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What is the "Critical Path" of medical product development?
The "Critical Path" is the scientific process through which a potential human drug, biological product, or medical device is transformed from a discovery or "proof of concept" into a medical product. It consists of scientific tests and tools used to predict whether a product candidate will be safe and effective, to assess how prototypes interact with the human body, and to guide the sponsor in choosing an appropriate dose and regimen or device size and/or placement. It consists of scientific tools to manufacture the product at commercial scale and assess the quality of the manufactured product. In order to bring a product to market successfully and efficiently, product sponsors need scientifically sound approaches to these challenges.
What is a Critical Path tool?
Critical path tools are scientific or technical methods used to:
Examples include:
What do you mean by "modernize" Critical Path tools? The report refers to "Opportunities," what does this mean?
Many of the tools used today to predict and evaluate product safety and efficacy, and to manufacture products, are badly outdated from a scientific perspective. We have not made a concerted effort to apply new scientific knowledge -- in areas such as gene expression, analytic methods, and bioinformatics -- to medical product development. There exists tremendous opportunities to create more effective tests and tools, if we focus on the hard work necessary to turn these innovations into reliable applied sciences.
Examples of Opportunities to use new science to modernize Critical Path tools include:
Why does FDA think the Critical Path infrastructure needs to be modernized?
Despite important investments in basic biomedical research, the number of applications to the FDA for new drugs and biologics has declined over the past decade. Of more concern, product development is not becoming more efficient over time – a drug entering Phase 1 trials in 2000 was not more likely to reach the market than one entering Phase 1 trials in 1985. And we are seeing more product candidate failures in the later stages of product development -- the most expensive way to fail. Recent biomedical research breakthroughs have not improved the ability to identify successful candidates and bring the most promising products to patients in a timely and affordable manner.
We can see a wide range of opportunities to improve the efficiency of product development. But without a concerted focus on the applied science necessary to develop these new approaches, the inefficiencies and unnecessary costs of product development will continue to escalate.
How will the Critical Path Initiative help bring new treatments to patients?
With tools that can predict which product candidates do not hold promise early in the development process, product sponsors can redirect resources to more promising products. Early failures are efficient failures – they free up resources that would otherwise be invested in product candidates that will eventually fail. New Critical Path tools will help product sponsors devote those resources to new and better candidates, thus facilitating the availability of more new medical products for patients.
In addition, modern evaluation tools will be more informative -- we will learn more about products before they are approved. This will give doctors and patients the best available information about how to use the product to maximize its benefit and minimize side effects. In fact many of the tools being considered would help individualize therapy, by identifying who is likely to responds well to a treatment, and who should avoid it.
The "Innovation / Stagnation" report refers to FDA guidances as Critical Path tools? Why is an FDA standard or guidance a Critical Path tool?
FDA guidances are science-based documents that eliminate uncertainty about what FDA will accept as valid evidence of product safety, product efficacy, and manufacturing quality. This information acts as a guide or "compass" that product sponsors can use to design their product development pathways to most efficiently meet FDA requirements.
FDA guidances have been shown to improve chances of product success and shorten time to market. For example, FDA adoption of CD4 cell counts and, subsequently, measures of viral load as surrogate markers for anti-HIV drug approvals allowed the rapid clinical development and approval of life-saving antiviral drugs, with time from first human use to market as short as 3.5 years. These standards greatly simplify effectiveness studies, thus reducing time and costs. Similarly, device guidances have been shown to reduce both product development time and the percent of applications found deficient on the first review cycle.
Why is the FDA the right entity to take responsibility for improving the Critical Path?
The FDA is not taking sole responsibility for improving the Critical Path. But it is the only entity capable of creating the focus necessary for this task. FDA is uniquely suited to serve as a hub for this effort, because it oversees evaluation of all U.S. medical products, and can identify the key product development hurdles that commonly cause setbacks for companies industry-wide. Because FDA is not a product development competitor, it can serve as a convenor for the coordination and information sharing necessary to identify and eliminate Critical Path hurdles. And because FDA sets the scientific standards for product development, only FDA can ensure that new Critical Path tools become the new standards for proving efficacy, assessing safety, and manufacturing medical products.
But industry, academic researchers, patient advocacy groups, and others are needed to do their part. Many of the Opportunities on the national Critical Path Challenges List will remain merely that -- unmet opportunities -- unless many people collaborate on the hard work of developing new tools for product development.
Will FDA collaborate with other public and private entities on Critical Path activities?
Yes. This is a complex task, requiring the expertise of all key stakeholders – academia, patient groups, government, industry, associations, and other private organizations.
Encourage other stakeholders to undertake work identified on the list. Some opportunities will require little or no FDA involvement, once the priority is identified. In these cases, FDA may play only a convening or communication role.
What is the FDA role in this collaboration?
FDA will play three roles. First, we will bring national focus to current product development issues, serving as a hub for problem identification and information exchange. Second, we will initiate projects and collaborations to help modernize the Critical Path. Third, we will encourage use of new Critical Path tools by accepting the results of the new tools as valid proof in product review (including up-dated science-based standards and guidances).
How does Critical Path research and science differ from investments in biomedical and translational research made by the Department of Health and Human Services, for example, through NIH research?
Critical Path science is the applied science partner of biomedical discovery science, and a productivity multiplier for our investments in basic and translational research.
What is the timeline for getting all of this accomplished?
We will publish the first National Critical Path Challenges List this fall. In late 2004 and early 2005, we hope to initiate some of the identified high priority projects that require FDA to play a key role. We hope the List will encourage others to undertake some this work, as well. We view this as a first step, and plan on working with stakeholders to determine how best the FDA can keep this effort going inside and outside the FDA.