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

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


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