For our families, Duchenne research can’t move fast enough.
There is no lack of ideas or interest—7 public companies and more than 15 biotechs are focused on finding treatments for Duchenne. But promise is only one part of the story. We need to actually change the landscape and bring drugs to market faster.
You can help make it happen with one simple action:…Continue
Added by Pat Furlong on December 4, 2013 at 11:00am — No Comments
2013 has been a year of incredibly high highs and incredibly low lows. Sometimes those lows have overshadowed all the highs—and for good reason. It’s impossible to put into words how devastating the setbacks are.
But our community is and has always been stronger than the setbacks. We may be fighting a disease that takes away muscle, but you are the strongest people I’ve ever known. It’s during our biggest challenges that strength matters most, and I know we’ll get through this…
Yesterday was a disappointing day for all parents, grandparents, aunts, uncles, and others touched by Duchenne. Like most of our community, I awoke to the news that multiple factors have led Sarepta to announce that it would delay the filing of its much-anticipated…Continue
PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community…Continue
Added by Pat Furlong on November 6, 2013 at 3:00pm — No Comments
When Chris and Patrick were diagnosed, there was no genetic testing. No therapies were targeted to the “CODE” and no way to DECODE Duchenne. We made assumptions about carrier status and lived many years with only assumptions. I thought my mother was a carrier. I knew I was a carrier. The doctor made that…Continue
Added by Pat Furlong on October 23, 2013 at 10:30am — No Comments
Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments
For many years, patient representatives have been invited to participate in meetings around drug development as the patient voice is extremely important if reviews are to understand how patients weigh benefit and risk within the context of their disease. I was honored to present earlier this week at meetings for NINDS and the FDA. Below are some of the notes I took and some of the information I heard that I wanted to share with you all.
How does it work? FDA issues guidance…Continue
Added by Pat Furlong on September 13, 2013 at 2:21pm — No Comments
Today is a landmark day for the entire Duchenne community. Not too long ago, the Duchenne therapy landscape was a barren field with little to no life. Thanks to a commitment to our patients and the field by the U.S. government, international governments, the venture community and industry, this landscape has changed markedly to the point where today we have the announcement for a…Continue
July 9, 2013.
PPMD met with Dr. Janet Woodcock and leadership from the Office of New Drugs, Medical Policy and the Division of Neurology Products, as well as, staff from the Office of Strategic Programs, Office of Health and Constituent Affairs. The purpose of the meeting was to continue our efforts to provide the agency relevant information and data about Duchenne that will encourage/enable them to be flexible in their review process.
Added by Pat Furlong on July 12, 2013 at 10:39am — No Comments
On July 9, PPMD will be meeting with individuals from FDA to include CDER, Division of Neurology, Office of Strategic Programs and Staff from the Office of health and Constituent Affairs.
The topics for discussion will include a review of PPMD’s Board Policy, developed in 2011. This policy embraces the following principles supporting an enhanced regulatory framework to advance development of drugs for rare diseases:
Added by Pat Furlong on July 3, 2013 at 1:30pm — No Comments
Today, I will be speaking with Treat NMD and European Medicines Agency (EMA) in response to their draft guidance on clinical studies in Duchenne muscular dystrophy. Elizabeth Vroom from Duchenne Parent Project in the Netherlands, Filippo Buccella from Parent Project Onlus in Italy, and I will be speaking on topics like clinical trials, inclusion criteria, and…Continue
June 7/8, 2013
In February, 2013 EMA (Europeans Medicines Agency) released draft “Guidelines on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy” open for comment. This is the result of advances in basic and clinical research with an ever increasing number of clinical trials, recruiting small numbers of patients for these studies. This has raised a number of issues to include study design, the choice of appropriate…Continue
Added by Pat Furlong on May 30, 2013 at 1:26pm — No Comments
Several weeks ago, I was invited to participate in a discussion focused on how large pharmaceutical companies should communicate with advocacy; how communication needs to be different in rare disease; and more specifically what advocates in rare diseases expect, want, and need from pharmaceutical companies. I was one of…Continue
Added by Pat Furlong on May 22, 2013 at 12:30pm — No Comments
Wading through policy is no easy task. But imagine policy as the white lines on the road, the boundaries within which FDA operates. Building our case, showing how Duchenne fits into those white lines and demonstrating how and when Accelerated Approval makes perfect sense is the purpose of PPMD’s white paper. …Continue
Added by Pat Furlong on May 20, 2013 at 11:51am — No Comments
Two weeks ago, PPMD proudly published Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne. In that short amount of time, the reaction to this white paper has been phenomenal. From interest by the media to accolades from industry and agencies, we have been truly humbled by the response we have received.
And while the…Continue
Added by Pat Furlong on May 9, 2013 at 1:00pm — No Comments
April 29-30 in Washington, D.C.
Recently I had the privilege of attending and participating in the Symposium on Best Practices in Clinical Study Design of Rare Diseases in Washington, D.C. The symposium was organized in collaboration with Children’s National Medical Center (CNMC), Clinical and Translational Science Institute of Children’s National (CTSI), The George Washington University, and Research in Pediatric Developmental Pharmacology Centers (RPDP). This…Continue
Added by Pat Furlong on May 2, 2013 at 4:16pm — No Comments
CLINICAL TRIALS – The path to approval.
Typically we think about the Clinical Trial Process in the context of our kids. We hear about Phase I trials. Depending on the drug/biologic, many of these compounds will be required to be tested in healthy human subjects. Some Phase I trials have inclusion criteria, suggesting they are looking for healthy adults of a certain age to take a single or multiple doses of a compound or biologic. The purpose of the study…Continue
Added by Pat Furlong on May 1, 2013 at 10:30am — No Comments
I have met some amazingly innovative people in my travels. Many who are dedicated to improving the lives of those living with rare diseases. Wendy White is one of those people. Wendy is the founder of Siren Interactive. Siren is a digital relationship marketing agency focused exclusively on addressing the…Continue
Added by Pat Furlong on April 29, 2013 at 10:00am — No Comments
Added by Pat Furlong on April 22, 2013 at 12:00pm — No Comments