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


Involvement with the organizations and nonprofit foundations that focus on specialty disease states can help specialty pharmacists gain perspective on the patients they treat.

This past year, I had the opportunity to work with the Cystic Fibrosis Foundation (CFF) of Western Pennsylvania to raise money towards finding a cure for the disease. Before partnering with the foundation, I did not have any close connection to cystic fibrosis (CF) other than being a specialty pharmacist.

No one in my immediate or extended family had CF. Other than counseling patients and caregivers in my daily work and hearing about other people’s experiences with the disease, there was no personal motive driving me to get involved with this organization.

However, when a friend approached me about being part of a large fundraising effort for CF, I didn’t think twice. As a specialty pharmacist, I felt like it made sense and I didn’t have to be personally affected by the disease to care.

I knew that the foundation was doing great things and that most of their fundraising efforts went towards funding the research and development of drugs to treat and, hopefully, cure CF someday.

In my relatively short career as a pharmacist, I could see the progress the foundation was making right before my eyes. CF was among a handful of disease states in which treatment breakthroughs, primarily over the last few years, had significantly extended the life expectancy and prognosis of individuals suffering from the disease.

It was not that long ago that patients with CF only had a life expectancy of approximately 10 years; however, patients are currently living well into mature adulthood. With recent drug developments, not only has life expectancy been greatly increased but the quality of life has also become much better as well.

About CF

CF is a hereditary disease that affects the mucus and sweat glands. There are approximately 30,000 people living with CF in the United States and approximately 1000 new cases are diagnosed each year.

Three-fourths of cases are diagnosed by 2 years of age, making this a lifelong disease. Most frequently associated with causing persistent lung infections, CF can also affect other parts of the body, such as the sinuses, the organs of the digestive system (liver, pancreas, and intestines) and the sex organs.

The genetic disorder is caused by various mutations in the cystic fibrosis transmembrane conductance regulator (CTFR) gene. There are more than 1800 known CF mutations but the actual number of mutations could exceed this figure as the majority of genetic tests only screen for known mutations.

Historically, the medications available for CF were limited in number and typically focused on treating the symptoms and complications of the disease, not necessarily the cause. However, over the last few years, several medications have been approved by the FDA that specifically target the CFTR gene.

Currently, half of patients living with CF are over 18 years old. As previously mentioned, a few short decades ago these patients were not even expected to live into adulthood. Following these medication advances, the median age of survival for someone living with CF is now 33.4 years old and some patients are living well beyond this into their fifties and sixties.

Biotech Startup Funding Sources

Just like any business endeavor, budding biotech companies need initial startup capital for a variety of things. Money is primarily used for research, product development, overhead costs, and other permits and licensure.

There are several ways that startups can seek out funding, such as grants, venture capitalists, bank loans, an initial public offering, or via private and angel investors. The term angel investor usually refers to individuals with capital that they use to invest privately in new businesses in exchange for a significant share of the company. Private investors are usually those who have a select interest in the company or the product being developed.

Bank loans are easier to obtain if the company already has paying customers and a thorough business plan. Another way to garner additional funding is going public with the company and selling shares of stock.

More established biotech companies should weigh the pros and cons of an initial public offering and whether it is in the best interest of the company. Finally, grants can also provide a substantial amount of funding for biotechnology companies.

Those that are still involved in the academic research stages may qualify for government funding for equipment and staff. The National Institutes of Health (NIH) can be a powerful partner for biotechs; however, in recent years, the NIH’s fluctuating budget have prompted many institutions to seek nonprofit disease foundations for another source of funding and grants.

The following list of entities have been among the top grant-giving disease foundations in the last 5 years. This list is not comprehensive but showcases the contributions that disease foundation fundraising has offered in recent years.

Many are focused on funding for specialty disease states.

  • Cancer Research Fund of the Damon Runyon-Walter Winchell Foundation
  • The Michael J. Fox Foundation for Parkinson’s Research
  • National Alliance for Research on Schizophrenia and Depression
  • Melanoma Research Alliance Foundation
  • Rheumatology Research Foundation
  • Alzheimer’s Drug Discovery Foundation
  • Brain Research Foundation
  • Parkinson’s Disease Foundation
  • CFF
  • American Association for Cancer Research
  • Hereditary Disease Foundation
  • Crohn’s & Colitis Foundation
  • Myelin Repair Foundation
  • The ALS Association
  • Leukemia & Lymphoma Society
  • National Multiple Sclerosis Society
  • Asthma and Allergy Foundation of America
  • National Hemophilia Foundation
  • American Liver Foundation
  • Susan G. Komen Breast Cancer Foundation

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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