Navigation Links
Parent Project Muscular Dystrophy and Expert Advisory Committee Releases Putting Patients First
Date:4/23/2013

HACKENSACK, N.J., April 23, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) has released Putting Patients First, a white paper outlining recommendations to speed responsible  access to new therapies for Duchenne muscular dystrophy (Duchenne) and other, rare, serious, and life-threatening neurological disorders. PPMD developed this white paper with an expert Advisory Committee on Policies to Promote Responsible Access to New Therapies, made up of leading authorities from the nonprofit, academic, medical, legal, and pharmaceutical sectors.

Putting Patients First urges the U.S. Food and Drug Administration (FDA) to work with PPMD and the rare disease community to take advantage of opportunities created by the 2012 passage of the Food and Drug Administration Safety and Innovation Act to enhance patient access to new therapies for serious and life-threatening disorders.  In implementing the law, PPMD and the Advisory Committee encourage the FDA to "strike a more appropriate balance between clinical certainty and patient access to potentially life-saving treatments." The white paper makes four recommendations:

  • Expand the use of accelerated approval for therapies intended to treat rare diseases, including Duchenne muscular dystrophy.
  • Issue clear guidance outlining the level of evidence required for the use of surrogate endpoints in order to expand the scope of acceptable endpoints, including novel surrogate and intermediate clinical endpoints, used to approve drugs for serious or life-threatening diseases with unmet medical need.
  • Pilot the use of adaptive approval for serious and life-threatening disorders with significant unmet medical need, using existing authority under current law.
  • Give greater weight to the demonstrated benefit/risk preferences of patients, as well as caregivers in the case of pediatric illness, when making risk benefit determinations.

Pat Furlong , Founding President and CEO of PPMD, hopes that Putting Patients First will illustrate the need for the FDA to accelerate approval of potentially life-saving drugs intended to treat rare disorders, like Duchenne.

Says Furlong, "We have been working with the FDA and other regulatory agencies for the last decade to educate them on Duchenne muscular dystrophy and the catastrophic affect this disorder has on both patients and families. As our relationship continues to grow and build, and with the implementation of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA), we are hopeful that the FDA will work with PPMD and the rare disease community to take advantage of the new opportunities created by this landmark legislation."

PPMD is disseminating Putting Patients First throughout the various regulatory agencies, Congressional offices, and to stakeholders in the Duchenne community.

If you would like to read Putting Patients First and to learn more about PPMD's advocacy efforts, please visit ParentProjectMD.org/puttingpatientsfirst.

About the Advisory Committee on Policies to Promote Responsible Access to New Therapies
The Advisory Committee includes a diverse and distinguished group of leading authorities from the nonprofit, academic, medical, legal, and pharmaceutical sectors. Members include Richard Finkel , MD, Chief, Division of Neurology, Nemours Children's Hospital; Marlene E. Haffner , MD, MPH, CEO of Haffner Associates, LLC; Emil Kakkis, MD, PhD, President, EveryLife Foundation for Rare Diseases; Laurie Letvak , MD, Senior Vice President, Novartis Pharmaceuticals Corporation; H. Lee Sweeney , PhD, Chairman of Physiology, University of Pennsylvania; and Susan L. Weiner , PhD, Founder and Board President, Children's Cause for Cancer Advocacy.

About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our  mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.


'/>"/>
SOURCE Parent Project Muscular Dystrophy
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. 40 percent of parents give young kids cough/cold medicine that they shouldnt
2. Pharmagen to Present Strategic Vision for Solving Nations Parenteral Drug Shortage Crisis at 2013 BIO International Convention
3. AAIPharma Appoints Senior Director of Parenteral Operations
4. Parents numb to misuse of narcotic pain meds by youth, new poll shows
5. CSL Behring Parent Company, CSL Limited, Issues Fourth Corporate Responsibility Report
6. MSA Helps Bring Infant CPR Training to New Parents in Butler
7. ALERT: Parents ... Learn What Your Teens Are Up to Now
8. U.S. Senate Resolution Urges Communities, Parents to Fight Teen Medicine Abuse
9. Webcams Help Parents Keep Watch Over Their Babies During Hurricane Isaac
10. TUV Rheinland Confirms Apparent Fraud in Mislabeling of DRX Products Manufactured by HTRD and Excite Medical
11. National Call-to-Action to Boost Meningitis Vaccination Rates Supported by Leading Health Organizations, Urges Minneapolis-St. Paul Parents to Vaccinate Preteens and Teens
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/25/2016)... , May 25, 2016 According ... by Type (3D, 2D, 4D), by Therapeutic Area (Oncology, ... End User (Medical Device Manufacturers, Hospitals/ Clinics) - Forecast ... global Medical Animation Market for the forecast period of ... USD 301.3 Million by 2021 from USD 117.3 Million ...
(Date:5/25/2016)... Germany and GERMANTOWN, Maryland ... (NASDAQ: QGEN ; Frankfurt Prime Standard: QIA) today ... co-development agreement with Therawis Diagnostics GmbH to develop and commercialize ... develop and market PITX2 as a marker to predict effectiveness ... cancer patients. "We are pleased to partner with ...
(Date:5/25/2016)... 2016  According to Kalorama Information, the world ... 2015.  Though these are challenging times in the ... success for companies that remain optimistic and seek ... growth prospects medical device companies spend a higher ... (R&D) than do companies in other industries.  Also, ...
Breaking Medicine Technology:
(Date:5/27/2016)... ... May 27, 2016 , ... An influential resource amongst nurses ... time to shed lights on the variety of topics detailing why we appreciate nurses ... tackles why this career has gone from being in a major recession to one ...
(Date:5/27/2016)... New York, NY (PRWEB) , ... May 27, 2016 , ... ... the factors of a stroke, which we as a society can control and change. ... a stroke occurs nearly every 40 seconds within the United States. Plus, with an ...
(Date:5/27/2016)... New York, NY (PRWEB) , ... May 27, ... ... has partnered with Mediaplanet to help educate the many who are unaware of ... section dedicated to aphasia will run within the “Stroke Awareness” campaign. , The ...
(Date:5/27/2016)... ... May 27, 2016 , ... ... is bolstered by inspiring human interest stories, courtesy of leaders in the nursing ... tech within the industry, from leading advocates and associations—namely Jones & Bartlett Learning. ...
(Date:5/27/2016)... ... May 27, 2016 , ... ... to students studying complementary medicine. Allison Outerbridge is this year’s Life University ... on May 18 at the university’s Student Leadership Awards ceremony. , Outerbridge is ...
Breaking Medicine News(10 mins):