The answer is ‘yes,’ which is why World Hemophilia Day 2017 is raising awareness of the many women with bleeding disorders who often go overlooked and undiagnosed.
By Jerry Powell, MD
April 13, 2017
No other genetic disease has made as much progress as hemophilia over the past half century. The typical life expectancy for boys born with severe hemophilia has increased from approximately 20 years to near normal life expectancy. One reason for this tremendous progress has been the involvement of patients and their families in supporting research and development (R&D) and participation in clinical trials, which has brought new innovative treatments to market.
Managing the cost of prescription drugs is a major economic challenge. As a result, prior authorization requirements are more demanding than ever. And, for doctors and patients, navigating the maze of regulations and restrictions can be frustrating at best. At worst, patient access to lifesaving therapies sometimes hangs in the balance.
A paradigm shift is required for people with rare medical conditions who require chronic care with medicines that are life-sustaining. This is a cost for life, and payers should focus on lifetime management cost. But instead, from the reimbursement coverage perspective, the process has become more granular and we are seeing more and more hurdles being created.
One of the things I like to remind our people to do is remember where we came from. In reflecting on my recent interview with Medical Marketing & Media (MM&M) magazine, in which I talked about CSL Limited’s unique beginning as a government agency, it strikes me that we really are unlike other biotechs.
Founded by the Australian government in 1916, CSL was born to serve people. It wasn’t until 1994 that we were privatized. CSL was established to ensure a continuous supply of medicines to Australians would not be disrupted by World War I. As I mentioned in my interview, we started with a mindset that reflected our government heritage – we’re always scrapping.
As we look ahead to spring, which is always a busy time for all of us at CSL Behring, we join the hereditary angioedema (HAE) and rare disease communities around the world in celebrating the 10th international Rare Disease Day on February 28. Every year at this time, we proudly join with hundreds of patient organizations worldwide to raise awareness of rare diseases and their impact on people’s lives.
There are upwards of 7,000 rare diseases, with approved treatments for only 5% of these. HAE, a rare and potentially life-threatening genetic condition, occurs in about 1 in 10,000 to 1 in 50,000 people. People with HAE can have considerable swelling episodes referred to as angioedema, which can affect many parts of the body, including the face, abdomen, larynx and extremities.
This year I again had the privilege of performing community service along with colleagues from CSL Behring on Martin Luther King, Jr. Day of Service. This opportunity was made possible through a giving back initiative supported and encouraged by our company.
We traveled from our offices in King of Prussia, Pa. to Cradles to Crayons, an organization based in Conshohocken, Pa. that does fantastic work providing children from birth to age 12, living in homeless or low-income situations, with the essential items they need to thrive. We spent our time cleaning and sorting shoes, preparing them for the little (and some not so little) feet of the children who would be receiving them.
It was a rewarding experience and a chance to take time out from our normal work day to reflect on the needs that exist in our community. Many of us are fortunate enough to not have to worry about how we’re going to get our next pair of shoes when the soles start to wear, or how we’ll keep our children warm when the temperature drops. But many children and parents in our communities are not as fortunate.
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