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Insights From a Patient Advocacy Summit

Posted by | 7:31pm on Thursday, November 8, 2012
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only 5% of all rare diseases have an FDA-approved drug treatment 

At the inaugural Global Genes Project 2012 Patient Advocacy Summit it was apparent that patient advocacy in the rare disease space is continuing to gain momentum and that patient advocacy organizations and patient opinion leaders can provide substantial value for pharma—their input can help in conducting research, expediting clinical trials and developing therapies. The summit (held September 28, 2012 in Newport Beach, California) served as the ideal forum for discussion around the unmet needs of rare disorder patients and caregivers.

Rare diseases affect 30 million people in the US and 350 million globally. “Only 10% of the 7,000 rare diseases have organizations or foundations providing support or driving research,” noted Nicole Boice, Global Genes | RARE Project Founder & President. The Global Genes Project is a rare and genetic disease patient advocacy nonprofit organization started in 2009. It’s estimated that only 5% of all rare diseases have an FDA-approved drug treatment, with less than 400 treatments approved for the nearly 7,000 diseases. According to estimates from the National Institutes of Health, at the current rate of FDA drug approvals, it will take 10,000 years to find therapies for all people suffering from rare diseases.

A bridge between pharma and patients
The event included discussions on patient resources and quality of life, disease research and funding, clinical care and payers. I had the pleasure of participating as a panelist, and much of the content that I shared was about the process for establishing a bridge between pharma and patient advocacy groups, and the value of the synergy. Biopharmaceutical companies and rare disorder patients/caregivers have a shared goal of developing an FDA-approved treatment. Patients are looking for pharma to invest in developing orphan drugs, bringing them to market, and providing support and access to information on rare disease diagnosis and treatment. See the video below of the hour-long panel discussion, skip to 25:20 if you’d like to hear my presentation.

Lack of understanding
Patients don’t always recognize corporate limitations and regulatory hurdles that can restrict pharma interaction and they often don’t know where to start when they want to engage with pharma. Similarly, pharma doesn’t always understand the burden of disease and the patient’s sense of urgency. It can also be difficult for pharma to navigate the landscape of patient advocacy groups to identify an appropriate partner with common objectives. Vital to effective collaboration is mutual commitment to well-defined goals with measurable benchmarks and ongoing communication.

Champions of hope
The night before the summit, the Global Genes | RARE Project also hosted 400 guests at the First Annual Tribute to Champions of Hope to raise funds and awareness of rare disorders. The gala acknowledged some of the amazing people behind the advancements in rare diseases. Most impressive were the heartfelt words shared by patients and the recognition bestowed upon a short list of industry honorees who received awards for specific achievements.

I was inspired and energized after attending the gala and summit, and I look forward to next year.

About Frieda Hernandez

Frieda leads Siren’s business development, marketing and alliance activities. She has a reputation for developing innovative marketing strategies and building mutually beneficial client partnerships with biopharmaceutical executives. She advocates for authenticity and transparency in creating trust relationships online and is an avid participant in industry conferences and social networks to stay abreast of all the current issues.

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