Legislation

• The Prescription Drug User Fee Act of 2007 (PDUFA).  The Prescription Drug User Fee Act of 2007 (PDUFA) was signed into law on September 27, 2007.

FDA / Prescription Drug User Fee Act (PDUFA) Fact Sheet

FDA Prescription Drug User Fees web page

Analysis of The Prescription Drug User Fee Amendments of 2007 by Parkinson's Action Network

     Prescription Drug User Fee Act Public Meeting (Feb. 2007) : Statements by Parkinson Pipeline members:

Perry Cohen : "A Patient's Perspective on Key Policy Issues for User Fees and Drug Safety Legislation"

Tony Decamp

Senate and House Approve Compromise Bill That Would Reauthorize Prescription Drug User Fee Act (PDUFA) and Improve Drug Safety Monitoring (9/21/07)

PDUFA Fact Sheet

Overview

• The Prescription Drug User Fee Act, or as it is commonly called, PDUFA, is the first of a series of laws that allow the agency to help fund the review of new drugs through fees paid by the companies that submit new drug applications. PDUFA was first enacted in 1992, and has been reauthorized twice, each time for five years, in 1997 and 2002.

• On September 30, the current PDUFA program will end. FDA has been working for over a year, in consultation with its stakeholders, to develop a new proposal for PDUFA reauthorization.

• The Agency has conducted meetings and consultations with all of FDA's stakeholders including Congress, industry, patient advocates and organizations representing health care professionals and consumers. The next iteration of the program will be known as PDUFA IV.

Proposed Recommendations

• To achieve the necessary public health benefits, the agency is seeking to increase total annual user fees to $392.8 million,^[1] an $87.4 million annual increase over the current base to:

  • Significantly broaden and upgrade FDA's postmarket safety system

  • Increase FDA's capacity to conduct advisory reviews of direct-to-consumer drug television advertising

  • Bring safe and effective new medications to the American public in a timely manner

Increases to Base User Fees

• $17.7 million to adjust the base amount for inflation and increases in salaries and benefits

• $11.7 million to cover a share of increased rents and the costs of the agency's required move to the new White Oak facility in Silver Spring, Maryland

• $20 million in additional fees to cover significant increases in FDA's drug review workload that were incurred but not compensated for under PDUFA III, and are expected to continue

Program Enhancements

• $29.3 million annually to be allocated to enhance the Agency's drug safety capabilities.

  • Increase in the number of FDA employees dedicated to safety evaluation of marketed medications.

  • Added resources to adopt new scientific approaches to drug safety, improve the utility of existing tools for the detection and prevention of adverse events, and incorporate them into our drug safety program

• $4.6 million annually to expedite and improve new drug development.

  • This includes development of new guidance to clarify requirements for new clinical trial designs, and work to clarify regulatory pathways in the areas of predictive toxicology, biomarker qualification and the handling of missing clinical trial data.

• $4 million annually to improve the information technology infrastructure for human drug review

  • This investment will help the agency to progress toward an all-electronic review environment

  • An all-electronic system would make our evaluation process more efficient, ease the identification of data inconsistencies and improve our use of clinical data submitted as part of new drug application to help monitor the overall safe and effective use of products once they are on the market.

• Establishment of a separate program to collect new fees from companies that seek FDA advisory reviews of their direct-to-consumer television commercials.

  • FDA anticipates that these fees will reach approximately $6.25 million a year and support 27 additional staff to carry out this function, and provide more timely reviews.

PDUFA Reauthorization Process

• Following today's posting of the PDUFA IV notice describing the proposals, FDA will present the proposals at a public meeting at the Grand Hyatt in downtown Washington DC. The meeting will be on February 16. Comments from that meeting and Federal Register notice will be evaluated and incorporated as appropriate in the final proposals that are submitted to Congress.

• PDUFA IV will go into effect only if it is enacted by Congress and signed by the President.

PDUFA History

• In 1992, Congress passed the Prescription Drug User Fee Act. The drugs user fee program was reauthorized by the Food and Drug Administration Modernization Act of 1997 and again by the Public Health Security and Bioterrorism Preparedness and Response Act of 2002.

