How to Facilitate Development of Cancer Immunotherapies

Challenges for the approval of anti-cancer immunotherapeutic drugs

A workshop on the challenges for the approval of immunotherapy medicines for cancer, held at the European Medicines Agency (EMA) on 4-5 February 2016, was organised jointly by EMA and the Cancer Drug Development Forum (CDDF). Attendance was by invitation only and the workshop was lively broadcasted. It was intended to oncologists/scientists, government officials (EMA, FDA, HTA representatives), pharmaceutical industry representatives, and patient organisations.

Immunotherapy is a fast growing area of new medicines in oncology. A new type of immunotherapy to fight cancer cells was approved for the first time in the European Union (EU) in 2015. These medicines target suppressive signals that prevent the development of an immune response, thereby enhancing the immune system to help fight cancer. These new agents have brought important advances in patient care and have considerably changed the landscape of treatment options in melanoma and lung cancer patients.

However, there are still many challenges related to bringing cancer immunotherapies through the regulatory approval process and into clinical practice. This joint EMA-CDDF workshop provided a platform for experts from academia, oncologists, policy makers, representatives from health-technology-assessment (HTA) bodies, the pharmaceutical industry, regulatory bodies and patient organisations to discuss the challenges related to the development and approval of these medicines.

Programme chairs were Silvy da Rocha Dias (European Medicines Agency, UK), Francesco Pignatti (European Medicines Agency, UK), Jan Schellens (Netherlands Cancer Institute, Netherlands), Martina Schüssler-Lenz (Paul-Ehrlich Institute, Germany), John Smyth (University of Edinburgh, UK), and Elena Wolff-Holz (Paul-Ehrlich Institute, Germany).

The experts discussed in particular appropriate patient population selection, new clinical trial designs, the rationale for the mechanism of action in different tumour types, innovative immunological-based products used in combination, and assessment of relative efficacy of these novel medicines. The aim was to address these highly relevant issues and their impact on the regulatory environment.

Following a keynote lecture entitled status quo and upcoming concepts in immunotherapy of cancer, academic, industry and regulatory perspectives were presented in the session on the challenges for the different stakeholders.

In the session on lessons learnt in melanoma and non-small cell lung cancers, the presenters provided perspective from academia, industry and regulatory bodies, as well as a presentation on safety – immune related adverse events.

A session on population selection featured presentations on biomarkers for PD-1/L1 inhibitors: regulatory considerations, blueprint project PD-L1 coDx comparability, novel biomarkers: pitfalls, limitations, emerging options, as well as a panel discussion.

A session on strategies such as peptides and cell therapy featured presentations on mRNA and peptide-based anticancer immunotherapies, chimeric antigen receptor Tcell therapies, regulatory view and panel discussion.

A session entitled endpoints, combinations and analysis featured presentations on differences between the use of immunotherapy in the adjuvant as opposed to advanced setting, study design and analysis in late-stage cancer immunotherapy trials, US and EU perspectives on sequencing or combination of immunotherapies with targeted therapies, challenges in developing novel-novel immuno-oncology combinations, and panel discussion.

A session on HTA assessing relative efficacy featured presentations on challenges in evaluating relative effectiveness, capturing added benefit, managed entry agreements, patients’ perspectives, patient reported outcomes: the impact of HRQOL, and panel discussion.

A wrap-up on future perspectives was organised through the panel discussion.

Presentations and a video recording of the event will be published after the workshop.