Masterarbeit
Theranostic Radiopharmaceuticals: A Clinical View on the Approaches to Marketing Approval in the EU and the US ***
Dr. Alessandra Bierwagen (2025)
Summary
Language: English
Radiopharmaceuticals are a niche sector in the global pharmaceutical market, but they are growing steadily and the theranostic concept is an important driver in this process. Theranostics, a combination of the words "therapeutics" and "diagnostics”, describes the use of radioisotopes to first image a patient's tumor for diagnostics and then therapeutically treat that tumor. In the last 10 years, two therapeutic radiopharmaceuticals with their respective diagnostic counterparts have been approved in the US and the EU. Given this low number, several people and publications demand streamlined approval processes to overcome the complex regulatory frameworks for theranostic radiopharmaceuticals.
This thesis evaluates the current status of the regulatory framework for theranostic radiopharmaceuticals focusing on clinical aspects of development and approval. Based on development and approval pathways of already marketed radiopharmaceuticals as well as through discussion of regulatory pathways for comparable drugs, the ideal strategy is delineated. Furthermore, some suggestions introduced in publications as well as development pathways of radiopharmaceuticals currently under development are considered and critically discussed.
For example, in the thesis the “Companion Diagnostic” framework, often used in the context of facilitating theranostic approvals, is considered. Besides from an EU regulatory perspective not foreseen, the concept of Companion Diagnostics with approvals needed from a notified body and a regulatory authority, comprises no advantage over the current regulatory framework for diagnostic radiopharmaceuticals. Same applies for the US Premarket Approval being a rigorous process involving the demonstration of the device´s safety and efficacy not differing much from the NDA process.
Reviewing the currently approved radiopharmaceuticals, one pair of theranostic radiopharmaceuticals has employed several aspects towards a framework for theranostic radiopharmaceuticals already, e.g. FDA and EMA reviewed the application in parallel within the same timelines, which not prolonged compared to other approvals. While most approvals in the last decade did not use the clinical synergies of a theranostic study design, in this case also the clinical studies were shared, a strategy which can also be observed for the current theranostic radiopharmaceuticals under development.
Nevertheless, embedding existing approaches like reduction of fees, linking designations between the diagnostic and therapeutic products and aligning assessment timelines in a “Theranostic development of radiopharmaceuticals” guideline could further promote the development of theranostic radiopharmaceuticals. Also, a common understanding which evidence needs to be shown when seeking approval, taking into account the special nature of diagnostics in general and the theranostic concept would provide clarity for trial design and conduct.
In summary, a parallel development of the diagnostic and therapeutic radiopharmaceutical allowing a coordinated overall strategy should be the aim of pharmaceutical companies and the regulatory authorities to bring these life-saving theranostic radiopharmaceuticals to the market more quickly.
Pages: 66
Annexes: 5, Pages: 23