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Food and Drug Administration
Learn about Drug Safety Legislation (PDUFA)
Learn about Follow-on Biologics
The Parkinson's Action Network applauds the recent enactment of the Food and Drug Administration (FDA) Amendments Act of 2007, which will improve patient access to safe and effective therapies in a timely manner. PAN has worked to include vital provisions in this bill that will enhance Critical Path drug development research, provide patients with valuable information about clinical trials and their results, protect patient safety, and maintain speedy review times.
On September 19, 2007, the House of Representatives passed the bill by a vote of 405 to 7 and the Senate passed the bill unanimously the following day. The President signed the bill into law on Thursday, September 27, 2007. PAN commends our congressional champions for drafting and passing this strong, comprehensive legislation.
The Food and Drug Administration (FDA) Amendments Act of 2007 also authorizes the collection of industry user fees that enable FDA to review drug applications in a timely manner. Created in 1992, the Prescription Drug User Fee Act (PDUFA) must be reauthorized every five years. These industry user fees are tied to FDA performance goals that aim to reduce review times for drugs and biological medications while increasing scientific consultations, clarifying issues involving drug development, and increasing oversight of post market safety.
This is the fourth time Congress has considered must-pass PDUFA legislation, but it is the first time that patient advocacy organizations have actively participated in the process. PAN, in conjunction with the Alliance for Drug Safety and Access (ADSA), successfully worked to ensure that the bill includes critical provisions that will hasten discovery and access to better therapies for people living with Parkinson's and other diseases.
The following provisions are particularly important for the Parkinson's community:
Creation of the Reagan-Udall Foundation
This legislation creates the Reagan-Udall Foundation for the FDA, which is a public-private partnership charged with advancing the FDA's Critical Path Initiative. The Critical Path Initiative aims to modernize drug development and accelerate discovery of breakthrough, novel therapies by funding research into areas such as biomarkers, animal models, inter-individual variability in drug response, data analysis technology and methodology for use in drug development, and improvements in designing and conducting clinical trials. PAN worked with both the House and Senate to ensure that the Critical Path Initiative's drug development priorities were included in this legislation.
Clinical Trial Databases
The bill establishes a clinical trials registry and a clinical trials results database, which will be publicly available through the Internet. The clinical trials registry will include information on all ongoing Phase II, Phase III, and Phase IV trials. In practical terms, this registry will not be that different from what we already have at clinicaltrials.gov. However, the database will, for the first time, require that after the approval of a drug, results from that drugs earlier trials must be added to the database. This information will include summaries of FDA committee meetings, advisories, assessments, and relevant journal articles and publications. PAN strongly supports the accessibility of this important information.
The bill also requires the Department of Health and Human Services to determine how to include lay-language, unbiased results summaries in the database and if it is feasible to add clinical trial results information for unapproved drugs in the future. Lay-language summaries of trial results would enhance patient assess to and understanding of trial results information. By making results for unapproved drugs public, researchers would have access, for the first time, to critical data on drugs that were deemed safe in Phase I testing but were shelved for other reasons. This could potentially shorten the drug development timeline, and hasten access to future therapies.
Enhancement of Post-Market Surveillance
The bill will also establish a surveillance system to better identify potential adverse effects of drugs that are already on the market. This will be done through enhanced interactions between the FDA and databases maintained by the Department of Veterans Affairs, large insurance provider groups, Medicare programs, and similar large databases. Scientists will routinely analyze data on millions of patients, looking for signals that drugs may pose serious risks. This type of active surveillance will help the FDA continue to gain needed information on products as they are being used by a large, diverse population and provide data that is virtually impossible to collect through pre-market trials.
Coalition
PAN has worked in coalition on this issue with other patient advocate and medical organizations. PAN is a member of the Alliance for Drug Safety and Access (ADSA) whose goals are to ensure the safety of drugs approved by FDA while making sure access to innovative therapies is not slowed through the development and approval pipeline. Read more about ADSA.
Support and Analysis
Read PAN's endorsement letter of the Senate Bill.
Read PAN's endorsement of House legislation.
Read PAN's letter to the Energy and Commerce Committee supporting the passage of drug safety legislation in the House Committee.
Other Activities
On Friday, February 16, 2007, Amy Comstock participated in an FDA public meeting for Prescription Drug User Fee Act. Read the archived “what’s new” article from February 2007: PAN Urges Increased Patient Voice to Enhance Clinical Drug Development.
On May 9, 2007, the Alliance for Drug Safety and Access (PAN’s coalition on drug development issues) testified at a House of Representatives Energy and Commerce Health Subcommittee hearing on “Assessing the Safety of our Nation’s Drug Supply.” Read Diane E. Thompson’s testimony on behalf of the coalition.
PAN sent a letter to Senators Kennedy and Enzi in support of The Biologics Price Competition and Innovation Act of 2007 (S. 1695), a bill that would allow for a regulatory pathway for safe, effective, affordable, and comparable (generic) versions of biologic drugs. This Act provides for an approval pathway for safe generic and interchangeable biological products while preserving the incentives that spur the development of these life-saving medicines. The complementary bill in the House is the Access to Lifesaving Medicine Act of 2007 (H.R. 1038).
Read PAN's endorsement letter of the Senate bill.
Generic drugs have been extremely successful in bringing down the high cost of prescription drugs. Generic drugs save patients and taxpayers $10 billion a year. But currently there is no generic competition for one of the fastest growing and most expensive category of drugs known as biological drugs or biopharmaceuticals. Biotech drugs, which are produced from living cell cultures rather than synthesized chemically, promise a new generation of life-altering treatments, but often at considerable cost. Generic versions of biologic drugs go by several names: biosimilars, biogenerics, and most accurately follow-on biologics. We refer to them as follow-on biologics since they “follow on” after a brand product’s FDA approval.
Generic drugs that are currently available are synthetically exact copies of the brand name original, based on a precise chemical composition. Follow-on biologics, on the other hand, would need to allow for nuances in cell cultures while meeting certain parameters that are strict enough to ensure they are just as safe and effective as the originals. Technologies to ensure biologic “copies” are not currently available.
The Biologics Price Competition and Innovation Act of 2007 establishes a scientifically rigorous process for approval of follow-on biologics, authorizing FDA to determine, on a product-by-product basis, what studies would be necessary to show that a new product is clinically comparable to the brand name product. Comparable biologics would be approved on the basis of the safety and effectiveness of the brand name product, together with such additional evidence as FDA scientists determine is necessary to show that there are no clinically meaningful differences between the two products (i.e., that the two products will produce the same effects in patients). The bill also establishes a period of data exclusivity for first-time drug innovators. This period of data exclusivity is important to ensure companies are encouraged to pursue innovative therapies.
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