WP leader: French National Cancer Institute (Marianne Duperray)

Outputs and deliverables

Innovative cancer therapies in clinical practice guidelines

The field of anticancer drugs has strongly evolved over the past few years. In order to point out challenges associated with the integration of innovative therapies into clinical practice guidelines, the WP9 has conducted a mapping and review of existing clinical practice guidelines placing innovative immunotherapies in the cancer treatment strategy (with a focus on checkpoint inhibitors and CAR-T cells). This report gathers the results of the literature review conducted over the summer 2018 and results collecting from 2 questionnaires sent out in 2018. The first questionnaire was addressed to iPAAC partners to get a better understanding in national practices around guidelines in the oncology field; the second questionnaire was sent to WP9 partners and organizations in charge of guidelines writing. This report also includes off-label recommendations as well as recommendations linked with biomarkers expression. Finally, main challenges regarding the integration of innovative immunotherapies in clinical practice guidelines and maintenance of guidelines up to date are highlighted. Some ideas were suggested to improve the length of production, availability, visibility and update of these guidelines.

Reference frameworks linked with the access to innovative therapies

This report gathers information on the reimbursement of immunotherapies in European countries with their potential restrictions of uses as well as existing frameworks enabling early access to innovative immunotherapies. After a preliminary literature review of HTA opinions, the first questionnaire was sent out in 2018 to iPAAC partners to collect information about conditions of reimbursement of checkpoint inhibitors and CAR-T cells and early access programs for unapproved indications. Opinions from different stakeholders collected via a second questionnaire are also presented regarding the access to innovative immunotherapies. Several suggestions aiming to reduce inequities in terms of access to innovative immunotherapies and for control rising prices are also discussed.

Horizon scanning systems applied for cancer control in Europe

Horizon scanning systems (HSS) have been shown to be a useful tool to support policy makers and healthcare professionals in predicting the availability of new medicines and their main impacts.This report presents information collected through a literature review on the main existing horizon scanning systems and related networks in Europe. A questionnaire was sent out in 2019 among organizations in charge of horizon scanning systems to characterize the methodological specificities needed to enable a better anticipation of anticancer drugs, gene and cells therapies, biomarkers and pediatric indications. In addition, a retrospective analysis of the efficiency of HSS was conducted in order to identify strengths of existing HSS as well as difficulties encountered in the oncology field. Finally, the remaining challenges and perspectives enabling an efficient anticipation of innovative therapies in oncology and their potential related biomarkers are highlighted.

Real-life monitoring of innovative immunotherapies

The recent arrival of innovative therapies such as specific immunotherapies in cancer treatment strategies was associated with strong hopes among cancer care communities. However, despite their arrival on the market, uncertainties can remain regarding the benefit-risk ratio or regarding their position in therapeutic strategies notably due to the nature of data from clinical trials (immaturity, non-comparative). Innovative therapies are also most of the time linked with high prices which can lead to major impacts on healthcare financing systems. The collection of efficacy and safety data in real-life settings appears thus essential to continue gathering clinical evidence to confirm the positive benefit-risk of new therapies. In this task, the WP9 choose to take CAR-T cells as an example to see what kind of programs and initiatives were implemented in European countries to monitor the use of innovative therapies. In this way, this report provides a state of play of existing initiatives for the real-life monitoring of patients treated with CAR-T cells and also presents the remaining challenges highlighted by the various stakeholders contacted and notably the gaps that would need further European coordinated approach.


02–03/07/2018, Boulogne

WP9 description and objectives

Today, plenty of new cancer drugs are available. Innovative therapies have an important place, especially specific immunotherapies. They have upset the landscape of cancer drugs available. New innovative treatments which have shown an improvement of overall survival, are involved in a modification of therapeutic strategy and patient follow-up. Unfortunately, before innovative therapies' marketed access, it is difficult to forecast their impact on therapeutic, organizational and economic strategies. In order to reach these objectives, clinical practices need to be known. Furthermore, early assessment and monitoring good use of these drugs should improve health expenditure, avoiding inappropriate prescription or additional costs due to hospitalization in order to manage side effects.

This WP aims at mapping all the existing guidelines on innovative treatments on cancer and creates a database accessible for professionals.

It is proposed to focus the WP on immunotherapies which currently represent the most innovative cancer treatments options and reflect the many challenges faced regarding the proper use of cancer drugs (clinical use & compliance with existing clinical guidelines), the numerous clinical developments (many clinical trials relate to immunotherapy and concern many therapeutic indications in oncology), the identification of responders (biomarkers are currently assessed to identify the best responders to immunotherapy), the anticipation of incoming new therapies & rising costs using systems like Horizon Scanning, the drug assessment based on real-life monitoring.

Although there seems to be currently no formal consensus regarding the definition of innovation in cancer drug, countries’ health authorities use a set of criteria like improved global survival and a response to unmet medical needs. In this sense, immunotherapies may represent the most innovative current option for cancer treatment. Countries also continuously face the challenge to anticipate and support the entry of new drugs. Few countries use systems to monitor drugs having a potential major impact on therapeutic strategies and potentially expensive, like Horizon Scanning. Promoting use of such systems among Member states would be most useful.