Dr. Gil Van Bokkelen
Chairman, Alliance for Regenerative Medicine
There is intense public interest in the medical potential of regenerative medicine, and in my opinion for very good reason. Those of us that are actively engaged in and/or closely follow what is going on in the field can see the transformative potential, and the steady progress being made. This is a field that could impact many areas of medicine, and help address critical areas of unmet clinical need. It could produce new and better treatments for heart disease, vascular disease, stroke, progressive neurological conditions like Parkinson’s, Multiple Sclerosis, ALS, and acute conditions like trauma and spinal cord injury. It could offer more effective solutions or even cures for a broad range of inflammatory and autoimmune diseases, and provide meaningful advances for a range of other chronic, orphan, and aging related conditions that traditional medicine has no real answer for, and that represent a huge quality of life and economic burden on society. In fact, there are already multiple forms of cell therapy available to physicians and patients, having been approved by the FDA and other regulatory agencies, and used successfully for a number of years. These therapies are providing remarkable benefits to patients suffering from diabetic ulcers, chronic wounds, orthopedic injury, and a range of other conditions.
Cell therapy, tissue engineering and the field of regenerative medicine isn’t just interesting medical research and cool science – it’s way more important than that. As the President’s Council on Science and Technology recently concluded, we are facing a huge problem in the years ahead. Our national healthcare bill is going WAY up, thanks to an aging population. We can either ignore the problem, which will inevitably lead to healthcare rationing, or we can innovate our way out of the box we are in. But for innovation to have an impact, we need to do things NOW that will enable us to address the challenges we face in the very near future and well beyond. Thankfully, there are some things we can do. In fact, I believe it should be a national imperative to develop new forms of medical treatment that effectively address defined areas of need – not just because it will help patients, but also because it will make healthcare more cost effective while broadening access.
There’s no denying we live in a very challenging time financially. With the aging of the baby boomer generation we face an unprecedented demographic shift that, according to government estimates, threatens to cause Medicare to go bankrupt within a few years, could lead to widespread healthcare rationing, and could saddle us and our children with unfathomable debt for decades to come. The debate in Washington is largely centered on how to control expenses (and implicit in that is limiting access or treatment options). However, the simple act of controlling how much we spend does nothing to address fundamental limitations in quality of care and outcomes, and I would argue is tantamount to looking through the wrong end of the telescope.
Improving care and reducing costs are mutually attainable goals. But the way to achieve those goals and simultaneously improve care, enhance patient (and family) quality of life, reduce overall healthcare and related costs, and expand access is through innovation. Specifically, we can achieve these goals through the development of new and disruptive technologies that have the potential to offer more than an incremental advance, but rather can help achieve a transformational shift.
In many ways the field of regenerative medicine embodies the most powerful kind of medical innovation. Many people intuitively recognize that it is a field that possesses the potential to deliver what we need. In biological terms, the promise of regenerative medicine is that it could enable us to repair, regenerate or replace damaged or diseased tissue, helping to heal patients in areas where current treatment options are largely or completely inadequate. It includes an array of potential approaches – stem cells and other forms of cell therapy, tissue engineering and devices, as well as small molecules and non-cellular biologics that can promote regeneration and healing. However, since it is so innovative, it also entails a steep learning curve, a big investment, and a lot of risk. Is it worth it? Just imagine the impact of success in even a few disease areas – what if we could effectively treat or even prevent congestive heart failure, where today the unavoidable outcome is death, even after significant and costly intervention that can stretch on for years? What would it mean to be able to treat serious vascular disease, where today the common result is amputation? What if we could treat or even cure patients that have suffered a stroke, or help those with Parkinson’s, Multiple Sclerosis, or other autoimmune diseases that are currently untreatable? What if we could limit the damage from trauma, or provide meaningful benefits to patients that have suffered spinal cord injury? What if we could more effectively treat aging related visual disorders or blindness? There are so many examples where we can have an impact.
The practical and economic consequences of success in even a few of these areas are enormous. An effective form of treatment could substantially reduce the impact of chronic disease, by reducing or eliminating long term intervention, the need for hospitalization or other institutional care costs, and all while dramatically improving patient (and family) quality of life and productivity.
To patients, their families, and to anyone that understands the ramifications of the ticking time bomb that our healthcare situation represents, time is of the essence. So what can we do?
Thankfully, as a result of the relentless determination and leadership of a growing number of people and organizations, many of whom are active members of the Alliance, there is already a lot going on. But we need to do more – as a nation. I believe the time has come for us to pull together and DEMAND that our political leadership develop a national strategy for regenerative medicine AND implement a national initiative for regenerative medicine technology. We can no longer afford to accept complacency and inaction. We need to raise our voices and be heard. This is an area that deserves the strategic investment necessary to solve a much bigger healthcare problem – and ineffective leadership, complacency in Congress and doing nothing is NOT an acceptable solution. Earlier this year, the White House and the Office of Science and Technology Policy unveiled a new “National Bioeconomy Blueprint”, which was viewed by many as a promising step in the right direction. However, regenerative medicine was barely even mentioned in the blueprint, and stem cells were not mentioned at all. Furthermore, to date neither presidential candidate has really discussed a concrete plan for how to use innovation as a way of addressing our fundamental healthcare challenges and the corresponding financial challenges we face as a nation.
