“Everyone Deserves A Deep Breath”
Interview originally published in PharmaTimes Magazine and available here
Emily Kramer-Golinkoff was six weeks old when she was diagnosed with cystic fibrosis. She is now 29, works in the social media and health innovation lab at Penn Medicine, and has set up a charity to raise funds for CF research
What is Emily’s Entourage?
In December 2012, my family, friends and I founded the charity Emily’s Entourage (www. emilysentourage.org), which raises funds and awareness to accelerate the pace of cystic fibrosis research. Recent breakthroughs are changing the face of CF but, unfortunately, they don’t benefit many people like me with rare mutations. So Emily’s Entourage is focused on finding new treatments and ultimately a cure for CF with an accent on these rare mutations. So far we’ve raised more than $650,000 and we’ve done that largely by mobilising our communities through email and social media campaigns on Facebook, Twitter and Instagram.
How do you use social media to manage your CF?
Social media plays an integral part in my disease management. People with cystic fibrosis can’t be near each other because of the possibility of cross infection, so online communities enabled by social networks are a lifeline for us. There is a thriving online CF community where we share information, innovative ideas, research, and the kind of compassion and support you only get from people who are living and breathing the same disease as you. Social media has introduced me to some of my most effective off-label therapies, which have played a tremendous role in stabilising my health.
How would you describe the healthcare and treatment you’ve received?
My healthcare has evolved significantly over the years. Earlier experiences at paediatric CF clinics didn’t jive well with my desire to be an active participant and decision- maker in my care, but I’m fortunate that I finally landed at an adult CF clinic that values my expertise as a patient and encourages active collaboration. My current healthcare team is outstanding and I credit our partnership with helping to keep me stable these past few years, despite the advanced state of my disease. I want to live a happy, fulfilling and productive life, see beautiful places, surround myself with the people I love, and contribute to the world in impactful ways. Successful treatment is having access to healthcare that enables me to do all that.
What are your thoughts on the pharmaceutical industry?
The 2012 approval of the breakthrough drug Kalydeco is saving lives for the small sub- population of people with one particular CF mutation – and that’s thanks to the pharma/biotech industry. They are doing amazing things; personalising therapies to individual genomic characteristics and giving us real hope. But it counts for nothing if patients can’t access these medications because of exorbitant costs. I understand we’re a small disease population, and I know that R&D is costly and risky, but the pharma and biotech industries must think of the people, the mums, dads, daughters, sons and best friends their work can save, and make sure these people can get the drugs they desperately need to live, and to live well.
What would you like from pharma companies?
I would love the pharma industry to tackle the development of new therapies and even cures for those with rare diseases, including rare mutations of rare diseases. The development and approval of Kalydeco proves that it’s possible. I’d also like them to remember the people at the end of the drug development process who are waiting desperately – in my case with bated breath – for new therapies, and to engage them as co-designers and partners in the process. We are all after the same thing here – breathroughs and medical advances – and we’ll get a lot further by working together. After all, everyone deserves a deep breath, don’t they?