It’s not often you see “FDA” and “innovation” in the same headline.
But with the publication of a recent report, FDA is trying to change that perception. FDA Commissioner Margaret A. Hamburg, MD recently released a blueprint titled “Driving Biomedical Innovation: Initiatives for Improving Products for Patients” at the 3rd annual Washington Ideas Forum.
The purpose of the blueprint is to address concerns about the sustainability of the medical product development pipeline. It describes actions being taken by FDA in 7 major areas, which are:
- Rebuilding FDA’s Small Business Outreach Services
- Building the Infrastructure and Drive to Support Personalized Medicine
- Expedited Drug Development Pathway
- Harnessing the Potential of Data Mining and Information Sharing
- The Future of Medical Devices
- Training the Next Generation of Innovators
- Improving FDA Regulations
The subject of biomedical innovation is of great interest to me, as is the content of this report. But unfortunately, I have not seen much discussion about the merits of the report content. To encourage discussion and make the contents more accessible, I’ve condensed 36 pages and described key points for your browsing enjoyment.
As you can see, each item in the list above is a link, which can be clicked to skip to the summary of that topic. These links allow you to quickly browse areas of particular interest. If you would like to delve deeper into the report, it’s available here.
Please leave your thoughts in the comments at the end of this post.
Though FDA has existing resources to aid small businesses, many small businesses fail to take advantage of these resources, most likely because they are unaware of them. The result is that small businesses continue to file applications that do not meet FDA’s requirements, and they are often not prepared to bring a drug to market.
Additionally, the perception among some is that FDA does not have a full appreciation for the hurdles faced by small businesses as they submit an application.
To address these issues, FDA is working on the following three programs.
Small Business Liason Program
FDA will establish a Small Business Liason (SBL) program composed of business people acting as liasons between FDA and small businesses. These business people will have experience “starting and running biomedical companies, obtaining venture capital funding, and successfully navigating the FDA regulatory process to bring a product to market.”
In addition to coordinating interaction with FDA and small businesses, SBLs will train FDA staff on how to work with small businesses more effectively, and they will provide staff with a better understanding of the small business perspective.
Young Entrepreneurs Program
Students with a promising entrepreneurial future will have access to 4-6 month internships in FDA small business offices, which will enhance their effectiveness as future business leaders.
Partnership with Small Business Administration
FDA is setting up a partnership with the Small Business Administration (SBA) to find better ways to support small businesses. Among the options being explored is an exchange program for FDA and SBA employees. This exchange would provide SBA with a better understanding of FDA requirements, and FDA would gain insight into the needs of small businesses.
The applied regulatory science for evaluating the strategies and outcomes for personalized medicine are mostly underdeveloped. Furthermore, the personalized medicine trend is creating additional challenges for FDA during the approval process, since an increasing number of products are within the purview of multiple FDA centers.
To meet these challenges, FDA will take scientific leadership and build the regulatory infrastructure necessary to support advances in personalized medicine.
Diagnostics and drugs will increasingly be “co-developed,” allowing the diagnostic to signal which patients will most benefit from the drug. This co-development approach will require increased investment in regulatory science. FDA called for investment in key regulatory science areas in two recent white papers, Advancing Regulatory Science for Public Health and Strategic Plan for Regulatory Science.
Novel clinical trial designs and improved identification of quality biomarkers are crucial. FDA will continue to invest in these scientific areas and expand its efforts via collaboration with other government agencies.
FDA is developing a series of regulatory policies to support the fruition of personalized medicine. To address the issue of companion diagnostics, FDA issued draft guidance titled In Vitro Companion Diagnostic Devices on July 12, 2011.
Draft guidance will also be issed “for co-developing a novel companion diagnostic and therapy simultaneously, where the approval and subsequent use of the therapy would incorporate a requirement for the use of the diagnostic test.” An internal plan for reviewing applications using co-development is also being developed.
FDA is holding public meetings with key stakeholders to develop evaluation tools that ensure genomic sequencing quality, which will allow for effective clinical application.
