Mike Lehmicke, Director of Science and Industry Affairs at the Alliance for Regenerative Medicine, discusses the alliance’s key focus areas, as well as topics such as the effects of the COVID-19 pandemic
The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organisation dedicated to realising the promise of regenerative medicines and advanced therapies. As the global voice of the sector, ARM represents the interests of 370 members across 25 countries, including small and large companies, academic research institutions, major medical centres and patient groups.
ARM promotes legislative, regulatory, and reimbursement initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies, and tissue-based therapies. Early products to market have demonstrated profound, durable, and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options.
Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative medicines and advanced therapies. In its 11-year history, ARM has become the global voice of the sector, including small and large companies, academic research institutions, major medical centres and patient groups.
The Innovation Platform’s Managing Editor, Michelle Gordon, spoke to ARM’s Director of Science and Industry Affairs, Mike Lehmicke, about ARM’s key focus areas, as well as topics such as the effects of the COVID-19 pandemic.
2020 has been a challenging year. How has the regenerative medicine sector fared and what impact has COVID-19 had in terms of investment, progression of clinical trials, etc.?
Investment into the sector in 2020, despite the pandemic, has been extraordinarily strong. Global financing for the regenerative medicine and advanced therapy sector attracted $15.9bn in financing in the first three quarters of the year, shattering the previous record of $13.5bn, set during 2018. There are now 1,026 therapeutic developers active in this space worldwide (135 of which are in tissue engineering/biomaterials).
Through September of this year, there were 1,109 ongoing clinical trials in regenerative medicine worldwide; of those, 16 are related to COVID-19 and complications. Initially, many clinical trials were affected by the pandemic, with sites pausing new patient enrolment. This has now been largely resolved, although there may be some longer-term effects due to follow-up issues (i.e. missing data). Manufacturing fared well after some initial obstacles related to supply chain disruptions. It remains to be seen whether there will be any knock-on effects as vaccine manufacturing scales up, which will consume a lot of CDMO (contract development and manufacturing organisation) capacity.
What have been the key areas of focus for the Alliance for Regenerative Medicine (ARM) during this year?
In 2020, ARM has been focusing on addressing market access and regulatory hurdles to ensure that the sector’s transformative therapies can reach patients in need. Notably, as a result of efforts by ARM, its members, and other partners, the US Centers for Medicare & Medicaid Services finalised a rule establishing a dedicated Diagnosis Related Group for CAR-T therapies which will help ensure that providers are appropriately and sustainably reimbursed for administering these therapies.
ARM’s focus in 2020 has included removing barriers to innovative payment models in both the US and Europe so that developers, for example, can work with payors on agreements to ensure reimbursement for therapies over time if those therapies meet clinical milestones. ARM has also called on the European Commission to fast-track ‘real-world evidence’ (RWE) for advanced therapy medicinal products (ATMPs) before rolling it out in the broader pharma and biotech sectors. RWE can facilitate access for patients by providing important data prior to approval, and then by providing a robust follow-up mechanism to determine efficacy.
ARM has also focused on making sure the sector can continue to thrive despite the COVID-19 pandemic. In fact, on the positive side, COVID-19 may provide lessons for regulators and developers that can be harnessed to advance the regenerative medicine sector. For example, while it is too early to know for sure, the surge in manufacturing to produce vaccines using viral vectors may ultimately benefit the manufacturing of viral vectors to be employed in gene therapies.
According to your Advancing innovation during COVID-19 report, several academic research centres and therapeutic developers are investigating the application of regenerative medicine technologies to treat COVID-19. Can you tell me more about this? Can these technologies be used to repair lung damage caused by the virus, for example?
As of the end of September, there were 16 clinical trials worldwide using regenerative medicines and advanced therapies to treat COVID-19. These include therapies that directly target the virus (e.g. virus-specific T-cells), and therapies that treat complications associated with COVID-19 – mainly Acute Respiratory Distress Syndrome (ARDS). We are not aware of any ongoing clinical trials using regenerative medicine therapies to repair lung damage specifically caused by COVID-19.
The National Institutes of Health published a report stating that some people who develop severe COVID-19 carry a genetic mutation that impacts immunity. Could gene therapy/editing alter the body’s response to COVID-19 and reduce the risk of death?
If a genetic mutation in fact predicts a poor response to COVID-19, it may be possible to use genetic screening for diagnostic purposes to more effectively guide the use of therapeutics in these patients. The cited study examined the effect of multiple genetic mutations, so it is difficult to say at this point whether a gene therapy could effectively be used as a treatment.
As we look ahead to 2021, what will be the main challenges/opportunities facing the regenerative medicine sector and what role will ARM play in supporting the sector?
As the global voice of the sector, ARM will continue to advocate for addressing market access and regulatory barriers in the US and Europe to ensure that our members’ transformative therapies can reach patients. This will include the adoption of innovative payment models and real-world evidence.
In an introductory ‘Letter’ to ARM’s Advancing innovation during COVID-19 report, Janet Lambert, ARM’s Chief Executive Officer, said:
“Despite unprecedented global challenges, the regenerative medicine and advanced therapy sector demonstrated remarkable resilience in the first half of 2020. Patients continued to benefit from the cell and gene therapies currently on the market and in trials. Investment in the sector was robust, and we made important gains in improving market access policy. Clinical progress also continued, albeit with new obstacles caused by the pandemic and its effects.
“On the clinical front, several academic research centres and therapeutic developers are investigating the application of regenerative medicine technologies to treat COVID-19 in the short term and address related complications in the future. In addition, therapeutic developers continue to advance clinical programmes in a wide variety of indications with unmet medical needs not related to COVID-19. Notably, over the past few months, we’ve seen initial clinical efficacy data for pioneering gene-editing technologies and longer-term durability data for approved and late-stage gene therapies.”
Lambert explained that there has also been “disruptions to the development of non-COVID-19 related regenerative medicines. Many global regenerative medicine companies reported challenges in clinical trial execution, including patient recruitment, enrolment, data collection and/or follow-up. In addition, given that managing and reacting to the ongoing public health crisis caused by COVID-19 has become the priority for many policymakers, regulators, and public sector payors, we have seen new demands for regulators that could result in delays for cell, gene, and tissue-based therapy product candidates outside of the COVID-19 space. Both developers and regulators recognise that interruptions can have devastating consequences for patients who rely on timely access to treatments, and regulators have shown a willingness to be flexible while retaining a high standard for health and safety.”
With these challenges in mind, Lambert concluded by stating that ARM will remain “committed to doing everything we can to ensure that patients will be able to access these profound, durable and potentially curative therapies.”
Mike Lehmicke
Director of Science and Industry Affairs
Alliance for Regenerative Medicine
info@alliancerm.org
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