Advanced therapy medicinal products have the potential to create completely new ways of treating – and sometimes even fully curing – illnesses. This is mainly because they can be tailored specifically to an individual. Yet, together with the new possibilities come new challenges, such as those relating to health economics.
Advanced therapy medicinal products (ATMPs) are medicines based on cells, tissues or genes. The medicines hold great potential in treating diseases and in halting or curing conditions which, in the past, have been entirely incurable. They also afford significant opportunities to repair, restore, or replace tissue.
With potential comes challenges
Despite the great potential the medicines hold, they present a number of challenges to, among other things, health economics. Calculation models for health economics within healthcare weigh up factors such as costs, value, and quality-adjusted life years, and are not relevant to these new medicines.
– “It is anticipated that ATMPs, in many cases, will be able to cure illnesses in a single treatment, and this is where the calculation models break down,” explains Anna Ridderstad Wollberg, Head of the New Therapies focus area at RISE. “It is difficult to calculate when the patient will be cured and it’s impossible to say how long they will live.”
Market approval needed
To make a medicine available to patients requires marketing authorisation through a regulatory framework which also applies to ATMPs.
– “When cells are used for something that they are not from the beginning, it is classified as a pharmaceutical product, says Ridderstad Wollberg. “The products therefore need to comply with existing pharmaceutical regulations before reaching the market.”
Compared to traditional medicines, however, ATMPs are often tailored to the individual, which severely restricts the market and makes each single treatment very expensive. In turn, this presents additional challenges for healthcare, the budget of which is not formulated to cover very high one-off costs.
– “For example, if we develop a drug in which we add a gene that controls the T cells to kill cancer cells, then such a treatment could cost millions, says Anna Ridderstad Wollberg. On the other hand, the treatment might only be administered a single time, so the total cost for healthcare won’t necessarily be higher overall.”
RISE is a partner in the national Vinnova-financed projects CAMP and Swelife-ATMP, which has an important goal to increase Sweden's attractiveness and competitiveness in the ATMP area and help more ATMP reach the patient. One of the sub-projects within the Swelife ATMP is coordinated by RISE and focuses on health economics and business development in the field of advanced therapy drugs, with Anna Ridderstad Wollberg as project manager. Through the project, a large number of actors have now been gathered to identify obstacles and opportunities to facilitate more advanced therapy drugs to reach clinical life and patients more quickly.
– “What used to be a dream has become a reality and several market-approved ATMPs are now knocking on Sweden's door. Here, all players in the field need to collaborate and find new solutions, quickly, both in terms of payment models, health economic evaluations, processes in the healthcare sector and collaboration with the pharmaceutical companies. Our project has brought together all these actors and we propose solutions that we align with the decision makers. This is only the beginning, but I already see that we will deliver results that contribute to the sharp situations that are actually happening in the regions right now, but also results that will pave the way for developing new ATMPs successfully in Sweden.”
Roadmap and checklist
The project will run until 2020, and the plan is to create an ATMP development guide aimed at multiple target audiences, such as innovation offices and financiers, so that they can support this type of project more efficiently.
– “I started as an immunologist, and I think it’s really exciting that we have caught up with a future that seemed far off. There are myriad illnesses and health challenges that still need to be treated, and what we have here is a way to treat illnesses which had previously been difficult to treat. It creates new possibilities,” concludes Ridderstad Wollberg.