But what specifically, should we do? As part of a national strategy and initiative to address our fundamental challenges, I believe that there are seven specific things we can do in order to have an impact. In my view these should represent the central components of a national strategy:
Conducting a strategic assessment of our current activities to achieve better coordination of federal activity – In order to effectively map out where we want to go, we first need to take a hard look at where we are currently at. This should start with a thorough strategic assessment of the current activities occurring at various federal agencies, so that we can invest limited resources more effectively. Furthermore, given just how important the healthcare issue is, there needs to be a clear point of contact in the White House that is focused on promoting Biomedical Innovation, and that should be their ONLY focus. Acting as an interface between the FDA, NIH, CMS, other federal agencies, and the private sector will be a huge job, and it deserves someone that is truly empowered and singularly focused on solving bottlenecks and accelerating progress in a cost effective and rational way.
Creation of incentives that will promote private investment in the areas we need most – Funding for emerging companies that are doing the most innovative research is much harder to come by these days. Furthermore, with sequestration and tax reform looming, most investors don’t know which way to turn. By creating meaningful incentives that promote targeted investment in defined areas of significant unmet medical need, it could catalyze a meaningful investment in the areas we need most. This could accelerate the development of innovative and cost effective solutions, and also spur economic activity at a time when we really need it. Ultimately the biggest payoff would be for patients and their enhanced quality of life, but the economic impact on productivity and reducing our national healthcare bill could be enormous. It’s also worth recognizing other countries have recognized the tremendous clinical and commercial potential of cell therapy, regenerative medicine, and tissue engineering, and are moving to position themselves as leaders in this exciting new area of medicine.
Streamlining clinical development in areas of serious unmet need – While always ensuring the protection of patient safety, we need to accelerate and improve the efficiency of clinical trials, particularly in areas where there is a serious unmet medical need. Thankfully, we’ve already taken some important first steps in the right direction. Recent legislation (FDASIA and PDUFA 5) included important provisions designed to help speed clinical development in areas of serious medical need. This legislation, which was passed in a bipartisan manner by Congress, and signed by the President, helps establish a framework for an expanded “Accelerated Approval Pathway” and a “Breakthrough Therapies” category. While the precise operational framework around these designations needs to be defined, this display of bipartisanship cooperation shows that Washington may not be irrevocably broken. However, an important next step is to explicitly identify the areas that we want to prioritize solutions for, and then establish an operational framework that will allow us to implement these solutions in a meaningful way. FDA will be developing these programs over the next 12-18 months and we have the opportunity to collaborate in that endeavor.
Creating an explicitly defined list of serious unmet medical needs that represent our national priorities in healthcare – We know what the major areas of burden are in our healthcare system, and we know approximately how big an impact these conditions will have in the years and decades ahead. By defining a clear list of priorities, we can implement a series of other steps, as defined below, that will better enable us to develop more effective solutions. This will be an important step in the establishment of a new Accelerated Approval process noted above and will help us move towards the ultimate goal of improving clinical outcomes, enhancing patient quality of life and reducing overall costs.
Establishing a more efficient coverage and reimbursement framework that will operate in parallel with an accelerated clinical approval framework – If companies are successful at developing innovative solutions, and ultimately obtain regulatory approval through an accelerated pathway, the implementation of the new therapies will be substantially delayed if they don’t have a path to coverage and reimbursement. If we want to get the most from a more efficient regulatory framework, we need to take steps on the coverage and reimbursement side as well.
Enabling better clinical trial design by broadening access and enabling more effective utilization of historical data from CMS, NCHS, CDC and other relevant agencies – Much of the innovative translational research is occurring at smaller (i.e. biotech) companies. But small companies are at a disadvantage in terms of access to capital, and frequently they have to run smaller, less informative clinical studies due to significant resource constraints. One way to enable companies to design more powerful studies would be to enable them to access and analyze more data from the National Center for Health Statistics (NCHS), Centers for Medicare & Medicaid (CMS), The Centers for Disease Control, and perhaps other federal agencies so that clinical trials can be designed more efficiently and effectively. These taxpayer funded organizations can and should provide access to data without charge to innovative organizations that are committed to developing therapies designed to address serious unmet needs (i.e. our list of national healthcare priorities). This data could help with trial endpoint selection, understanding and modeling the frequency of clinically relevant events, establishing benchmarks, and also help in establishing sensible reimbursement for products that are ultimately approved. Obviously such analysis must be done in a manner that never compromises patient identity – but this type of analysis is already done, so those types of safeguards exist and can be applied.
Creation of a National Clinical Trial Network that is focused on accelerating development of solutions in high priority disease areas – By first establishing regional (e.g. statewide) Clinical Trial Networks, we can create a system that can be linked together to create (a) national Clinical Trial Network(s) that enable faster initiation of clinical trials through the use of centralized Institutional Review Boards and standardized contracting. Statewide CTN’s can be linked to create a vast national network that will expand patient access and participation, and that will broaden clinical participation in active studies.
These are high level descriptions of the types of specific things that I believe we need to be doing. In a series of blogs to come, I will lay out some specific components for each of these seven objectives, as well as lay out a strategy for how we can move this vision toward becoming a reality.