After almost two years since Lightning Health attended an in-person congress, some of our team were at the Advanced Therapies Congress & Expo, 5-6 October 2021. The congress gathered academics, manufacturers, suppliers, CROs, payers, and consultancies to discuss the fast-growing area of Advanced Therapy Medicinal Products (ATMPs).

Yasmina Zegaoui, Senior Consultant and Nasos Kipentzoglou, Senior Analyst at Lightning Health were at the meeting and report on some of the key topics from the two-day event.

Applying cell-based knowledge to COVID-19 and cancer vaccines

The technologies to use cell and gene therapies are varied, with some companies introducing cell-based vaccines for on-demand, on-site production of COVID-19 vaccines utilising a patient’s own blood sample. Vaccines have already been used in Indonesia and South Africa, allowing patients who are the most reluctant to vaccination to get closer to the production of the vaccine they are being injected with. The added value of this on-site production? A reduction of wasting costs as each vaccine is manufactured for a given patient, at a price allowing emerging markets a low-income market to afford the COVID-19 vaccination outside of the usual systems. The manufacturer is now working on using the same technology for personalised cancer vaccines.

Regulatory processes for ATMPs

With the growing availability and development of ATMPs, regulatory processes are shifting towards more simplified access routes. In recent years, 71 ATMPs received an accelerated investigational or ATMPs approval in addition to the 25 approved “ATMP” without pre-designation.

Outside of the current regulatory tools in place for ATMPs (such as the early scientific advice procedure, the PRIME (EU) and RMAT (US) designations) that already help manufacturers to access the market earlier on in the development process, the upcoming EUnetHTA 21 will give priority to orphan drugs and ATMPs to allow patients to access these innovative therapies.

Broadening the scope of gene therapies, expanding towards less rare conditions

How to broaden the scope currently covered by gene therapies? This was one of the key topics discussed by manufacturers at the congress. With the wider access to COVID-19 vaccines around the world, that some consider an ATMP, manufacturers are shifting towards a business model allowing them not only to develop gene-specific therapies but also more agnostic therapies to be used in some of the more common disorders such as hyperlipidaemia or dementia.

From a pricing point of view, each model would come with a different pricing potential, with less targeted therapies being priced at a more affordable rate due to the high volume of patients expected to be treated.

Manufacturers also highlighted the need to translate the clinical evidence packages into successful market access by choosing the correct initial indication for the therapy and initiating earlier interactions with regulatory authorities and payers would increase the likelihood of positive outcomes and align the product’s value proposition with HTA bodies’ expectations. In rare conditions where no standard of care is currently available, earlier interactions with regulatory authorities are even more crucial to inform clinical trial design, engagement with patients to ensure the relevance of endpoints from a patients perspective and seek alignment with the value proposition of the gene therapy.

The manufacturing of gene therapies will also be key issue to overcome, with the current restrictions associated with the time to ship and administer gene therapies to the patient potentially limiting the efficient access of advanced therapies to patients. Despite the ongoing innovations to improve the way gene therapies are produced, there is still a lot to be done in the logistical infrastructure and delivery systems. A global effort may be required to scale up the manufacturing of gene therapies, much like the effort countries put in more than 30 years ago to fully understand the genome.

Payers expectations regarding pricing and market access for ATMPs

With the rapid development of high value ATMPs, a key topic discussed at the meeting was the pricing and market access activities for such therapies.

Given that ATMPs are developed usually for orphan/ultra-orphan diseases and are administered once per lifetime with potentially life-long benefits, this creates several questions to manufacturers and payers regarding payment schemes and certainty of evidence at launch.

Payers highlighted that there is a critical need for the standardisation of clinical evidence generation, with simple and clear endpoints. This would aid the discussions between manufactures and national health authorities when faced with the potential of limited certainty at assessment. Payers consider that outcome-based agreements may not be appropriate for all ATMPs, especially considering the potential complexities of such agreements. On balance however, these type of agreements are preferred as they have the potential to ensure the collection of long-term data (5-years at least).

Contact us

Lightning Health can support you to navigate the complexities of bringing a new ATMP to market and to ensure robust value demonstration across healthcare systems. Contact us to discuss how we can support your European launch and patient access strategy.

Article published 11 October 2021