Digital therapeutics, the costs of gene and cell therapies, and a challenging financing situation were some of the hot topics in 2023 that SwedenBIO sniffed out at J.P. Morgan’s annual healthcare meeting in San Francisco.
The international life sciences year started off with J.P. Morgan’s Annual Healthcare week, which drew thousands of life science experts to San Francisco in January. SwedenBIO was on the ground to scout out the most pressing matters on the minds of global life science leaders this year. Three particular trends stood out, which are as relevant in the Nordic context as they are internationally.
Digital therapeutics, also called software-as-a-medical-device, is software with the aim to prevent, manage, or treat a specific disease or condition. This class of therapeutics is growing fast and is challenging traditional regulatory and reimbursement and payment models, forcing authorities to reconsider the ways therapeutics are evaluated.
As digital therapeutics are evolving, they often require clinical evidence. To advance the safety and efficacy of digital therapeutics, the US Food and Drug Administration (FDA) launched the Software Precertification Pilot Program in 2017 to explore and evaluate innovative regulatory approaches. Upon completion of the program in September 2022, they concluded that rapidly evolving technologies would benefit from a new regulatory paradigm and the FDA will continue to develop policies and tools to improve regulatory oversight. Meanwhile, in the European Union digital therapeutics are defined as medical devices and need to follow the new EU Medical Device Regulation concerning safety, efficency, and performance measures.
The FDA and the European Medicines Agency (EMA) have approved several digital therapeutics, upon completion of clinical trials with positive outcomes, to treat conditions like chronic insomnia, ADHD and substance use disorders – and clinicians are prescribing these therapeutics actively.”
Given the high level of digitalization in the Nordic countries, digital therapeutics are expected to become popular among patients. The FDA and the European Medicines Agency (EMA) have approved several digital therapeutics, upon completion of clinical trials with positive outcomes, to treat conditions like chronic insomnia, ADHD and substance use disorders – and clinicians are prescribing these therapeutics actively. Clear regulatory and reimbursement pathways are however needed. Many initiatives, such as the Nordic Digital Health and Medication Platform, are ongoing to advance implementation of digital therapeutics.
Gene and cell therapies
Gene and cell therapies, also called advanced therapy medicinal products (ATMPs), are getting approved for several diseases, such as cancer and severe genetic diseases like hemophilia. As the number of approved gene and cell therapies is increasing, many actors are worried about the price tags of these therapies.
The cost of a single treatment of these types of therapies is high. However, if the treatment can cure the patient for life, the cost of treatment is easier to justify. The total cost of gene and cell therapies boils down to the durability of the treatment – how long the patient stays healthy with one treatment. More data is needed on durability of gene and cell therapies, such as the first FDA and EMA-approved hemophilia B gene therapy developed by CSL Behring, which costs 3.5 million USD per treatment.
In Sweden, several small-sized biotech companies are actively developing gene and cell therapies, but are struggling to showcase the market value of gene and cell therapies for investors due to ambiguous reimbursement models.”
In Sweden, several small-sized biotech companies are actively developing gene and cell therapies, but are struggling to showcase the market value of gene and cell therapies for investors due to ambiguous reimbursement models. Establishing reimbursement models for new types of therapeutics is essential for advancing the development of novel therapies.
Here, the total cost of the therapy is critical when evaluating new reimbursement models for gene and cell therapies. Many actors such as ATMP Sweden and SwedenBIO’s ATMP task force are addressing challenges related to advancing gene and cell therapies.
Financing in an economic down-turn
The challenging financial climate is forcing companies to rethink their business models and R&D processes – it’s all about more bang for your buck in 2023. Many companies are exploring opportunities to share R&D and manufacturing facilities – and even employees – with several corporates to reduce running costs. R&D companies are looking into new income sources, such as offering services or licensing parts of their platforms along with their internal R&D projects.
Many companies are exploring opportunities to share R&D and manufacturing facilities – and even employees – with several corporates to reduce running costs.”
As the global financial landscape becomes more challenging, collaboration across the Nordic borders is even more crucial than before to ensure that the region can compete on the international market.
These trends and many other topics are currently under consideration as themes for the Nordic Life Science Days’ conference program, which takes place in Copenhagen in November 2023.
This column was originally written by Marjo Puumalainen, International Director, SwedenBIO, for NLS magazine, No 01 2023, out February 2023
Photo of Marjo Puumalainen: Camille Sonally