Since the birth of the biotechnology industry in the late 1970s, many transformational technologies and new treatments have been introduced, improving medical care and benefitting patients. And in many areas of medicine, exciting progress continues. However, there are many areas where standard of care is fundamentally limited, and significant challenges remain. So, it’s natural to ask, what advances will the next decade bring? How will the landscape evolve in light of key healthcare and technology trends?
One way existing challenges could be addressed is through the development of innovative technologies and treatments that could help restore health more effectively and efficiently, shorten hospital stays, lessen dependence on supportive care, and reduce the need for home healthcare or nonclinical institutional care facilities.
Many areas of clinical medicine could be transformed by advances in regenerative medicine, a field that includes cell therapy, gene therapy, gene modified cell therapy, and tissue engineering. Over the past few years, this field has started to come of age. Over the next decade, it will, many believe, achieve its long-anticipated potential and begin to fundamentally reshape clinical medicine as we know it.
Healthcare systems stressed by aging populations
Where is the field likely headed in the next few years? What are some of the key trends in healthcare that regenerative medicine technology might be helpful in addressing?
From a therapeutic standpoint, clinical trials evaluating regenerative medicine technologies and treatments have considered a broad range of disease indications and clinical challenges. These include cancer; neurological indications; cardiovascular disease; inflammatory and immune conditions; endocrine, metabolic, and genetic disorders; musculoskeletal and opthalmological conditions; and a host of others. The therapeutic indications under evaluation reveal the enormous scope regenerative medicine could achieve.
There are two major healthcare trends that these innovative technologies may help to address. One is the continued expansion of the elderly segment of the global population. The other is the corresponding impact on healthcare systems. By the year 2030, the entire population cohort of “baby boomers,” defined as those individuals born between 1945 and 1965, will all be 65 years of age or older.
According to the American Association of Medical Colleges (AAMC), between 2018 and 2033, the portion of the U.S. population under the age of 18 will increase by approximately 3.9%, while during the same period, the population 65 years of age or older will grow by more than 45%—to more than 77 million people. By 2034, the U.S. Census Bureau predicts that that for the first time in our nation’s history, people over the age of 65 will outnumber those under the age of 18.
This unprecedented demographic shift matters because it will have a profound impact on healthcare resource utilization here in the United States. (Similar shifts, and consequences, are expected elsewhere around the world.) As the population gets older and as we collectively become more susceptible to age-associated health problems, we face another serious challenge—a growing shortage of physicians and nurses. By 2033, the AAMC estimates, there will be a shortage of between 54,000 and 139,000 physicians across all specialties.
According to the American Nurses Association (ANA), there will be a national shortage of more than 100,000 registered nurses by 2022. The ANA also estimates that the number of nurses needed in the United States will grow from 2.8 million in 2018 to 3.6 million in 2030. The prospect of an expanding elderly population, the corresponding increase in demand for healthcare resources, and an escalating shortage of qualified physicians and nurses are discomforting to say the least.
According to the Alliance for Regenerative Medicine (ARM), currently there are approximately 1,000 companies around the globe that are developing regenerative medicine treatments and technologies, with most of them located here in the United States. Furthermore, there are now more than 1,000 clinical trials currently being run, with 587 of them in Phase II and 97 of them in Phase III. Many of these programs are focused on serious diseases and conditions that will have an increasing impact in an aging society, as well as other areas of unmet medical need.
Developments in regenerative medicine
Age-associated health problems that affect us individually and collectively may be alleviated by regenerative medicine. At present, the continued growth and evolution of regenerative medicine seems assured by five key trends.
1. The number of late-stage clinical trials will continue to rise, and the number of approved therapies will meaningfully increase.
Phase II trials are notorious for being the riskiest phase of development—with the greatest amount of attrition. With nearly 600 Phase II trials ongoing, we are likely to see both some exciting advances and some frustrating failures in the next few years.
