Early stage cooperation between patients, developers, regulators and HTA bodies is critical for success in cancer treatment development
During the FIGON Dutch Medicines Days on October 1-2, the RSNN organized two regulatory science sessions around novel cancer treatments and the associated regulatory challenges. We look back at engaging presentations and discussions with a large, varied group of stakeholders.
Click here for the speaker presentations
A wave of innovative oncology treatments is moving from bench to clinic. Examples include therapies based on tissue engineered products, gene therapies, and cell therapies using, for instance, CAR-T cells. During the RSNN sessions, eight speakers from academia, government, pharmaceutical companies, and a patient organization presented their views on the challenges that come with these new developments – and on the fact that, as one speaker said, “nowadays, every new treatment in oncology is innovative.”
Interaction
During the morning session, potential implications for regulators, companies and other stakeholders were discussed, using so-called Advanced Therapy Medicinal Products or ‘ATMP’ as an example. These products require innovative approaches to trial design, risk-benefit assessments, and market access. Presentations by Marcel Hoefnagel (CBG-MEB), Harm Hermsen (Xendo), Pauline Evers (NFK) and Renske Ten Ham (Utrecht University) gave rise to a lively discussion about the interaction needed between the research, innovative industry, regulatory, patient, and prescriber communities present in the audience.
Real-world evidence
During the afternoon session, Paula van Hennik (CBG-MEB), Alwin Otten (Janssen), Jürgen Kuball (UMC Utrecht), and Brenda Leeneman (Erasmus University) discussed evidence generation for oncology products. The speakers and audience questioned whether or not randomized controlled trial design (RCT) is still the best trial design for studying ATMPs, and wondered how real world evidence (RWE) fits in. Brenda Leeneman presented results of a nationwide population-based Dutch Melanoma Treatment Registry (DMTR), and indicated that RCTs are crucial for establishing efficacy. However, RWE still adds significant value, enhancing generalizability.
Start with the end in mind
Knowledge exchange
During the two sessions, it became clear that exchanging knowledge and information during the whole development process is essential. For example, trial design requirements that come with the introduction of treatments need to be discussed at an early stage to avoid costly failures or unnecessary exposure of patient populations. It was also suggested that the regulatory process should not obstruct developers, but instead enable a control strategy to manage the risks related to drug development and use in clinical practice. This risk-based approach should be, as Marcel Hoefnagel stated, nothing more than ‘formalized common sense’. Innovative cancer therapies, being much more personalized and targeted than traditional treatments, require us to find a balance between standardization and a case-by-case approach.
The key takeaway from the day can be summarized in the words of Harm Hermsen who said, in line with Stephen Covey, that we should always, “start with the end in mind.” Companies, especially SMEs, should start (planning) the regulatory process early, and integrate it within the development process to maximize the likelihood of success for developers, patients, and society.
We look back at a successful day and look forward to seeing you again at one of our upcoming Regulatory Science events.
Picture courtesy Bart Versteeg