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.
We often say innovation is in our DNA at CSL. But without financial and scientific commitment to a dynamic R&D program, innovation would not be possible. The long-term sustainability of CSL requires a full R&D pipeline of high-quality prospective products. This is why CSL has invested $2.3 billion in R&D over the last five years and why we support research teams all over the world. It is also the reason we are committed to identifying and supporting the best and brightest biomedical researchers in Australia.
Our commitment to R&D extends to the broader scientific community beyond the CSL laboratories. An example of this is the CSL Centenary Fellowship program. This $AUD 25 million program awards two, five-year $1.25 million fellowships each year to early/mid-career Australian medical researchers, who are working on world-class discovery or translational research in the areas of rare and serious diseases, immunology and inflammation.
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