Navigation Links
Patient Advocates Ask FDA to Let Them Help Define Risk
Date:10/5/2011

WASHINGTON, Oct. 5, 2011 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) and about 30 of its advocacy partners have sent letters to key officials at the Food and Drug Administration (FDA) seeking more frequent and regular opportunities to provide input on decisions related to relative risks and potential benefit for new drugs and medical devices.

(Logo: http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b)

The letters are a follow-up to meetings the patient advocates had with senior FDA officials over the past few months. They include specific proposals for enhanced communication between the patient community and FDA to ensure that the voices of patients with chronic and rare diseases are heard in risk-benefit determinations and related policy decisions.

"Patients need to have opportunities to communicate with FDA medical reviewers on the risks they are willing to run in exchange for a potential though unproven benefit," said Peter L. Saltonstall, president and CEO of NORD. "For example, a patient with a serious disease and no approved therapy may have a perception of risk that is very different from that of someone who has treatment alternatives. We believe that FDA can make more informed decisions about investigational products and about which products to approve if they hear directly from patients."

The letters were sent to Dr. Theresa Mullin, Director of FDA's Department of Planning & Informatics, and Dr. Jeffrey Shuren, Director of FDA's Center for Devices and Radiological Health (CDRH). Copies were sent to Health and Human Services Secretary Kathleen Sebelius, FDA Commissioner Dr. Margaret Hamburg, and Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research (CDER).

The patient advocates note that, when they met with the FDA officials in April and May of this year, the officials asked them to follow up with a proposal outlining specifics for more frequent and regular communication between the patient community and FDA on benefit-risk determinations. While mechanisms currently exist for patient input on this topic, they add, the opportunity to provide such input doesn't necessarily occur at the time that critical issues are being deliberated and may not represent a broad spectrum of patient views.

"We have had discussions at length within the patient community as to what the most appropriate milestones should be for patient contribution," the letters note. They go on to say that the rare disease patient advocates have concluded that the two milestones should be:

1) When the IND (Investigational New Drug Application) or IDE (Investigational Device Exemption) is filed and,

2) Any point at which the agency (FDA) decides, based on issues related to risk, that it must disapprove or delay commencement of a clinical trial or impose an unscheduled suspension on an existing clinical trial.

In addition to laying out specific suggestions as to how and when patient input might be submitted, the letters propose that FDA create a portal on its website, www.fda.gov, where it can post specific requests for patient contributions to certain issues.

"The patient groups who have signed this letter represent a broad spectrum of diseases, some rare and some more common. But all patients with chronic or rare diseases know that the benefit-risk ratio for any new drug or device is an important consideration," said Diane Dorman, NORD's vice president of public policy. "We are pleased that the patient community has been able to band together to bring these proposals to the FDA, and that senior FDA officials have been so receptive."

NORD is an umbrella organization providing advocacy on behalf of the 30 million Americans with rare diseases, working with its member organizations, which represent patients with specific rare diseases, and other advocacy partners.

The letters and the list of patient organizations signing them are posted on the NORD website.


'/>"/>
SOURCE National Organization for Rare Disorder (NORD)
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. NxStage Says Thank You to Patients and Care Partners at the 2nd Annual NxStageUsers Meet-Up and Conference in New Orleans
2. Journal of Clinical Oncology Publishes Phase 2 Clinical Trial Results of KRX-0401 (Perifosine) Plus Capecitabine in Patients with Metastatic Colorectal Cancer
3. Halozyme Begins Randomized, Controlled Clinical Trial with PEGPH20 in Patients with Advanced Pancreatic Cancer
4. Incisive Surgical Launches New National Patient Advocacy Campaign -- Eliminate Traumatic Metal Skin Staples for Routine Skin Closure
5. voiceTech Launches ServiceLink-Rx, the First Comprehensive Patient Communication Platform for Retail Pharmacies
6. NxStage System One Patients Join Patient Advocacy Groups on Capitol Hill
7. Circadian Files IND With FDA for VGX-100 for the Treatment of Cancer Patients With Solid Tumors
8. First Prostate Cancer Registry for Robotic Radiosurgery Patients Enrolls More than 500 Men
9. FDA Participates in Global Efforts to Protect Consumers and Patients from Unsafe Drugs on the Internet
10. Visionsense Corp Announces 500 Patients Milestone With Neurosurgical 3D Camera at Emory University Hospital in Atlanta, GA
11. Study by Ocala Oncology/The US Oncology Network Shows Use of Bevacizumab Beyond Progression in Metastatic Colorectal Cancer Patients Improves Overall Survival
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/24/2016)... CAMBRIDGE, Mass. , June 24, 2016 /PRNewswire/ ... the Spaulding Rehabilitation Network,s Dean Center for ... of Physical Medicine and Rehabilitation, MIT Hacking Medicine, ... Center for Innovation, today announced the five finalists ... Hackathon for Lyme disease.  More than 100 scientists, ...
(Date:6/24/2016)... Dehaier Medical Systems Ltd. (NASDAQ: DHRM ) ... medical devices and wearable sleep respiratory products in ... with Hongyuan Supply Chain Management Co., Ltd. (hereinafter referred ... to develop Dehaier,s new Internet medical technology business. ... Hongyuan Supply Chain,s sales platform to reach Dehaier,s dealers ...
(Date:6/24/2016)... , June 24, 2016   Pulmatrix, Inc ., ... developing innovative inhaled drugs, announced today that it was ... Investments reconstituted its comprehensive set of U.S. and ... "This is an important milestone for Pulmatrix," said Chief ... shareholder awareness of our progress in developing drugs for ...
Breaking Medicine Technology:
(Date:6/26/2016)... ... 2016 , ... Brent Kasmer, a legally blind and certified personal trainer is helping to develop ... The fitness app plans to fix the two major problems leading the fitness industry today:, ... all type program , They don’t eliminate all the reasons people quit their ...
(Date:6/25/2016)... Austin, TX (PRWEB) , ... June 25, 2016 , ... ... Fellow of the American College of Mohs Surgery and to Dr. Russell Peckham for ... popular and highly effective treatment for skin cancer. The selective fellowship in Mohs Micrographic ...
(Date:6/25/2016)... TX (PRWEB) , ... June 25, 2016 , ... ... the United States, named Dr. Sesan Ogunleye, as the Medical Director of its new ... the facility Medical Director of our new Mesquite location,” said Dr. James M. Muzzarelli, ...
(Date:6/25/2016)... ... June 25, 2016 , ... On Friday, June 10, Van Mitchell, ... at Work award to iHire in recognition of their exemplary accomplishments in worksite health ... annual Maryland Workplace Health & Wellness Symposium at the BWI Marriott in Linthicum Heights. ...
(Date:6/24/2016)... ... June 24, 2016 , ... Those who have experienced traumatic events may ... to unhealthy avenues, such as drug or alcohol abuse, as a coping mechanism. To ... for healthy coping following a traumatic event. , Trauma sufferers tend to feel a ...
Breaking Medicine News(10 mins):