Meet the Alliance for Regenerative Medicine – the international advocate for gene therapy, cell therapy, & tissue engineering.
Regenerative medicine includes gene therapies, cell therapies, and tissue-engineered products intended to augment, repair, replace or regenerate organs, tissues, cells, genes, and metabolic processes in the body. Regenerative medicine aims to alter the current practice of medicine by treating the root causes of disease and disorders.
The regenerative medicine and advanced therapies sector is creating transformative, durable treatments and potential cures for some of humankind’s most devastating diseases (many currently untreatable via conventional treatments) through the use of ground-breaking scientific discoveries and technologies.
The Alliance for Regenerative Medicine (ARM) is an international community of small and large companies, non-profit research institutions, patient organisations, and other sector stakeholders dedicated to realising the promise of regenerative medicine for patients around the world.
SciTech Europa Quarterly speaks to Janet Lambert, CEO of ARM, about the importance of regenerative medicine and some of the current trends in the regenerative medicine industry.
Can you start by telling us about the work and role of the alliance for regenerative medicine (ARM)?
I have been the CEO of ARM for two years now. Prior to that I worked in the US Congress, and the US, NIH and for large and small life sciences companies; it’s been a pleasure to come here when there’s so much exciting science reaching patients.
ARM is about to celebrate its 10th anniversary. The organisation is an alliance of companies, research institutions and patient advocacy groups, all of which care about cell and gene therapy and tissue engineering. We work together with those member organisations to address issues that affect the sector as a whole. Right now, those key issues are reimbursement and market access for regenerative medicines.
We are also spending a good deal of time focused on some of the manufacturing challenges that are unique to this sector. We work primarily in the US and in Europe, with regulators, payers, and other stakeholders in those two regions.
What are some of the current trends in regenerative medicine? Where do you see some of the biggest developments in research?
One notable trend is the growing visibility and awareness of this sector; there’s a lot of activity in this space, including a lot of investments. We have had several product come to market over the last couple of years, and we have had an increasing number of clinical trials.
At ARM, we track and curate our own database of the regenerative medicine sector globally. We are tracking more than 900 companies around the world in this sector, and more than 1000 active clinical trials. We saw just in the second quarter of 2019 more than $2.5bn invested in regenerative medicine. Investors and large pharma companies who haven’t previously developed internal programmes in this sector, are now rather active in partnerships, development and acquisitions as the public start to come around to the idea that this is a really exciting sector of medicine and something that investors want to be a part of.
In terms of the conditions and diseases that these medicines are addressing, a lot of the early work has been in rare disease, monogenetic diseases, as well as in oncology. I think the other trend we are seeing is that we are starting to see more and more activity across a broader spectrum of indications. For example, there is activity in diseases of the eye, cardiovascular disease, a lot of interesting science going on in CNS. Therefore, there’s a breadth of indications that are being explored with these new therapies.
Alongside this however, challenges obviously come with this. When it comes to regenerative medicine development and research, what would you say are some of the biggest challenges? How are these overcome?
Right now, the biggest challenge is trying to fit a new kind of medicine into an old kind of reimbursement system. Most medicines are taken over a long period of time. Many of the most exciting new medicines in this field are likely to be one-time treatments. That is exciting for patients, of course; however, that means it’s a onetime charge to a health care system and in many cases (as we have found when those products have come to market), these products can be expensive.
Therefore, both public and private payers are trying to figure out, how is it that they can incorporate these kind of medicines into their health system? Can they pay for these medicines over time, even in public health systems that require a balanced budget? Can they adopt pay-for-performance systems where there is a rebate if a patient doesn’t respond to a given therapy or doesn’t respond as completely as hoped? I think there’s a lot of excitement about developers’ willingness to enter into the novel agreements. However, the fact is, developing the systems needed to accomodate these novel reimbursement schemes can be challenging for insurance companies and public healthcare systems.
Another challenge at the moment is manufacturing. Many of these early products are being developed for rare, or even ultra-rare, diseases. Therefore, the clinical trial populations are pretty limited and the amount of manufacturing that is required to conduct those trials, is likewise at a pretty small scale. As a result, the industry as a whole is figuring out how to go from small scale development of product to a larger scale development. That requires advances in viral vector manufacturing and cell line production, as well as in the development of related technologies that will ensure quality control and the ability to compare one batch of medicine to another.
Those are things that are not so much policy challenges as they are technical, evolutionary challenges, and to some extent logistical challenges. One of the things that ARM is doing is bringing the sector together to really identify what are the key roadblocks or missing links in manufacturing these therapies to scale, and how can we work together to expedite the solutions to those challenges.
Where do you hope to see regenerative medicine in five year’s time?
Both the FDA and the EMA have said that they anticipate there being 10 to 20 therapies in this category being approved every year by 2025. I think in five years we are going to see a whole bunch of gene therapies and tissue engineered products on the market in the US and Europe. Those will benefit patients of all sorts and across multiple indications. I think we’re going to see an improved regulatory and reimbursement process that evolves from what’s currently a kind of one by one negotiation for developers.
I think we’re going to see continued scientific advances that allow for improvements in manufacturing and that we will start to see more long term data around patients and confirming or understanding the durability of these products. Our anticipation, and the initial studies today suggest that this is true, is that these products with be quite durable, and we look forward to in five years’ time having more data around that. We expect that the number of clinical trials will continue to grow. We have a great number of clinical trials in the late stages now, and I think we’ll continue to see that pipeline grow over the next five years.
ARM has grown in the two years I’ve been here from a 260 member organisation to a 350 member organisation. We have had the distinct pleasure of working with more and more of the new players in the sector as well as those who have been along for the ride for the past 10 years or more. I think that ARM will continue to grow along with the sector.
I would imagine that we will see our global presence expands; there’s a lot of regenerative medicine work going on in Asia and ARM has yet to really expand its reach to Asia. Right now we are really focused on the US and Europe, and trying to get things right in those markets, which tend to be the initial markets where products first gain market authorisation, but we’re excited to grow in numbers and in global reach.
Alliance for Regenerative Medicine