DVS Biomarkers and Companion Tests

Background and challenges

The pharmaceutical industry faces many difficulties when introducing new drugs to the market. The number of drugs obtaining marketing authorisation is falling, even though the number of molecules submitted to regulatory authorities remains constant (Nature Reviews/Drug Discovery (2009) vol 8, pg. 959-967). The cost of developing a new drug has risen considerably in recent years. This is caused by considerable attrition due to low predictive power for animal models, signs of toxicity in humans and even a lack of clinical efficacy on the affected population.

In response to these new pressures, the concept of personalised healthcare, introduced by the Swiss laboratory Roche, has been growing for the past twenty years. This concept is based on the fact that the same drug can cause different reactions according to the disease and recipient. Some drugs are effective, while others are not. With the introduction of Herceptin to treat breast cancer in the 1990s, Roche showed that it was possible to predict the outcome by conducting a simple genetic test on patients to identify which patients would benefit from treatment and which would probably not. Herceptin is used to treat breast cancer and is only administered to females with cancer that has an overexpression of HER2. As such, it was the first approved drug from stratified medicine. Treatment and companion tests were therefore introduced.

The pharmaceutical industry must reinvent its business model where biomarkers will undoubtedly play a key role in future developments.

As of yet, nothing is set in stone regarding biomarkers. The pharmaceutical (Sanofi, Roche, Pfizer, etc.), diagnostic (Abbot, Roche, BioMerieux, BioRad, etc.) and device (Illumina, Brücker, LifeTech, etc.) industries are changing, and coordination and structuring initiatives will have a positive impact on both academic research and the economy. Indeed, although all are convinced that biomarkers will play a critical role, there are several obstacles in place that continue to restrict their usage. Furthermore, while many biomarkers have already been identified, very few have reached the market.

Market and Industry Expectations

The current business model for pharmaceutical manufacturers is changing, notably due economic pressures. The newly emerging model for biomarkers is not currently defined and will most certainly bring new players to value chains.

As regards drug manufacturers, some phase 2 drugs do not provide sufficient efficacy to be entitled to later reimbursement and should be abandoned. At this stage, the identification of susceptible biomarkers that could distinguish between responsive and non-responsive patients would increase the efficacy of the drug candidate on the population in question. This approach may lead to a decrease in the target population concerned. However, an improvement in efficacy would be expected. This approach will most certainly lead to a rethinking of business models.

Diagnostics manufacturers also have great expectations for biomarkers. If drug manufacturers are taking up biomarkers for the development of drugs (target biomarkers, stratification biomarkers, etc.), diagnostics manufacturers are focussing on diagnostic biomarkers, risk qualification and response to therapeutic treatment. There are several reasons for this difference. Firstly, time scales for drug development make it difficult for diagnostics manufacturers to invest in an associated "companion test" over a prolonged period. Moreover, the regulatory aspects that they must go through register a specific act is only possible when the drug in question receives market authorisation, which complicates the companion biomarker approach. Also, like drug manufacturers, business models remain unidentified.

Finally, certain ICT manufacturers associated with databases, modelling complex systems or simulation (Dassault, CAP GEMINI, IBM, Google,etc.) are beginning to focus on basic aspects and medical data analyses. These new entrants may play a leading role in the standardisation of biomarker approaches in the near future.

Planned action

DVS Biomarkers and companion tests proposes to bring together all value chain players in order to:

  • Identify the teams involved in biomarker research and validation (pathological or technological).
  • Make an inventory of biomarkers and place them according to their development stage with regards to medical validation (identified, verified, validated) and technical validation (reproducibility, sensitivity, specificity, robustness of the analysis) as well as offer support, all in project mode.
  • Work alongside pharmaceutical, diagnostics and device manufacturers to assess the development stage and level of interaction to establish between these players and academics.
  • Identify analyses methods or biomarker measures and the players who develop them.
  • Work with manufacturers concerning molecules that they would like to conduct specific biomarker research on.
  • Drive thoughts on issues concerning business models and reimbursement based on real cases and an exact definition of "clinical utility".

Creation date: June 2014
Coordinating institutions: Inserm Transfert / ITMO Technologies pour la Santé (TS)
Coordinators: Pauline Solignac (Inserm Transfert) / Franck Lethimonnier (ITMO TS)
Project manager: Inês Amado (Aviesan)

The key innovation domain’s (“Domaine de Valorisation Stratégique = DVS” in French) steering committee is responsible for monitoring the evolution of the objectives defined by the DVS, as well as for the validation of reports and strategic documents produced by the DVS. It consists of a biannual meeting of 40 to 50 people.

The list of participants includes:

  • DVS co-coordinators: Pauline Solignac et Franck Lethimonnier
  • Project manager: Inês Amado
  • Representatives: Inserm, Inserm Transfert, INRA Transfert, CNRS, FIST SA, ANSM, ITMOs (public healthcare, cancer, etc.), EFS, INRIA, APHP, Institut Curie, Eurobiomed, Medicen, Institut régional du Cancer de Montpellier (Montpellier Cancer Institute), Biorad, Roche, GSK, Servier, SAS Kyomed , Cysbio Bioassays, etc.
  • Industry representatives from ARIIS, Leem and SiDiV.
  • Representatives of technology acceleration and transfer societies (SATTs)
  • A representative of the management committee
  • Experts scientists

Latest news

The steering committee meeting number 5 took place on Mars 18th 2016.

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