A Meeting of Minds: Cell & Gene Conference Brings Tight-Knit Community Together
After two years stuck at home and only able to attend virtual conferences, the 2022 Cell & Gene Meeting on the Med was alive with excitement and positivity. The event, held from 20 to 22 April and organized by the Alliance for Regenerative Medicine (ARM), brought together a tight-knit community focused on one objective: to bring transformative medicines to patients in need.
Hundreds of biopharma companies were at the meeting, from top 20 biopharma companies to new drug developers. Discussions between colleagues and competitors were held on stage, allowing attendees to hear different views.
Janet Lambert, CEO of ARM, welcomed participants to the meeting with a very positive speech about the advances being made in cell and gene therapy, noting that to date there have been more than 2,000 clinical trials globally in the category. In terms of regenerative medicine approvals, six were approved in Europe, the US and China in 2021 and an additional therapy was approved in February 2022. In the field of CAR-T therapies, three were approved in 2021. In the US, the first regenerative medicine advanced therapy (RMAT) reached the market in early 2021 (Breyanzi, to treat blood cancers), with a second in June 2021 (Stratagraft, for burns) and Rethymic, for pediatric patients with congenital athymia, in October.
However, while innovation in Europe remains buoyant, the region is falling behind from a commercialization perspective when compared with the United States and even Asia Pacific. There are several issues affecting approvals. One is funding availability, which typically tends to be higher in the U.S., while another is the regulatory process. While the U.S. has a centralized way of handling clinical trial approvals, each member state in the EU has its own way of managing approvals. Although the Clinical Trials Regulation seeks to harmonize processes, currently many member states require additional documentation and translations, adding to complications. Separately, Europe is also looking at how to harmonize GMO regulations around clinical trial authorization. While these are performed at the national level, there are additional complications due to the fact that other agencies are assigned to assess GMOs for clinical trials, and each implement the GMO framework differently.
Access and reimbursement
One of the biggest topics of the meeting was the issue of market access and reimbursement, and the importance of bringing Health Technology Assessment (HTA) organizations into discussions much earlier. This is a hugely complex issue for cell and gene therapies, which are expensive treatments but potentially life changing. Importantly, therapies targeting genetic diseases could bring curative benefits to young patients, which is attractive for the healthcare system and could help to justify high price of the treatment.
Recently, the EMA and the European Network for HTA (EUnetHTA) consortium published a plan ahead of the implementation of new HTA rules that includes joint scientific consultations, real-world evidence and generating patient relevant data. There has been talk about centralized decision-making about pricing and reimbursement, though this seems unrealistic given each country has its own healthcare budget to manage and some would not be able to pay for these more costly treatments.
Other groups keenly focused on this topic are patient advocacy groups and many were in attendance at the meeting, pushing for developers and regulatory authorities to allow early access. These groups have long urged industry to engage with them early on, particularly for the development of therapies for rare and ultra-rare diseases.
Some discussions were around the critical points for developers of any drug, from clinical design and strategy to issues around comparative analysis for products that are completely novel and where it’s questionable whether the standard of care can be used as a control. In these cases, developers are pushing for increased use of historical controls through the use of real-world data and Bayesian statistics.
Another significant topic is the proposal to move cell and gene therapies to earlier lines of treatment rather than depending on the standard of care, particularly for diseases such as cancer. Healthcare systems have tended to go for established, traditional approaches based on current standard of care, which also is cheaper; however, there’s growing recognition of the need to move these therapies to earlier lines of treatment because they have proved their potential.
Overcoming hurdles
While cell and gene therapies have huge potential, there are also enormous hurdles. One in particular is in the move from R&D to manufacturing and the higher CMC failure rate in cell and gene therapy compared with other biologics. The difficulty is that many of these ideas begin at universities or hospitals, and the transition from R&D into clinical development represents a huge challenge for these institutions, since they are not equipped with the required expertise. Similarly, the progression from phase 1 to phase 3 trials would require clinical research capabilities that are difficult to manage within academic or clinical institutions.
There were also discussions about manufacturing processes. The complex and proprietary nature of cell and gene therapies mean many companies are eager to do their own manufacturing but this is both complex and costly. For many, the better option is to work with contract development and manufacturing organizations (CDMOs) but this does require consideration as to how best to solve potential questions related to the ownership of processes and cell lines. Nevertheless, there is growing demand for CDMO services, which is attracting new players to the field.
The cell and gene therapy sector is an exciting and rapidly evolving one, but it’s also a highly concentrated one and, while the pool of experts has grown over the past decade, the rapid growth of the field means there is always a need for specific expertise. The challenge here is that there are many products with unique features that require specific knowledge and often a customized approach, thus the pool of those able to contribute to the solutions is small, and even the regulators are learning on the go. Therefore, it’s crucial for innovator companies to have their finger on the pulse and be prepared to deal with complex, changing issues in real-time. Collaborative sessions such as the Meeting on the Med, discussion and interaction with the regulators, and finding the right advice from the small pool of experts will be key to bringing lifesaving therapies to market.
About the authors:
Emma Cheesman-Ungstrup is Director of Development Consulting and Scientific Affairs at Biopharma Excellence, Liron Sarid-Krebs is Team Lead at Biopharma Excellence, and Elena Meurer is Principal Consultant at Biopharma Excellence.
