Introduction: Mortality in cancer has declined during recent years due to more efficient cancer treatments. Some of the novel immunotherapies against cancer under development has a risk of lacking regulatory guidance for the approval, such as neoantigen-based therapeutic vaccines. Aim: The purpose of the degree project is to evaluate the potential process of approval of neoantigen based vaccines from a regulatory perspective. Methods: The method used to gather data in the study is through qualitative interviews, regulatory documents, guidelines and data from current trials. Results: The results indicate that during the preclinical phase, neoantigen vaccines can receive warrants of a case-by-case approach leading to a more optimized development pathway. During clinical trials, the trial design could consist of master protocols, singlearm trials, comparative trials and adaptive trials where the control group may use the standard of care or historical controls. The indication in the trials should be cancer types with a high tumor mutational burden, low mortality rate, and high medical need. The biomarkers should evaluate immune and tumor response. Endpoints in early trials should evaluate safety and efficacy, and the tumor and immune response in pivotal trials. Neoantigen vaccines can use several incentives during the development and can be approved through conventional or conditional approval. Finally, the study suggests that it is yet too early for the agencies to have established any guidelines. However, advices from regulatory agencies can guide developers towards regulatory approval. Conclusion: This study shows that different study designs and various incentives can be used for the regulatory approval of individualized neoantigen based vaccines. Further guidelines need to be developed to enhance future development.

Cancer is a global health issue and is estimated to be the second leading cause of death worldwide. Within cancer treatment, it has become attractive to introduce precision medicine to harness the body’s own immune system to fight cancer. Personalised peptide cancer vaccine can activate the immune system and elicit an immune response by using the patient’s own blood and tumour cells.   The aim of this study was to gather information to better understand the production of individual-specific peptides and the challenges when producing these peptides with focus from a GMP perspective. The methodology for conducting this study and retrieve information for the result was gathered by three different data bases; EMA, European Commission and FDA, and by qualitative semi-structured interviews.   The findings show that these personalised peptides should be manufactured by using solid-phase peptide synthesis, cleavage from the resin, HPLC purification, and final salt exchange. The traditional GMP requirements must be used as a basis to build a pragmatic program. In terms of delivery time, the peptides can be delivered within a few weeks. The main challenges will be to manufacture successful peptides since the peptide sequences vary from patient to patient and due to that fact have different production efficacy, deliver within the set timeframe, and have flexibility in the manufacture process.   Multiple guidelines need to be followed in order to set up a process that consistently delivers the intended product. When producing oncology drugs on-demand it is essential to keep the timeline since the patients are severely ill. However, further studies are needed to determine how the manufacture of personalised peptides could be performed to harmonise with regulatory guidelines.