I applaud this Committee for initiating the 21st Century Cures Call to Action and its commitment to finding solutions that will ensure
This hearing is focused on a critical component of ensuring a forward-leaning and robust biopharmaceutical industry - what incentives are needed to advance treatment and cures? Before I discuss specific policies and offer ideas for consideration, it is important to understand that successful development of new medicines is dependent on policies that support the entire life science ecosystem - beginning with basic research and ending with providing treatments and therapies to patients. Disruption or weakening of policies that negatively impact any part of this ecosystem weakens the entire enterprise.
Assuming that a strong foundation of societal investment in basic research exits, then developing modern medicines from that point onward is a capital- and time-intensive endeavor taking an average of 10 years and
The U.S. Must Commit to Funding Discovery
A keystone to ensuring a robust life science industry is a national commitment to support basic research. Our nation's historical commitment to life sciences basic research is viewed as a precious jewel among nations. However, funding for the
Research dollars provided by the
Enabling Adoption of Modern Approaches to
This Committee has heard and received written testimony regarding the enormous increase in requirements and duration of clinical trials. n6, n7, n8, n9 These increases are especially acute for drugs designed to treat chronic diseases with larger patient populations. As a consequence, the cost of developing drugs has likewise been increasing. The Committee is right to ask whether these trends could be minimized by more effectively incorporating modern tools and approaches.
Venture funding is the life-blood of the small biotechnology companies working on disruptive science, and these venture-backed small biotechnology companies are the life-blood of innovative new medicines. In fact, a study published in 2010 found that in
Venture capitalists invested
The decision to deploy capital is directly impacted by regulatory decisions and behaviors. Better enabling and encouraging
Likewise, in recent years
In 2012, the Food and Drug Administration Safety and Innovation Act (FDASIA) created a new Breakthrough Therapy designation that will provide increased interactions with
The benefit of these programs has clearly been mostly realized in the oncology and rare disease space. As a society, while we celebrate these incredible successes, and indeed we should celebrate these successes, we have to ask ourselves what we want to do to improve how we treat the most egregious diseases affecting the greatest numbers our citizenry and long-term health costs, such as obesity, diabetes, and Alzheimer's, among others. As we examine the successes of these programs in terms of number of approvals for cancer and rare genetic diseases, we should endeavor to learn from the flexible and modern approaches utilized under these programs and work to apply them more broadly across therapeutic areas.
The fact is that while there are several examples where
When it comes to chronic diseases with varying stages of progression and severity, there seems to be an actual reticence to employ modern tools and approaches. Recent ideas such as Special Medical Use and
From early-stage life sciences venture investment perspective, we know that when we start a company with breakthrough innovations in new areas of science and medicine it will take a long time to turn that innovation into a drug that will reach patients and physicians and improve public health. The reality is the time required to put a drug on the market is, more often than not, longer than the length of our investment funds. Thus, when we create a new innovative company in a new area of science and medicine we are counting on the new medicine being developed being seen as important and valuable when it is still in the early stages of development. This is often referred to as the "proof of concept in the clinic," or Phase IIA. At that point, we are counting on the company and the product being sufficient to either take the company public on the NASDAQ or to have the company and/or product acquired by a pharmaceutical or larger biotech company.
The modern approach to regulation that exists now for cancer and rare genetic diseases allows this to work very well for three reasons. First, the regulatory process is more interactive, flexible, and reflective of the disease and patient being treated. Second, the amount, of time, and size of investment required to fund a company through 'proof of concept' is better understood. And, third, the next steps in our innovation ecosystem, larger companies and public investors, value the early-stage proof of concept data because they feel more confident about the development and approval process for these drugs. However, the same cannot be said for diseases such as obesity, diabetes, and Alzheimer's, where the time, amount of funds, and regulatory requirements are greater and there is less understanding about how to utilize modern tools and approaches. Without improving these processes, it is very difficult to imagine how early-stage investment can occur in such important areas.
In addition to the need for understanding the criteria which
Another, perhaps more critical, barrier to the advancement of diagnostic development is the fact that there are no consistent reimbursement policies for diagnostics.
I would like to recommend that the Committee consider a process whereby the
Utilization of Real-World Data: A Life-Cycle Approach to Drug Development
We currently have a system that requires a life-cycle approach to drug development with increasing abilities to monitor the safety and efficacy of drugs in the real world. However, we have not turned any of these new abilities to collect and share information into tools to advance drug development and improve how we treat patients. As we think about how we can accelerate drug development in chronic diseases such as diabetes, obesity, and Alzheimer's, and as we consider what is the evidence required for approval in such areas, we should keep in mind that real world data post-approval may be a very effective tool in understanding the scope of such drugs and may allow us to get to approval of these important new medicines more quickly. We should be actively working to integrate real-world data into the drug development and review process, and to achieve the right balance of what data we are requiring before and after approval. These approaches would help ensure patients have access to new medicines more expeditiously and could serve to support expanded indications in a more effective manner.
To ensure the promise of such real world data is realized, we must incorporate it as scientifically appropriate in the drug development process and make sure such data is available for use. As such, we must advance our healthcare system to one that has inter-operability capabilities that would enable more efficient use of electronic medical records with the real-world data required. We must also ensure that our systems have the ability to exchange such information in a privacy-protected manner. A balkanized set of such data would be missing an opportunity to support tremendous innovation in our health care ecosystem.
These are just a few incentives that could serve to advance how we develop medicines and treat patients. There are other critical policy areas that have the ability to impact or weaken the life science ecosystem not mentioned in this statement, but I would be happy to discuss these areas further with this Committee. For example, we must ensure that intellectual property is protected. There is no investment if the primary asset of an industry is not protected in a manner that allows for returns on investments. Data exclusivity of sufficient lengths of time can also be a powerful incentive, and we could consider aligning small molecule data exclusivity to the 12 years for biologic drugs, as the current 5 years of exclusivity for small molecules does not provide incentive from a venture capital perspective. We must work to ensure we encourage investment in small, innovative pre-revenue life science companies. n19 Lastly, we must ensure that reimbursement policies are determined in the context of the disease and patient being treated and the impact of a drug is evaluated over appropriate time lines. We must not create a system that will severely diminish investment in the next generation of cures and treatments. Thank you for the opportunity to share my thoughts and I would be happy to answer any questions.
n2 Copley, Caroline. With biotech hot on
n3 Adams CP and Bratner VV (2006) Spending on New Drug Development. Health Economics. 19, 13-141.
n5 Battelle Technology Partnership Practice. "
n6 Scannell JW, Blanckley A, Boldon H, and Warrington B (2012) Diagnosing the decline in pharmaceutical R&D efficiency. Nature Reviews: Drug Discovery 11, 191-200.
n7 Avik R (2012) The Stifling Cost of Lengthy Clinical Drug Trials.
n8 Tufts Center for the Study of Drug Development (
n9 Allison M (2012) Reinventing clinical trials. Nature Biotechnology 30 (1): 41-49
n10 Kneller, Robert. "The importance of new companies for drug discovery: origins of a decade of new drugs" Nature Reviews Drug Discovery 9, 867-882 (2010)
n13 JP Morgan. 2014 Global Biotech Outlook.
n14 FDA FY 2013 Innovative Drug Approvals. December, 2012
n16 Jarvis, Lisa M. Orphans Find a Home. C&EN Volume 91 Issue 19 | pp. 10-12.
n19 CSBI. http://www.smallbusinessinnovators.org/
Read this original document at: http://docs.house.gov/meetings/IF/IF14/20140611/102316/HHRG-113-IF14-Wstate-BorisyA-20140611.pdf
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