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Breaking Down Barriers for Patients With Rare Diseases

Why improving access to care is on my mind this February – and every day.

CSL CEO and Managing Director Paul Perreault

There are a few dates on my calendar each year that lead me to reflect. Like family birthdays or New Year’s, Rare Disease Day is one of those special dates for me. The days leading up to February 28 are a time when I like to take stock of how we have delivered on our promise to patients over the past year. I ask myself if we are doing everything we can to help our patients live their best lives.

Patient focus is, of course, nothing new for me or for or the tens of thousands of CSL employees who work every day like someone’s life depends on it, because it does. Over the past year we have continued to develop truly innovative medicines that help save and improve the lives of those living with rare and serious diseases. Unfortunately, another constant has been continued barriers for patients to access lifesaving therapies.

These barriers can take many forms. In Europe, it may be a regulatory delay. In some developing nations, the pathway for patients to receive medicine has yet to be cleared. A global challenge for rare disease patients is the time it takes to get an accurate diagnosis. Tales of years-long waits and many visits to multiple doctors before receiving the right diagnosis are far too common among those with rare diseases.

These frustrating, yet solvable, hurdles to access are at the forefront of my thoughts this month as we lead into Rare Disease Day. Ensuring patients have access to medicines is an imperative in our industry, not an afterthought. That is why CSL Behring supports efforts for quicker diagnoses, faster approvals, more treatment options and finding ways to bring medicines to patients in emerging and developing markets.

I hope you will join me and the more than 22,000 Chief Patient Officers who work with me at CSL in taking some time over the next few days to reflect on the progress that has been made and the challenges that remain for helping people with rare and serious diseases. We will be setting aside some extra time this year to think about how we can make sure each and every one of our patients has the access to the lifesaving medicines we are proud to develop and deliver every day.


Paul Perreault is CEO & Managing Director of CSL Limited, the world’s fifth largest biotech company which provides lifesaving medicines for patients with rare and serious diseases in more than 60 countries.