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:10/17/2019)... (PRWEB) , ... October 17, 2019 , ... One of the most prominent features on ... are overly large or they stick out too far from the skull. This can cause ... for a way to alter and reduce the appearance of their ears. In order to ...
(Date:10/15/2019)... ... 14, 2019 , ... An Alabama jury has returned a $9 million verdict ... breaks records for Lee County. , In December of 2014, Hope Johnson visited Auburn ... there just two days earlier with similar, but milder complaints. On that first visit, ...
(Date:10/11/2019)... (PRWEB) , ... October 11, 2019 , ... ... Week starting October 13, 2019, to recognize the dedication, compassion and quality patient ... a collaborative process, providing a unique and vital link between patients, providers and ...
Breaking Medicine Technology:
(Date:10/15/2019)... ... October 15, 2019 , ... In 2019, Unitek opened its doors ... in late 2018 . Students who started in July were only entering their ... if these students would have to start over. Then the leadership team at Unitek ...
(Date:10/11/2019)... ... October 11, 2019 , ... Only 6% of orthopedic surgeons in the country ... as a female physician leading the sports medicine program. These four work with a ... succeed in traditionally male-oriented fields. , Orthopedic medicine deals with the correction ...
(Date:10/10/2019)... ... , ... AltMed Florida , one of the fastest growing Medical Marijuana ... opening of its second MÜV™ Medical Cannabis Dispensary on the First Coast – scheduled ... Beach. , Located at Beach Plaza Shopping Center at the corner of Beach Boulevard ...
(Date:10/10/2019)... ... October 10, 2019 , ... In attendance at ... excited to hear the significant results of an independent study presented by researchers ... The clinical study poster entitled “The Acute Effects of Citrulline Malate and Bonded ...
(Date:10/8/2019)... ... ... SoCal Adolescent Wellness announces the opening of a second location in Lake ... better serve the families in Orange County," said Becca Moody, CEO of SoCal Adolescent ... this pre-intervention program has on the lives of the adolescents and families we work ...
Breaking Medicine News(10 mins):