The Pink Sheet Reports:
Gaining the expertise and infrastructure needed to assess products of the future - such as biosimilars and those based on stem cells and nanotechnology - is the goal of a $25 million scientific modernization effort proposed in the fiscal 2011 FDA budget.
The Advancing Regulatory Science Initiative (ARSI) is a cross-center program whose ultimate objective is to provide FDA scientists with the skills to evaluate the safety, effectiveness and quality of products developed with new and emerging technologies, as well as give industry the standards by which such products will be judged.
The agency bills the initiative as the first major effort to address a 2007 FDA Science Board report that cited FDA's inability to keep up with scientific advances, such as those in biology and bioinformatics.
The Center for Drug Evaluation and Research plans to "establish regulatory guidance to provide a scientifically sound and safe pathway to better characterize and develop biosimilars," the agency says in supporting documentation for its budget.
ARSI, which FDA characterizes as a new program and an increase in budget authority, provides $2 million for the biosimilar effort. Another $3.7 million in carry-over balances from user fees will be used for the program.
As part of a $15.55 million increase for infrastructure, workforce and collaboration, ARSI includes money to prepare for reviewing products that utilize stem cells and nanotechnology.
With the removal in 2009 of the ban on federal funding for embryonic stem cell research, FDA "anticipates a significant increase in investigational new drug applications and biologics license applications for stem cell products during the next few years," the agency says.
In anticipation of that, ARSI gives the Center for Biologics Evaluation and Research $950,000 and five full-time equivalents to prepare for reviewing stem cell products (i.e., a therapy comprised of, or derived from, human cells, tissues or cellular or tissue-based products, and gene therapy products) as novel biologic therapies.
The FY '11 funds will allow CBER to develop and communicate guidance that provides agency expectations for pre-clinical studies, clinical trial design and regulatory policy. CBER also will work with the scientific community to develop analytic tests for characterizing stem cell products.
The largest chunk of ARSI is $7.3 million for nanotechnology regulatory science across the agency. CBER and CDER each will receive $475,000 and one FTE to participate in the effort.
The intent is to build laboratory testing capacity, develop and train staff in nanotechnology and conduct research into the character and safety of products. The latter will help FDA scientists as they review the benefits and risks of products, such as drugs delivered by nanoparticles to cancer cells.
Included in ARSI is a $4.45 million increase in funding for the Critical Path Initiative. The money will enable FDA to launch new projects for validating biomarkers, modernize and increase the efficiency of clinical trials; improve tools to predict product safety and effectiveness, including the use of information technology; and modernize toxicology studies.
FDA's documents do not provide a baseline figure for CPI, but includes it under the Office of the Chief Scientist (OCS), which is budgeted at a total of $29.59 million. Funding for CPI in the current fiscal year is $16 million.
The money also will enable OCS to staff the Office of Science and Innovation, which is being set up to serve as a core resource of scientific expertise in emerging areas. OSI was created in a reorganization implemented by Commissioner Margaret Hamburg in mid-2009 to improve the agency's scientific capabilities.
