Market Access of Atmps: Overview and Expected Challenges

Abstract

The goal of this research is to understand the extent to which payers around the world are using discounted or net prices for international reference pricing (IRP) purposes as an alternative to list, manufacturing or wholesale prices. The research also examines what sources are being used to obtain these discounted prices. MethOds: Primary research was conducted with payers in 45 countries to establish the methodology of IRP in those countries. Specific questions were asked around both the current and potential future use of different types of prices for IRP. This was supplemented with secondary search and literature reviews of legislation and healthcare sources related to IRP to understand mechanisms of IRP. Results: Overall, it was shown that 8 countries used some form of discounted prices for IRP purposes, and that the number of countries is increasing. In addition, one country (Bulgaria), which previously had a requirement to reference discounted prices for

Advanced therapy medicinal products: current and future perspectives

“…ATMPs are a new class of biopharmaceuticals. Eight years after the adoption of the regulation, only five products have obtained regulatory approval in the EU and achieved limited success in securing reimbursement ( 20 ). This fact suggests a low impact of ATMPs to date on health insurance budgets but also on patients’ health.…”

“…The payers are facing the challenge of creating a balance between ensuring the financial sustainability of the healthcare system and encouraging the innovation and development of new therapies to address unmet needs. This situation, together with poor preparation of pharmaceutical companies, may explain the limited success of the five approved ATMPs to secure reimbursement in the EU ( 20 ). The manufacturer of alipogene tiparvovec (Glybera ® ), the first gene therapy, is seeking a price of 53,000 euros/vial, thus 1.1 million euros per patient.…”

Preparing for cell culture scale-out: establishing parity of bioreactor- and flask-expanded mesenchymal stromal cell cultures

“…Manufacturing efficiency is the third crucial aspect for the viability of cell therapies as a whole. Previous pioneering therapies involving expensive production processes have failed due to unfavorable cost–benefit assessments and lack of reimbursement [29]. Therefore, there is a clear need for alternatives to traditional labor-intensive flask-based cell expansion processes.…”

Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe

“…Reimbursement of ATMPs is frequently mentioned as a major hurdle, both from a developer and health technology assessment (HTA) body point of view. 28 , 30 , 31 In our survey, financial challenges represented only 10% of responses, of which 5% specifically address reimbursement perspectives. It is likely that this low percentage can be explained by the early development stages of the ATMP field and high SME representation.…”

“… 32 On top of that, the manufacturing of ATMPs is considered to be more expensive by nature and is expected to pose pressure on healthcare budgets. 30 Combining the active ATMP pipelines with the prospect of healthcare budget constraints, sustainable ATMP reimbursement will become the next major challenge in this field if not already a reality. Companies should therefore address commercialization of their ATMP early in development.…”