News for Immediate Release
February 28, 2025
Harrisburg – The Pennsylvania Health Care Cost Containment Council (PHC4) is committed to ensuring better outcomes for all Pennsylvanians and has collaborated with the Pennsylvania Rare Disease Advisory Council (PARDAC) to bring a new resource to stakeholders invested in rare disease care in Pennsylvania.
This collaboration between PHC4 and PARDAC began in 2023, when PHC4 assisted PARDAC in analyzing over 1,200 survey responses collected from the rare disease community. The primary goal of PARDAC’s survey was to learn more about the needs of individuals, families and loved ones affected by rare diseases in the Commonwealth of Pennsylvania to make recommendations to improve access to needed resources. Adding on to our Current Events series, this second installment shows the results of this survey and includes an in-depth examination of why researching rare diseases is so challenging.
The rare disease community considers this project important in shining light on the lack of rare disease research and hopes it will encourage more researchers to get involved. Dr. Vockley M.D., Ph.D., PARDAC Board Member, stated “Individually, rare diseases are rare, but collectively they’re not. This collaboration is tremendous for progressing the discussion in rare disease research and highlighting some of the common challenges.” Dr. Vockley is the University of Pittsburgh Cleveland Family Endowed Chair in Pediatric Research, Professor of Human Genetics and UPMC Children’s Hospital of Pittsburgh’s Chief of Genetic and Genomic Medicine, and Director of the Center for Rare Disease Therapy. PARDAC seeks to further use this information to advocate in the spirit of their friend, and colleague, Anna Payne. She was a long-time champion of PARDAC’s mission, who until her passing this February from stage four colon cancer at age 37, served as the organization’s Vice Chair. Anna, born with Cystic Fibrosis, was known as a fierce advocate, as can be gleaned from this quote published in an article in 2021, “I learned at an early age to advocate for myself,” she said. “I learned to ask a lot of hard questions — questions maybe you really don’t want to know the answers to. I think it’s a good quality to have in life. It’s how you know where you’re going, and what you can do to change things…”
A common initial struggle for rare disease patients is reaching a diagnosis. The data in PARDAC’s survey shows many patients waited more than 5 years and saw more than 10 health care providers before receiving a final or accurate diagnosis.
PHC4 is an independent council formed under Pennsylvania statute (Act 89 of 1986, as amended by Act 15 of 2020) in order to address rapidly growing health care costs. PHC4 continues to produce comparative information about the most efficient and effective health care to individual consumers and group purchasers of health services. In addition, PHC4 produces information used to identify opportunities to contain costs and improve the quality of care delivered.
For more information, visit phc4.org or access the article here.
Media contact:
Barry D. Buckingham, Executive Director, PHC4
