The cell and gene therapy field has made progress despite the COVID-19 pandemic, according to Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), which organized the three-day event.
"Six new regenerative medicines were approved globally in 2021, the second-best year on record," she told the audience in her opening speech. "(2021) was also the best year on record for CAR T-cell therapies, with three approvals. Just in the last few weeks, one of the earliest CAR T-cell therapies, [axicabtagene ciloleucel] Yescarta, was approved by the [U.S. Food and Drug Administration] as an early lymphoma treatment."
What's more, proof of concept that CRISPR gene editing can work when inserted in vivo was delivered for the first time in 2021, with additional promising data released earlier this year.
In all, 2,406 clinical trials involving cell, gene, and tissue-based therapy are currently underway worldwide. Half of those studies are being conducted by 1,300 corporate therapeutic developers, a number that has grown by nearly 20% over the past year alone.
In addition, 2022 could be a breakout year for the approval of new gene therapies to treat rare disease, with four such therapies up for approval across the U.S. and Europe, Lambert added.
"The promise of treating serious rare disease with transformative therapies has never been brighter," she said.
The cell and gene therapy sector is also making great strides on the path toward helping larger patient populations, with 52% of global clinical trials targeting cancer and nearly half of those trials targeting cancers such as gastrointestinal, skin, and breast cancer.
In addition, 60% of total clinical trials in the cell, gene, and tissue space are targeting prevalent diseases such as diabetes and related complications, as well as central nervous system (CNS) diseases including Parkinson's.
This scientific progress has been driven by a robust global investment environment, which has in turn fueled incredible scientific advances, Lambert explained.
"Investment in our sector has grown from nearly $10 billion in 2019 to nearly $20 billion in 2020 and $22.7 billion in 2021," she said. "There are still significant challenges for biotech companies to be tackled but long-term investment in the sector remains robust."
Paying for the coming wave of ATMPs
How to pay for the next wave of advanced therapy medicinal products (ATMPs) was the focus of a panel discussion moderated by Francis Pang, vice director and head of global markets access at Orchard Therapeutics.
Speakers pondered how to establish earlier discussions among developers, payers, and health technology assessment (HTA) authorities in Europe. They also addressed cross-border barriers that can delay patient access.
"The ATMPs field is at a critical inflexion point," Pang said. "The main objective is for HTA developers to bring to market products which add value to patients but also (to) healthcare ecosystems."
These sentiments were echoed by Valérie Paris, president of the Economic and Public Health Committee at the French National Authority for Health, who spoke in the session from Paris.
"The role of HTA is to give more information about the value of a new or already existing product ... and to provide economic assessment and assess opportunity costs of these new therapies," Paris said.
In Europe, each country has its own process to define the reimbursement policies of the products that are coming on the market, but consensus is emerging, she explained.
"We're seeing and expecting to see more and more consensus on the clinical front of this assessment of public benefit ... and we really hope to obtain an agreement of HTA release about the clinical added value of a new product," she said.
There is no reason to judge the value of ATMPs differently to other products, she added.
"Economic evaluation is in principle the best way to give justice to [ATMPs] because you'll be able to demonstrate that the new product brings benefits over time," she said.
The two main challenges with ATMPs are to collect enough data even after launch to be able to prove and show benefit over time, and to meet countries' expectations in terms of willingness to pay, she explained.
"That's the tricky part, because not every country publishes thresholds on their willingness to pay for such therapies," Paris said.
Patients don't care about innovation, but rather about how it translates into patient care, according to Yann Le Cam, CEO of the EURORDIS-Rare Diseases Europe, who also took part in the discussion.
"With ATMPs ... the value at the end is if you get the medicine for you or your relatives," he said. "This is what counts."
"By 10 years, we expect to see three to five times more medicines, two to three times cheaper," he added. "We want to have many more therapies for rare diseases."
More resources should be allocated to rare diseases, as few treatments are available today.
"It's more difficult to assess a product's value for rare diseases than for cancer. The conversation is more difficult with rare diseases. If we agree that the objective down the road is to make medicine more accessible, then it should be cheaper," Le Cam concluded.