Holly Peay's Blog (23)

PPMD’s Benefit/Risk work highlighted in groundbreaking reports

On May 13th the Medical Device Innovation Consortium (MDIC) released a report on incorporating patient preferences into the FDA’s benefit/risk assessments. It was much anticipated by PPMD as we continue our “aggressive engagement” with the FDA and industry trial sponsors. The report is full of really…


Added by Holly Peay on June 2, 2015 at 1:00pm — No Comments

FDA Acknowledges the Importance of Benefit-Risk Preferences: Why this Matters to Duchenne

This started as a blog about the second publication on PPMD’s benefit/risk study, which was published in late December in a journal called The Patient (and more on that later). But something remarkable happened this week that helps frame…


Added by Holly Peay on January 26, 2015 at 10:23am — No Comments

PPMD and Santhera Pharmaceuticals Team Up on New Benefit/Risk Study Focused on Pulmonary Therapies

PPMD is proud to announce a collaboration with Santhera Pharmaceuticals on a benefit/risk study that will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera’s successful phase III clinical trial of idebenone. One of the recommendations in our community’s…


Added by Holly Peay on November 25, 2014 at 9:00am — No Comments

Let’s improve DuchenneConnect’s informed consent. Give your opinion by September 7th.

You may be thinking, “An informed consent document? No way do I want to read one of those!” But you can demonstrate the power of our community and help us write a more understandable, responsible informed consent for our community’s registry.

As many of you know, DuchenneConnect was…


Added by Holly Peay on August 28, 2014 at 11:25am — No Comments

PPMD Benefit/Risk Study Results Published in Clinical Therapeutics

PPMD is thrilled that results from the recent benefit/risk project our team led have been published in Clinical Therapeutics. The article concludes that caregivers are willing to accept a serious risk when balanced with noncurative slowing or stopping of the progression…


Added by Holly Peay on June 9, 2014 at 1:30pm — No Comments

Simple Actions: Real data can drive change

For years our community has been making noise so that the scientific and medical communities would listen and focus on Duchenne. They're listening – now we need to talk! Filling out a survey or doing an interview about your family's experiences with Duchenne creates important data. This data is what clinicians, scientists, and government agencies need to develop…


Added by Holly Peay on March 18, 2014 at 9:30am — No Comments

DuchenneConnect Receives $970,000 Award

DuchenneConnect was recently awarded close to $1 million dollars in funding from PCORI, the Patient-Centered Outcomes Research Institute! 


DuchenneConnect will be one of 29 members of PCORnet, the National Patient-Centered Clinical Research Network.  The goal of PCORnet is to create a large, highly representative, national network for conducting clinical research.  This is especially…


Added by Holly Peay on February 19, 2014 at 12:30pm — No Comments

FDA Public Workshop Report – Our Message Is Having an Impact

Last week, Pat Furlong and I were invited by the FDA to their meeting on Complex Issues in Developing Drugs and Biological Products for Rare Diseases. I was asked to participate on the Tolerating Risk and Uncertainty in Pediatric Clinical Trials Panel, in recognition of the work PPMD has done to:


  1. Systematically collect data…

Added by Holly Peay on January 15, 2014 at 11:00am — No Comments

What do you think about clinical trials?

Though clinical trials aren’t for everyone, each trial helps move us toward better treatments.  What do you think about clinical trials?  Share your thoughts, so PPMD can give the best advice to professionals running trials.