The successes will beget additional capital investment, growth, and development—and the failures will demand critical reassessment. Some failures will be due to unexpected safety issues and/or a lack of efficacy, some prompting a discontinuation of programs, and others will be due to clinical trial design issues that might be addressed through more effective inclusion/exclusion criteria, or utilization of clinical endpoints that more appropriately evaluate and ultimately demonstrate therapeutic benefit.
Given the nearly 600 Phase II programs, there will likely be several hundred programs that advance to Phase III development. Given the approximately 100 Phase III programs now underway, we might expect dozens of these to be successful and clinically validated, and to obtain subsequent regulatory approval.
2. There will be a shift away from more expensive, highly personalized treatment approaches, toward more scalable and standardized “off the shelf” treatment approaches.
The advent of chimeric antigen receptor (CAR) T-cell technology to treat patients with refractory hematological malignancies is in many ways illustrative of both the challenges and opportunities facing the sector as a whole. Unquestionably, the technology represents treatment breakthroughs—but while demonstrating promising effectiveness, the initial approaches using gene-modified autologous cells face scalability challenges and are prohibitively expensive.
As a result, we already see dozens of companies and programs that are focused on next-generation approaches applying the same concepts using allogeneic platforms that could be administered “off the shelf,” and would be far more scalable and cost effective. Manufacturing scalability and consistency will improve—but only for those technology platforms that permit it.
3. Mergers and acquisitions in the sector will increase appreciably.
In the past five years, major biopharmaceutical companies have recognized the enormous potential of the regenerative medicine sector. These companies anticipate that the sector will have an impact in disease indications and clinical situations where there is unmet medical need due to limitations in standard of care, and where traditional pharmaceuticals or biotechnology products may not provide effective or durable relief. Large companies also have a relentless need to grow and rejuvenate their pipelines, and when promising approaches emerge, many of these will likely be acquired by larger established companies with strong channels to market.
4. Supply chain technologies and distribution capabilities will accommodate novel therapeutic modalities.
Traditional pharmaceutical supply chain and logistics practices are not well suited for advanced biologics—regenerative medicine technology generally requires specialized capabilities including cold chain or cryogenic product handing, storage, and distribution. Companies that solve these challenges will have stronger and more efficient channels to market. Those companies that wait for someone else to solve these issues will either sacrifice value or be commercially disadvantaged.
5. Coding, coverage, and reimbursement models will accommodate innovative regenerative medicine products and emphasize value.
Regenerative medicine treatment approaches have the potential to provide durable relief and, in some cases, even curative outcomes. However, if such approaches utilize highly personalized therapies or if they address small patient populations, reimbursement levels may have to be very high to justify the significant investments involved in developing such treatments.
Traditional coding, coverage, and payment systems are not generally designed to facilitate the rapid adoption or coverage of such approaches—even when they have the potential to transform medical care and outcomes for patients. But given the stakes, patients and their families will demand access to more effective treatments, and third-party payers will adapt to accommodate them. Payors and innovators will have to work together to holistically evaluate the value of these medicines, as well as to pay for them, to accommodate patient demand.
Limitations in standard of care and unmet clinical needs, as well as the corresponding desires for better outcomes and improved quality of life by patients and their families, will continue to drive medical innovation. Such advancements are possible only if investment fuels the innovation and sensible policies are implemented to help enable the efficient development of new medicines. There are serious challenges and obstacles ahead, but in many ways, the future of regenerative medicine couldn’t be brighter.
Gil Van Bokkelen, PhD, is chairman and CEO, of Athersys.
To help us preview the future, we asked opinion leaders, all from outstanding technology companies, to discuss a range of new initiatives. The full list of articles is below.
Leroy Hood: Reflections on a Legendary Career
Uncharted Territory: Top Challenges Facing Gene Therapy Development
Envisioning Future Trends in Regenerative Medicine
Engineering Biology—Accelerating Transition
Bioprocessing in a Post-COVID-19 World
Sustainability and the Synthetic Biology Revolution
Sowing the Seeds of Agricultural Biotechnology
Neuroscience Widens Its Investigations of Disease Mechanisms