• PDUFA authorized FDA to collect fees from companies that produce certain human drug and biological products. To market a new drug or biologic, a company must submit an application along with a fee. In addition, companies are assessed annual fees for each prescription drug product marketed and for each establishment that manufactures a product assessed a fee. Previously, taxpayers alone paid for product reviews through budgets provided by Congress. Under PDUFA, industry provides funding that is added to FDA's appropriated budget, and FDA commits to certain performance goals, which emphasize review timeliness as well as other performance measures.

PAST PDUFA GOALS

• PDUFA I

  • Review and act on a progressively increasing proportion of original NDAs, BLAs, and efficacy supplements within 12 months and resubmissions and manufacturing supplements within 6 months.

  • Review and act on 90 percent of priority NDAs, BLAs, and efficacy supplements (submissions that are for products providing significant therapeutic gains) submitted in FY 1997 within 6 months.

• PDFUA II

  • Review times were shortened and FDA met or exceeded nearly all its review goals

  • Expanded the scope of PDUFA work by including new goals intended to improve communication between FDA and application sponsors during the drug development process.

• PDUFA III

  • Review performance goals and the procedural and processing goals were largely the same as the PDUFAII FY 2002 performance levels for these goals.

  • Established several new initiatives to improve application submissions and FDA-sponsor interactions during drug development and application review.

  • Authorized FDA to spend user fee funds on certain aspects of postmarket risk management, including surveillance of products approved after October 1, 2002, for up to 3 years.

[1] All amounts will be adjusted annually for inflation and to reflect workload increases.

Source: FDA / PDUFA Fact Sheet

Abigail Alliance: Access, Compassion, Care, and Ethics for Seriously Ill Patients Act

The Parkinson Pipeline Project is supporting the Abigail Alliance for Better Access to Developmental Drugs in its initiative to make promising experimental treatments more readily available early in the drug development process. This applies to patients with life-threatening or chronically disabling diseases, who have exhausted all approved treatment options, including clinical trials, and compassionate and expanded use programs.

Aligned with the Pipeline Project’s underlying philosophy, the “Access, Compassion, Care, and Ethics for Seriously Ill Patients Act” seeks to:

• "Empower patients to choose their own treatment course by fully informing them of the risks and benefits of experimental medicines and ensuring they get counsel from their doctor."

A patient facing certain death is different from that of a patient with a non-terminal condition or remaining approved treatment options,” said Frank Burroughs, president of the Abigail Alliance. “When the risk of taking a less-well proven drug that might help an individual is weighed against the reality of certain death, allowing access to that drug is not only reasonable, it is their only chance.”

• Institute a “Tier 1” status for experimental treatments based on positive clinical evaluations of safety and efficacy as early as in Phase 1 testing. This supports the Pipeline Project’s view that a more effective approval process would stretch out the longer term study of new medicines into several smaller steps rather than the current single major step of “approval.”

“The need for certainty in drug approval is killing people,” Burroughs said. "Excessive caution is delaying the availability of potentially helpful treatments; slowing the absorption of new knowledge and diagnostic tools into medical practice; and discouraging the pursuit of vaccines and next-generation antibiotics that could save tens of millions of lives.

This “Deadly Caution” is the subject of an in-depth analysis of the role of FDA in making risk and benefit tradeoffs in development of the “miracles of scientific medicine” by award winning senior editor of Fortune, Clifton Leif.

Role of placebo in clinical trials

One provision in the ACCESS Act we would like to see softened calls for a ban on placebo-controlled trials in patients with life-threatening illnesses, challenging the ethics and efficacy of placebo-controlled studies as the “gold standard” for approval of new therapies. The Pipeline Project would ask the Abigail Alliance to refine the language to recognize the value as well as the limitations of placebo-controlled trials. We know that placebos are not always the best control group, and in the case of PD, the mechanism of the placebo effect (thought to result from release of dopamine in the brain as a result of expected benefits from the treatment), is directly confounded with the disease. When it comes to sham surgery, the placebo is a far cry from doing nothing.