Take a breath. Some news about the FDA and comparative effectiveness. Relax – it’s not what you think (or what the people at Consumer’s Union want).
By the end of September, the FDA will launch several initiatives to aggregate data on medical products, assess the information and eventually answer a myriad of questions on patient populations and class-wide issues, according to agency officials.
The new efforts will not affect product approvals and instead focus on simply answering a slew of outstanding questions by leveraging the abundance of collected data stored at the agency. And it’s about time. The FDA sits at the nexus of vast amount of untapped and highly important data. And while data is great, knowledge is power.
The American Recovery and Reinvestment Act (aka, “the stimulus package”), the FDA received funding (via AHRQ) to develop a CER infrastructure with outside assistance.
The agency's multi-step CER approach that includes creating the Janus data repository and developing at least one external center through the Partnership in Applied Comparative Effectiveness Science (PACES) initiative to examine the collected data, assess the information and make recommendations to answer questions on different therapies and patient groups. The data will include information from new product submissions and previously submitted product applications.
The Janus data repository will serve as the crux of the program and a "hub" that aggregates a substantial portion of collected agency information. One of the FDA officials described the data aggregation design as a "federated model" that will take advantage of the wealth of agency data collected for decades.
The data will come from standardized electronic product applications, previously submitted products, the Sentinel post-market analysis system, the MedWatch program, the common electronic document room, the automated laboratory management system and other data sources, officials said.
In parallel to the creation of the Janus data repository, the agency will also convert legacy data into a standardized format that can be inputted into the system. Agency officials acknowledged that the data retrofit will be costly, and the ARRA funding will focus on piloting the initiative to determine whether the program's benefits outweigh its costs.
The program will not aggregate data on unapproved drugs, and only focus on new submissions and retrofitting information from approved products that were previously introduced on the market.
It’s a smart move. The more information the agency has on both individual and class MOAs the better it can understand how things work in the real world (beyond the neatly designed, gold standard world of RCTs).
FDA science adviser Vicki Seyfert-Margolis said the program is not focused on directing regulatory actions, restricting randomized controlled clinical trials or limiting access to healthcare services.
Instead, she said the program will address the real world impact of therapies, help improve consistency and transparency in the approval process and identify healthcare gaps. For example, the examination of data could identify sub-populations that are not impacted by certain classes of drugs, with those patients potentially obtaining improved health outcomes from different therapies, such as certain devices.
Pulse check – this is not CER as part of the PDUFA process.
Janet Woodcock: "I think the science is still too early to be able to really design comparative trials that stand much chance of being conducted, at least pre-approval.”
According to FDA Week, “Agency sources said the Janus data repository and the PACES centers are not intended for use in making product-specific decisions in the premarket arena, and instead will simply enable the agency to understand the science behind certain classes of drugs, how they compare to other treatment options in patient subpopulations and assess the effect of genetics on therapies.” This is about personalized medicine [which is] a major new area of investment," an agency official stressed. "We're not using this to do cost assessments."
Personalized medicine? Well, yes – if you consider the use of outcomes data to be personalized medicine. And it is. It’s certainly an important first step towards realizing the “four rights” (right medicine in the right dose for the right patient at the right time).
The FDA will not release product-specific data from this initiative to the public, but the agency hopes to publish information on general CER methods and strategies developed through the PACES program. The agency could also (and should) release answers to questions on the impact of genetics on certain therapies and class-wide observations.
How does this relate to the Critical Path initiative?
FDA Chief Scientist Jesse Goodman: "In the long run for FDA and the sponsors, this will make everything more efficient. … Moving towards identifying what's the right way to do it does take some maturation of technology.”
Oh yes. And to that end the FDA will strive to implement “modern analytical tools” to examine the data. Easier said than done – but it’s money well spent.
