The Catalyst

Stephanie Fischer

Stephanie is former Senior Director of Communications at PhRMA, focusing on science advocacy and regulatory issues. As a rare disease patient and stroke survivor, she is very passionate about the need for public policy that encourages innovation and the development of safe and effective new therapies.
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Recent Posts

New Report Highlights a Decade of Innovation in Rare Diseases

Posted by Stephanie Fischer on March 5, 2015 at 12:00 PM

Despite Scientific Challenges, Biopharmaceutical Researchers Making Progress against Rare Disease

While any given rare disease affects fewer than 200,000 people in the U.S., rare diseases cumulatively affect 1 in 10 Americans and an estimated 350 million people worldwide.

On Saturday, there were efforts around the world to mark Rare Disease Day. This year’s theme, Living with a Rare Disease, put the focus on the daily impact on patients, families and caregivers:

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Topics: Medicines in Development, Science, FDA, Rare Diseases, Patients, chronic disease, Cystic Fibrosis

Empowering Patients to Be Active Participants in Clinical Trials

Posted by Stephanie Fischer on October 2, 2014 at 6:58 AM

One of the topics of discussion at the recent Rare Patient Advocacy Summit hosted by Global Genes was the role of patients in healthcare.  A statement that clearly resonated with the audience, from Roni Zeiger of Smart Patients, was that patients are not passive recipients of care and in clinical trials, but rather are co-collaborators. 

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Topics: Rare Diseases, Patients, Eli Lilly, Clinical Trials

Collaboration is What Will Help Drive New Successes for the Rare Disease Community

Posted by Stephanie Fischer on March 26, 2014 at 2:15 PM

While the number of patients with any individual rare disease is relatively small (less than 200,000 in the U.S., as defined by the Orphan Drug Act of 1983), they collectively affect 1 in 10 Americans.  The community successfully comes together to raise awareness on international Rare Disease Day every February, but the challenge is how to maintain the momentum – and remain united - for the remaining 364 days of the year. 

Global Genes has been tackling this challenge since 2009, with a mission that centers on unifying and empowering the rare disease community.  It is fitting that our next guest blog in the series on collaboration in rare diseases is from Andrea Epstein, Executive Director of Global Genes. 

You can find the first two guest contributions in our series here (by Dr. Stephen Groft of the Office of Rare Diseases Research at the National Institutes of Health) and here (by Peter Saltonstall of the National Organization for Rare Disorders).

On the heels of the 7th annual World Rare Disease Day, rare disease stakeholders from across the globe gathered in places of business, at events, in schools, places of worship, all in an effort to build awareness collectively for rare disease, a cause affecting over 350 million people worldwide.   This has become the most successful campaign ever for rare diseases because it was the first truly collaborative effort, allowing individuals and organizations to become part of the global campaign while making their local efforts their own.  Collaboration for rare disease is progress, collaboration is essential, and collaboration is what will help drive new successes for the rare disease community now and well into the future.

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Topics: Rare Diseases

NORD on the Need for More Collaboration to Drive Progress for Rare Disease Patients

Posted by Stephanie Fischer on February 17, 2014 at 2:28 PM

Many biopharmaceutical companies are actively engaged in the research and development of therapies to treat rare disease, despite challenges such as much smaller patient populations than more common diseases. Companies seek opportunities to partner with patient advocacy organizations, academia and the government in order to make even greater progress against rare disease, which collectively affect 1 in 10 Americans.

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Topics: FDA, Rare Diseases

#RarePOV Conversation Generates Strong Enthusiasm for Rare Disease Day on February 28th

Posted by Stephanie Fischer on February 12, 2014 at 2:58 PM

In last week’s #RarePOV tweetchat, leading rare disease advocacy organizations and individual patient advocates came together to share resources and plans for the upcoming international Rare Disease Day. More than 100 individuals and organizations participated, generating more than 680 tweets in the hour-long conversation and building on the momentum from the first #RarePOV tweetchat

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Topics: Rare Diseases

Addressing Challenges in Rare Disease Drug Development - Together

Posted by Stephanie Fischer on January 27, 2014 at 2:02 PM

Dr. Richard Moscicki, Deputy Center Director for Science Operations at the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA), noted in his remarks  at the FDA public workshop on Complex Issues in Developing Drug and Biological Products for Rare Diseases held earlier this month that more than a third of the novel drugs approved in CY13 treat rare disease.  With 450 therapies in the pipeline, as shown in PhRMA’s report on Medicines in Development for Rare Diseases, we are optimistic that this trend will continue. 

One of the themes of the FDA workshop was the need for closer collaboration between the many stakeholders in the development of therapies for rare disease.  Together, the biopharmaceutical industry, academia, government (particularly FDA and National Institutes of Health) and patient advocacy organizations can make more progress in the quest for new treatments.

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Topics: Medicines in Development, FDA, Rare Diseases, NIH

Rare Disease Community Loses a Champion

Posted by Stephanie Fischer on January 15, 2014 at 3:09 PM

The rare disease community lost a champion last week when Sam Berns passed away at age 17 due to complications from progeria, an extremely rare genetic disease which causes accelerated aging.  Last October, Sam shared his inspiring philosophy for a happy life in his talk at TEDxMidAtlantic.

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Topics: Rare Diseases

Know the Signs of Stroke and Act Quickly

Posted by Stephanie Fischer on May 14, 2013 at 2:50 PM

May 15, 2003 came to be known as the Very Bad Day.  It started off like any other, and I remember thinking in the morning that work was quieter than expected.  Several hours later, I was in the ICU while doctors at George Washington University Hospital monitored my vital signs and ran tests to determine what had caused a stroke in a seemingly-healthy 29 year old (who didn’t smoke, use illegal drugs or abuse alcohol).  

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Topics: Stroke Awareness

PDUFA: Still A Weird Word Worth Knowing

Posted by Stephanie Fischer on January 24, 2013 at 12:02 PM

As my predecessor noted, PDUFA is indeed a weird word worth knowing. The Prescription Drug User Fee Act (PDUFA), reauthorized last year as part of the Food and Drug Administration (FDA) Safety and Innovation Act, represents an agreement between the FDA and biopharmaceutical industry with significant input from patients and other stakeholders. It allows the FDA to collect user fees from biopharmaceutical companies so that the Agency has the resources it needs for a thorough and timely review of a medicine's safety and efficacy.

In the last 20 years, PDUFA has provided patients with timelier access to more than 1,500 new drugs and biologics and decreased review times for these treatments by more than 60 percent.

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Topics: FDA, Drug Development, PDUFA

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