Sustainable Innovation in Treatment Development: Re-engineering the Process for Access

Denis Lacombe, Director General, European Organisation for Research and Treatment of Cancer

Bringing therapeutic innovation and latest science to patients while keeping patient interests at the centre is a challenging mission. Precision medicine and new approaches to clinical research have dramatically changed the field of treatment, notably for cancer. New treatments become available based on solid scientific rationale thanks to the understanding of molecular biology and immunology.

However, optimal use of new anti-cancer treatments remains poorly documented. Patient populations (defined for example by biomarkers), treatment duration, sequence and combination of treatments within the current treatment pathways are often poorly defined by clinical developments for regulatory purposes. Therefore, major limiting factors to delivering cost-effective and affordable, evidenced-based care to clinical practice remain unaddressed. This leaves many gaps in the knowledge concerning the efficacy and therapeutic use of medicines which can impose a significant financial burden on the health care systems possibly to the detriment of more cost-effective interventions. A new balance between the interests and needs of all stakeholders needs to be re-defined around patient centredness. We argue that the evidence generated during the clinical development of these new products for the purpose of getting marketing authorisations often does not address fundamental questions concerning the impact of these new interventions on the most relevant clinical outcomes, quality of life and survival of the patients. Therefore, a societal gap in clinical and translational research exists, between early and late drug development often artificially split by the process of marketing authorization, after which the commercial sector may no longer pursue research for treatment optimisation.

Applied clinical research is needed to define effective practice-changing approaches, to bring therapeutic innovation efficiently and consistently to cancer patients, and to address patient populations who do not fall within the primary interests of the commercial sector. Truly identifying innovation and how to bring such innovation optimally to the clinic needs a profound reform of the forms and the methods, treatments are developed and implemented. Studies have reported that a vast majority of recently approved anti-cancer agents do not substantially impact on clinically meaning full outcome such as prolongation or quality of life [1–3]. This is echoed by Health Technology Assessment committees [4].

Clinical research and health care systems are long due for transformation. Access, affordability and effective use of treatments need intimate cross-links between research and care for successful implementation of precision medicine for European patients. Equality, affordability and the high-quality utilisation of new treatments need intimate cross-links between research and care for precision medicine to become a paradigm with real impact. Re-engineering the forms and methods of therapeutic innovation around the understanding of biology, through applied clinical research, would benefit to all, facilitating access while delivering more adequate information on value of treatments. Europe should develop solutions generating the appropriate datasets that patients and health care systems need for access. Independent applied clinical research should play a critical role in a transformative society.


[1]  C. Davis, H. Naci, E. Gurpinar, E. Poplavska, A. Pinto, A. Aggarwal, Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, BMJ. 73 (2017) j4530. doi:10.1136/bmj.j4530.
[2]  E.Y. Chen, V. Raghunathan, V. Prasad, An Overview of Cancer Drugs Approved by the US Food and Drug Administration Based on the Surrogate End Point of Response Rate, JAMA Intern. Med. (2019). doi:10.1001/jamainternmed.2019.0583.
[3]  B. Gyawali, S.P. Hey, A.S. Kesselheim, Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval, JAMA Intern. Med. (2019). doi:10.1001/jamainternmed.2019.0462.
[4]  B. Wieseler, N. McGauran, T. Kaiser, New drugs: Where did we go wrong and what can we do better?, BMJ. (2019). doi:10.1136/bmj.l4340.

Denis Lacombe will be sharing his views about the EU’s plans around cancer during the next European Commission mandate at EPHA’s upcoming Universal Access and Affordable Medicines Forum “Time for a New Deal: Challenging the status quo on medicines policy” which takes place on 14 November, at Scotland House in Brussels. Norway’s view on access to medicines, medicines shortages and transparency in pharmaceuticals policy are also on the agenda. 

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