At the recent World Muscle Society and Child Neurology Society meetings, we heard reports from…


Added by Holly Peay on November 13, 2013 at 1:04pm — No Comments

Pushing the FDA to Accelerate Treatments

We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for rare disease versus treatments for treat common…


Added by Holly Peay on October 10, 2013 at 9:30am — No Comments

Clinical Trial Expectations

I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might improve the clinical trial process. Highlighting the…


Added by Holly Peay on October 7, 2013 at 10:58am — No Comments

Benefit/Risk Survey Results to Be Submitted to FDA

Earlier this week, PPMD hosted a webinar to discuss our FDA “aggressive collaboration” approach and the results from our recent benefit/risk survey. We conducted a rigorous survey of more than 100 parents whose children have Duchenne, gathering invaluable perspectives as to treatment priorities and risk threshold based on realistic but hypothetical treatments – information that PPMD is encouraging regulators to use to…


Added by Holly Peay on June 14, 2013 at 10:00am — No Comments

Update on PPMD’s Risk/Benefit in Duchenne Therapies program

We were thrilled that 119 parents/guardians were willing and able to complete the entire treatment preferences survey. We are analyzing the data. We are doing the first part of the data analysis in-house, and the second part with our health economist collaborator. Our plan is to present this information to the FDA, to industry and clinical…


Added by Holly Peay on March 13, 2013 at 10:30am — No Comments

LAST CALL: Phase 1 Survey - Risk/Benefit in Duchenne Therapies

By now you’ve probably heard about PPMD’s Risk/Benefit in Duchenne Therapies program, which aims to inform the FDA and other government agencies, biopharmaceutical companies, and others about the treatment priorities and risk tolerance of the Duchenne community. We’ve had a great response to our first survey, with more than 90 participants so…


Added by Holly Peay on February 19, 2013 at 1:56pm — 1 Comment

Risk Tolerance, Benefit Preferences, and Treatment Priorities

The Duchenne community has entered a new era where more clinical trials are coming online and the possibility of treatment is becoming a reality. Upon completion of a trial, the FDA weighs the evidence presented. But at the end of the day, it is often a value judgment about potential benefits and risks to the patient.

PPMD has launched a program – Benefit/Risk in Duchenne Therapies – to help inform the FDA and other government agencies, biopharmaceutical…


Added by Holly Peay on February 13, 2013 at 3:30pm — 2 Comments

Educating healthcare providers about the need for early diagnosis

Families of children with muscular dystrophy and other muscle disorders often experience a diagnostic odyssey that is too long and too emotionally difficult. PPMD, with support from The Centers for Disease Control and Prevention (CDC), created the National Task Force for Early Identification of Childhood Neuromuscular Disorders to address the continued gap between the time when the first symptoms of muscle weakness…


Added by Holly Peay on January 9, 2013 at 2:00pm — 1 Comment

Developing a Risk Tolerance Study for FDA

We, the Duchenne community, have entered into a new era where more trials are coming online and the possibility of treatments becoming a reality. PPMD remains focused on working with the FDA to inform them about Duchenne so they can better understand the complexities of the disease and its patient population.

We have been working hard to create a survey about perceptions of benefits and risk tolerance of parents/guardians of individuals with Duchenne,…


Added by Holly Peay on November 21, 2012 at 10:37am — No Comments

Congratulations & Thank You

Congratulations to this year’s winner of the American Public Health Association Disability Section Lifetime Achievement Award, Dr. Gloria Krahn, the Director of the Division of Human Development and Disability at the Centers for Disease Control and Prevention’s (CDC’s) National Center on Birth Defects and Developmental Disabilities (NCBDDD). Dr. Krahn and her associates at…


Added by Holly Peay on November 15, 2012 at 12:20pm — No Comments

ImagingDMD: Making muscle biopsies a thing of the past

In the clinical trial world, there are some things that divide us these days- most specifically, what mutation type your son has. However, we are united in wanting better ways to look for the benefits of potential drugs; who isn’t hoping for a day when the muscle biopsy is a relic from the past? 

There are several dedicated research teams doing exciting work…


Added by Holly Peay on October 13, 2012 at 10:00am — No Comments

Promising News from Sarepta on Exon 51 Skipping Trial

The much-anticipated 48-week results on eteplirsen, a drug designed to skip exon 51, were presented today by Sarepta Therapeutics on a webinar and press release. On the webinar, Sarepta’s Chief Executive Chris Garabedian presented data about the dystrophin found in study participants’ muscle biopsies and the…


Added by Holly Peay on October 3, 2012 at 4:00pm — 5 Comments

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