All Blog Posts Tagged 'pdufa' (16)

Help Protect Federal Spending on Rare Diseases like Duchenne

PPMD Taking Action to Avert Sequestration

Those engaged in federal advocacy probably thought the Prescription Drug User Fee Act and its acronym PDUFA would take the cake in terms of policy jargon. But as the nation's eyes are fixed on…


Added by Ryan Fischer on November 19, 2012 at 4:57pm — 2 Comments

FDA’s Patient Focused Drug Development Initiative – Request for Public Comment

PPMD and other groups submit joint statement making the case for neuromuscular disorders affecting children

As many advocates know from earlier blog postings, PPMD has been deeply and passionately engaged to enhance how the Food and Drug Administration (FDA) goes about its business of reviewing potential therapies and treatments for patients with Duchenne. Our work is focusing on ensuring that the agency moves as expeditiously as possible and…


Added by Ryan Fischer on November 15, 2012 at 11:00am — No Comments

Representing Duchenne at the Congressional Rare Disease Caucus Briefing

Today, Parent Advocate and Founder of Two Smiles One Hope Foundation, Alison Willis and I represented the Duchenne community at the Congressional Rare Disease Caucus Briefing in Washington, DC. Alison did a remarkable job speaking on behalf of PPMD regarding the Patient Participation in Medical Product Discussions Provision within the newly passed…


Added by Ryan Fischer on November 14, 2012 at 3:00pm — No Comments

Partnering with the FDA to accelerate approvals- The question of risk tolerance in the Duchenne community


Friends, we have been actively pursuing a regulatory strategy and have had two meetings with the neurology division at the FDA. We are currently working on the 'to do' list for the community and are trying to understand barriers/obstacles to approval from the FDA side, as well as, from the sponsor side of the issue. We are working a smart, targeted call to action. We have learned through years…


Added by Pat Furlong on October 23, 2012 at 11:30pm — 6 Comments

Implementing PDUFA

With Congress decamping from Washington and not expected to return until after the November election, we thought we'd revisit one of the few bright spots of bipartisanship this Congress – the enactment of the latest FDA user fee law (PDUFA), known as the FDA Safety and Innovation Act or FDASIA. (Washington loves a good acronym).



Added by Ryan Fischer on September 26, 2012 at 10:00am — No Comments

Landmark FDA legislation becomes law

Major win for the Duchenne community

PPMD applauds the President for signing into law the Prescription Drug User Fee Act also known as The Food and Drug Administration Safety and Innovation Act. Most recently, PPMD worked with the Everylife Foundation for Rare Diseases and Genetic Alliance to organize a letter to…


Added by Ryan Fischer on July 9, 2012 at 6:00pm — No Comments

PPMD Leads Critical Letter for Rare Disease Community

Parent Project Muscular Dystrophy (PPMD) has teamed up with EveryLife Foundation for Rare Diseases and Genetic Alliance to galvanize the rare disease community and send a strong message to…


Added by Ryan Fischer on June 13, 2012 at 3:00pm — No Comments

PDUFA Sails Through House of Representatives

Following recent overwhelming passage in the Senate, The Prescription Drug User Fee Act comes closer to becoming law after swift passage in the House of their version of the bill last night by a vote of 387-5. Both bills now move to conference for reconciliation.…


Added by Ryan Fischer on May 31, 2012 at 10:30pm — No Comments

PPMD endorses Senate version of Prescription Drug User Fee Act (PDUFA) after patient voice strengthened

As the U.S. Senate continues debate on legislation that seeks to accelerate delivery of safe and effective therapies to patients and to increase the patient voice as part of the therapy review process, PPMD is pleased to offer our enthusiastic endorsement of the legislation.


Over the past several months, PPMD has fought to include several key provisions included in the Senate version of the Food and Drug Administration Safety and Innovation Act, commonly referred to as the…


Added by PPMD on May 24, 2012 at 3:00pm — 1 Comment

PDUFA Moves Closer to Passage

Few pieces of legislation may be as peculiar – and as important to the development of drugs and devices in this country – as the package of bills known as Food and Drug Administration (FDA) Prescription Drug User Fees Acts, known as PDUFA, or user fees for short. Born out of a frustration – amplified by the lack of therapies to respond to the AIDS epidemic – that FDA was taking too long to review drug applications, Congress enacted the first such law 20 years ago, in 1992. The law set out…


Added by Ryan Fischer on May 17, 2012 at 10:28am — No Comments

Tis the Season, for FDA legislation called PDUFA

The Senate and House are moving closer to taking action on critical legislation focused on the Food and Drug Administration called PDUFA. Every five years Congress must pass a package of policies, known as user fees, that dictate the terms for the pharmaceutical and biotechnology and related industries to help fund the FDA and its review of candidate therapies. The bills…


Added by Ryan Fischer on April 24, 2012 at 10:30am — No Comments

Take Action: Ask Your Representative to Co-Sponsor FAST Act

We are coming to you today to ask that you devote your attention and energy towards legislation that seeks to expedite the process FDA uses to review drug applications.…


Added by Ryan Fischer on March 30, 2012 at 9:30am — No Comments

PPMD Announces Board Approved FDA Policy

As you know, ensuring appropriate federal funding for Duchenne research has been a cornerstone of PPMD’s advocacy agenda over the past decade. Our persistence – your persistent and collective voice - has paid off with over $250 million directed to Duchenne research.


Today we now find ourselves at a pivotal moment with multiple potential therapies for Duchenne in various stages of the development process. We must now, once again, look forward to what is next for Duchenne.…


Added by PPMD on February 21, 2012 at 10:00am — No Comments


I don’t know about you, but when I am in DC or receive government related information, I am always about 3 acronyms behind in the conversation.  PDUFA – this is one acronym you want to put in your memory bank for 2012.  PDUFA – Prescription Drug User Fee Act.


This legislation is all about FDA, about what biotech’s/pharmaceuticals have to pay for the process, about rare disorders and incentives to develop drugs for rare conditions, and about the process of…


Added by Pat Furlong on February 20, 2012 at 10:30am — 5 Comments

Advocacy: Get Active!

With deficit reduction remaining a top priority in Congress and a $1.2 trillion spending reduction on the horizon, 2012 will be a critical year to protect Duchenne research and public health priorities. For this to happen, we need your voice on Capitol Hill now more than ever before. Whether that be by joining us in Washington at the Annual Advocacy Conference or advocating from home by contacting…


Added by Ryan Fischer on January 5, 2012 at 2:00pm — No Comments

An Amazing September Starts a Fall of Promise

Dear Friends,

As we say goodbye to September and hello to autumn, I wanted to take a moment and thank you.

PPMD and the entire Duchenne community are busier than ever. There is so much to do, to push forward, to get done, in our daily lives and in the care of our sons. Every now and then, though, it is important to take a minute to exhale and realize what you’ve done, what’s been accomplished, and what is happening around you.



Added by Pat Furlong on October 5, 2011 at 9:00pm — No Comments

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