This article has been updated to clarify statements about FDA's VXDS program.
By Turna Ray
A recent survey of 50 life sciences companies found that the majority of drug, biologic, and diagnostic manufacturers feel the US Food and Drug Administration is not sufficiently working to advance personalized medicine.
The overall takeaway of the survey, conducted by PricewaterhouseCoopers and published last week, is that while the life sciences industry feels that its "communication with FDA has improved steadily since the passage of the FDA Modernization Act of 1997, … new expectations are fueling frustration with the regulatory review process."
The survey revealed that only 8 percent of drug and device makers agreed with the statement, "The FDA is doing enough to advance personalized medicine." Of the remaining respondents, 36 percent disagreed with the statement, and 56 percent were neutral.
Moreover, 56 percent of respondents familiar with FDA's Critical Path Initiative indicated that the agency lacks the capability to implement the effort which aims to bring innovative drugs to market. Historically, FDA's personalized medicine research efforts have often dovetailed with the C-Path Initiative. Only 2 percent of respondents disagreed with the statement, "The FDA lacks capability to implement Critical Path."
In addition, 56 percent of respondents said that the FDA should increase funding for the development of biomarkers that measure the progress of disease or the effects of treatment, "indication that the industry feels the agency's current commitment falls short," according to the survey.
Overall, among the companies PwC spoke to, many of which were smaller drug and device makers with less than 5,000 employees and under $500 million in annual revenues, 80 percent felt that the agency is providing better guidance about its expectations and 64 percent said they are incorporating FDA feedback in their product development.
This stands somewhat in contrast to the view of industry players developing personalized medicine products, a subset of the industry that the PwC report did not specifically target. Industry dissatisfaction is growing among personalized medicine stakeholders due to the lack of guidance from the agency on a number of regulatory issues involving laboratory-developed tests, companion diagnostics, and drug/diagnostic codevelopment (see related story).
According to Mike Mentesana, US pharmaceuticals and life sciences R&D advisory services leader at PwC and a co-author of the report, when it comes to emerging science, such as personalized or regenerative medicine, industry's frustration with the inability of FDA to keep up with the rapid pace of innovation may be due to the agency's resource limitations.
"The FDA's capacity is constrained, and many of the companies we talked to said they felt the agency had inadequate review resources," Mentesana told PGx Reporter.
In this regard, the PwC report highlights two developments in the last four years — the 2007 FDA reauthorization and the Prescription Drug User Fee Act — in order to account for the "less positive industry feedback about FDA." The FDA reauthorization expanded the agency's responsibility and PDUFA requires the FDA to collect user fees of upwards $1.25 million to review drug applications.
According to the survey, 46 percent of industry respondents do not find that increased user fees paid to FDA have accelerated the process, and around 48 percent of respondents felt that the agency has not been clear about how it applies these funds.
Certainly, when it comes to personalize medicine, more sponsors are meeting with the FDA to discuss their early genomics data through agency efforts such as the Voluntary Exploratory Data Submissions Program. Although over the years, non-regulatory submissions to VXDS have increased exponentially, the program is well staffed to handle these interactions, according to Issam Zineh, associate director for genomics at FDA's Office of Clinical Pharmacology.
However, once VXDS submissions translate to more regulatory submissions to bring new PGx drugs and devices to market, the agency will need more resources to review the deluge of genomics data that will come its way.
Despite the agency's resource challenges, the FDA has urged industry to meet early and often with the agency to plan ahead for regulatory submissions, as well as to have pre-submission discussions. In fact, at a recent meeting on personalized medicine, an FDA official discussing regulatory pathways for companion diagnostics development urged companies to take advantage of pre-Investigational Device Exemption submission meetings, but said that there might be some delays in the review process.
"Pre-IDEs are wonderful tools. They are free. They are a mechanism for getting feedback from CDRH," said Donna Roscoe, a scientific reviewer at the Office of In Vitro Diagnostics Device Evaluation and Safety at FDA's Center for Devices and Radiological Health, in September at the conference held in Arlington, Va. However, Roscoe acknowledged that although CDRH is supposed to provide feedback to sponsors' pre-IDE requests within two months, the timeline might be longer since the agency is "inundated" with pre-IDEs and is "a little behind," because "companion diagnostics has just exploded" (PGx Reporter 09/22/10).
According to the PwC survey, many drug firms have taken up FDA's advice to meet often and early. PwC's survey revealed that 64 percent of companies felt that meeting with FDA before submitting review materials "improved the quality of their applications," and 87 percent said such meetings sped up the review process.
"But industry did not always take advantage of the meetings," the report states, "and only about half (53 percent) said FDA consistently encouraged these meetings."
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