Deputy Commissioner for Medical Products
Coordination between CDRH, CBER, and CDER will be necessary for applications incorporating diagnostics as a requirement for therapy. A Deputy Commissioner for Medical Products has been appointed to oversee and promote integration among these three product development centers.
When new therapies show exceptional promise, it is challenging to balance the demands of scientific rigor with the ethical obligation to ensure people with serious diseases are getting the most promising therapy. To meet this challenge, FDA will hold meetings with key stakeholders to aid in developing draft guidance on an expedited development pathway.
FDA is also working on two immediate steps in this area. First, it’s developing draft guidance on enrichment strategies in clinical drug development, which will improve the efficiency of clinical trials and facilitate expedited development. Second, CDER will publish a working example of an expedited pathway in breast cancer as draft guidance.
The FDA houses the largest known repository of clinical data, and the ability to integrate and analyze these data could revolutionize development of new patient treatment.
Improved data leveraging capabilities would allow us to answer fundamental questions about how different patients respond to therapy, provide enhanced knowledge of disease parameters, and prompt earlier identification of ineffective products during the development process. Furthermore, the ability to share information publicly (while protecting proprietary data) would prevent duplication of failure, saving companies millions of dollars.
Scientific Computing and Scientific Enclaves at FDA
New technologies allow FDA to combine data from disparate sources, while improved computational methods allow complex data analysis.
“The FDA scientific computing model provides an environment where communities of scientists, known as enclaves, can come together to analyze large, integrated data sets and address important questions confronting clinical medicine.” Integration of data sets across multiple clinical trials, post-market surveillance data, and pre-clinical data will provide FDA with new insight into product development and use.
Modernizing the FDA IT Infrastructure to Support Scientific Computing
A major initiative called Information Computing Technologies for the Twenty-First Century (ICT21) was launched to modernize FDA’s aging IT infrastructure. The Data Center Consolidation strategy enables rapid migration to a Cloud First policy, which will maximize computing capacity and efficiency.
Building an Infrastructure for Patient-Centered Outcomes Research
FDA is developing tools for analysis of clinical study data cross multiple studies, which will form the core of the clinical data repository. This core will then be expanded to include other data sources, including pre-clinical pharmacology and toxicology data or post-market safety
surveillance clinical data.
In addition, FDA launched Partnerships in Comparative Effectiveness Science (PACES), which will support development of new mathematical methods for patient-centered outcomes research.
Opportunities Through Public Private Partnerships
A framework is being created to facilitate collaborative public-private scientific computing communities.
FDA recently announced plans to improve consistency and clarity in the medical device review process. After continued discussion with the medical device community, FDA will move forward with several new actions through the CDRH Medical Device Innovation Initiative.
- New Expedited Review Program – A new expedited review program called the Innovation Pathway will create a more streamlined path to market for important technologies.
- Understanding Emerging Technologies – CDRH will identify emerging technology trends by enhancing its horizon scanning methodology and developing a Network of Experts to serve as a resource.
- Increasing Consistency Through Improved Training – All new reviewers will be required to participate in a Reviewer Certification Program. In addition, FDA is developing an experiential learning program to provide staff with real-world training.
- Providing Additional Clarity to Industry – FDA is developing a series of new guidance to address important questions by industry.
FDA is designing a Future Innovators Program, which will provide candidates who hold outstanding promise in their field with practical regulatory science and policy training. During this short-term position with the FDA, candidates will receive hands-on training in multiple disciplines. This program provides candidates with highly marketable skills, while providing the FDA with outside expertise and perspectives.
With input from stakeholders, FDA is identifying burdensome, unclear, obsolete, ineffective, and inefficient regulations. Additionally, FDA is revising rules to spur innovation and improve access to care.
So what do you think of FDA’s blueprint? Can the actions proposed by FDA meaningfully spur innovation? Do you think this blueprint will be implemented in a manner that spurs innovation? Are there elements you think need to be added? Please share your thoughts in the comments below.
Image Credits: Driving Biomedical Innovation report