Key Takeaways
- Kiji Therapeutics’ CEO Miguel Forte says that investment in cell and gene therapies might take a hit in light of ongoing global “turmoil” but he remains confident in the future of advanced therapies.
- The EU Health Technology Assessment Regulation is based on the right principles, according to Forte, and a “pragmatic approach” must be applied to its implementation.
- He applauded the European Commission for its plans to make the “hospital exemption” provision for ATMPs more transparent under its proposed review of the pharmaceutical legislation.
Changes to global political, investment and regulatory systems could impact the speed at which cell and gene therapy companies are able to develop products, but Miguel Forte, CEO of Kiji Therapeutics, remains confident in the “intrinsic value” of the technology itself.
“Am I confident in the future of [gene therapies]? Absolutely. We’re seeing the value, and we’ll continue to see the value,” Forte, who is also a board member of the Alliance for Regenerative Medicine advocacy group, told the Pink Sheet.
However, he acknowledged the world was in “turmoil” due to the political and economic environment, and that “everything, including cell and gene therapy, is suffering.”
“If cell and gene therapy is particularly challenging from a technical, cost of development or cost of production [perspective], it is going to be particularly hit, because if you’re risk averse, where you see the biggest risk, that’s probably where you have the biggest aversion,” Forte explained.
“I don’t doubt the value of cell and gene therapy, but obviously that value will depend on the conditions that prevail and the speed of how quickly we’re going to see the value being translated into real products and delivered to real patients,” he added.
On the approval of cell and gene therapies, Forte noted that challenges varied from country to country. Under the EU’s centralized procedure, for example, multiple member states need to come together and make an “articulated decision” on the approval of a new product, which differs from the UK where the regulator can make decisions independently. Meanwhile in the US, he said that the restructuring of the Food and Drug Administration could pose difficulties for manufacturers.
“All of those are the day to day operations which could definitely impact the speed that we’re able to develop [products],” he said.
EU HTA ‘The Right Principle’
In the EU, the ongoing revision of the general pharmaceutical legislation in addition to the introduction of the new Health Technology Assessment (HTA) Regulation are key changes impacting the gene therapy industry, Forte said.
Overall, though, he supports the ambitions of the regulatory changes in the bloc, stating that when it came to the HTA Regulation, “the principle is the right principle.”
“It’s making sure we have a centralized approach, making sure we take advantage of the dispersed expertise, and making sure that you only do it once and it’s applicable everywhere,” Forte said.
He said the biggest challenge with the HTA Regulation was that each country will still decide on the reimbursement and pricing of a product, after taking into consideration the outcome of the joint clinical assessment (JCA) conducted at an EU-level.
“It means member states will do whatever they want vis a vis the product, based on their budget, which is okay, but still gives them that loophole,” Forte stated.
JCAs are similar to relative efficacy assessments that have previously taken place at a member state level. From 12 January, all new advanced therapy medicinal products (ATMPs) and oncology treatments filed in the EU have been subject to the JCA process. It will begin to apply for some other products from 2028 and to all new products in 2030.
He said that the HTA Regulation needed to take into consideration the “specificity of a product,” such as difficulties in conducting comparative trials for orphan drugs, which are intended for smaller patient populations.
“You need to have some pragmatic approach on the decisions, and that pragmatic approach is not embedded in legislation, but hopefully will be the result of practice.”
Hospital Exemption ‘A Useful Tool’
Forte also said that the European Commission’s proposed reforms of the “hospital exemption” provision for ATMPs as part of its overhaul of the general pharmaceutical legislation could have a positive impact for the industry.
While some industry representatives have criticized the hospital exemption, Forte described it as “a useful tool” for providing sporadic access to ATMPs for patients with no other treatment option.
Under the hospital exemption provision, member states can allow the use of ATMPs without a marketing authorization in certain circumstances. For example, products must be made in a hospital setting for an individual patient, and should only be used in the member state where it was made. However, the uneven implementation of the provision has prompted concerns around misuse to circumvent strict legal requirements for the marketing of safe and effective ATMPs.
The commission set out new measures on traceability and the collection of data around the use of the hospital exemption in its legislative proposal in a bid to improve and harmonize the way the exemption works in the EU.
Forte said that he applauded the commission’s aim of making the hospital exemption more transparent, rather than taking it away as an option for patients.
“It serves a purpose that we all have, which is to bring value to patients in need,” he said, adding that the mechanism should not compete with products that have gained a marketing authorization in the EU.
This is the second of two articles based on the Pink Sheet’s interview with Forte. In the first piece, he discusses why all stakeholders must be committed to the EU Clinical Trials Regulation if it is to be a success.