Made Possible

In 1989, the CF gene was discovered by a team of scientists who were supported by the CFF. This discovery was the beginning of being able to comprehend the disease at the most basic level.
In the 1990s, treatments that tackled symptoms of the disease became available, however, there were still no drugs that pinpointed the underlying cause of the disease. At the time, pharmaceutical companies were not focused on developing drugs for rare disease states such as CF. Instead, manufacturers were focused on developing blockbuster therapies that would treat the masses and subsequently turn a large profit.

A decade later and spurred by desperation for faster progress, the foundation sought to encourage the pharmaceutical industry to become more involved in CF research. Venture philanthropy was a risky move and had no guarantees, but the CFF pushed forward.

The CFF donated millions of dollars, leading the movement of voluntary health organizations funding drug development with for-profit companies. Although the majority of drug companies were not interested in the proposition put forth by the foundation, 2 smaller companies returned the call.

In 2000, Aurora Biosciences (now Vertex Pharmaceuticals), was the first pharmaceutical manufacturer to receive a large investment from the foundation with the intent to uncover compounds that would correct the core defective gene associated with CF.

On January 31, 2012, the foundation’s investment finally paid off when the FDA approved ivacaftor (Kalydeco). Ivacaftor was the first drug to address the fundamental cause of CF.

Although this drug only treated a minority of patients, it was a historic breakthrough for the CF community and gave way to the development and approval of the combination medication lumacaftor/ivacaftor (Orkambi). The combination was approved by the FDA in 2015 and worked in half of the CF population.

In February 2018, the FDA approved Vertex’s third CF product tezacaftor/ivacaftor and ivacaftor (Symdeko), another milestone that furthered the commitment to eventually be able to treat every patient with CF.  

Next Steps

Now, this article is not intended to be a plug for the CFF (although they are an incredible organization), but rather is a call to other clinicians and specialty pharmacies to consider getting involved with the organizations that focus on specialty disease states. The CFF’s venture philanthropy model has received praise from leaders in medicine, health care, business, and even by President Barack Obama.

It has also been emulated by other rare disease foundations in an effort to advance the research and development of treatments for other rare disease states. Furthermore, the CFF is a perfect example of how nonprofits and patient groups can form mutually beneficial partnerships with pharmaceutical manufacturers and researchers to collaborate and produce innovative treatments for diseases.  
In our role as specialty pharmacists, we encounter many patients who are living with a serious and chronic condition, such as CF. However, most of us never get the opportunity to meet our patients face-to-face or to even talk to them on a regular basis.

Through working with the foundation, I got to meet patients and families who were living with CF and whose lives have been changed by the progress that has been made. I felt more connected to the people who we service on a daily basis. It also put things in perspective, such as how critical it is to get a patient’s order out and how much our patients rely on our timeliness, expertise, and professionalism.

Whether it is a cause that is close to your heart or that you feel passionate about for other reasons, there are great ways to get involved. Contributing does not have to be monetary and giving your time or talents to fundraising efforts can make a world of difference.

It takes a lot of effort to put on a successful fundraising event and lending a hand is one of the best ways to help. Countless coworkers and friends jumped on board to help make my events successful.

It was touching to see but it also made me realize that what we do every day in specialty pharmacy is meaningful not only to our patients but to each other as well. As I found out through my experience, it wasn’t just money that we raised, but instead a sense of community and awareness. It was a different way to go above and beyond to support the specialty pharmacy patient.

Small biotech companies are fueling the progress of how we treat rare diseases in novel ways. It is both inspiring and astounding to live in a day and age in which gene therapy and precision medicine are giving way to potential cures for hereditary conditions.

The work being done today with nonprofits such as the CFF is funding research for the breakthrough therapies that we will dispense in specialty pharmacy tomorrow…and for our patients, those breakthroughs are adding tomorrows.

About the Author
Jacqueline Hanna earned her Bachelor of Science in Biological Sciences from the University of Pittsburgh before earning her Doctor of Pharmacy degree from Duquesne University in 2011. She recently received her Master of Science in Pharmacy Business Administration (MSPBA) program at the University of Pittsburgh, a 12-month, executive-style graduate education program designed for working professionals striving to be tomorrow’s leaders in the business of medicines. Jacqueline has spent the last 4 years working in Specialty Pharmacy, initially as a clinical pharmacist and most recently working on a variety of high-profile Specialty Operations Projects. In her current role, she is able to channel her passion for patient care into innovation and process design while being part of a concerted effort to transform Specialty Operations and improve the patient experience.
 
 






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