ECC 2013 Report: Joint ECCO/BDA Special Session: Accelerating patient access to innovative drugs in Europe

by Peter Mas Mollinedo – Improvements in diagnostics and medical treatments mean the number of patients living with cancer is increasing around the world. The World Health Organization is expecting the rate of cancer to increase by 50% to 15 million new cases in 2020.  With growing patient numbers and ailing public finances, patient access to innovative new cancer drugs is becoming increasingly restricted. A joint session with ECCO and the Biotherapy Development Association (BDA) highlighted the widening gap between patients and advanced new treatments both from the clinician’s and the patient’s perspective and discussed opportunities for improving access.

Chairman Professor Lothar Bergmann from Frankfurt, Germany set the scene by reminding delegates that the average time from a new medication receiving European marketing authorisation to it becoming available for patients can be up to 450 days in some countries [1]. Most European countries have government-funded bodies in place which assess the cost-effectiveness of a new drug at a national level once the drug has been approved through the centralised EMA procedure – such assessment procedures, together with lengthy pricing negotiations, can delay the access to new drugs by many months.

Jan Schellens from the Dutch national cancer institute illustrated the dilemma of regulators and oncologists – to make potentially life-saving medications available in the shortest possible time without jeopardising patient safety. In his talk Dr Schellens suggested several ways in which access can be improved, by targeting selected patients based on prognostic factors such as KRAS mutation status, and limit access to these well-defined subsets at an early stage through adaptive licensing. Later, as more data becomes available, access can be expanded to include other patient groups as appropriate. Global professional networks for biomarker identification and delivery of experimental access could also provide the means of accessing new drugs in some countries. However, according to Dr Schellens, the most important barrier to new drugs is risk, and taking more risks can only be acceptable with appropriate active surveillance and legislation in place to ensure prompt market withdrawal if necessary.

Kathy Oliver is one of the founders of patient advocacy group International Brain Tumour Alliance; she described the patient perspective on access. In many parts of Europe, the barriers to access are largely cultural – paternalistic and hierarchical doctor/patient relationships prevent patients from being actively involved in the treatment decision-making and often result in patient needs being ignored. Ms Oliver urged clinicians to regard their patients not only as research subjects but as research partners and allies as well, to ensure access to the best treatment for all patients.

In the final presentation, Dr Wim Goettsch from the European network for Health Technology Assessment (EUnetHTA) pointed out that although cost is the most common barrier to patient access to innovative drugs, the criteria for what actually constitutes an expensive drug can vary a great deal between countries and settings. In the Netherlands, the national Healthcare Insurance Board (College voor Zorgverzekeringen, CVZ) has developed a scheme for managed entry of high-cost drugs under which indications are restricted and the price reduced as the drug is introduced onto the market. Payment for performance and conditional reimbursement as further evidence becomes available ensures that innovation remains stimulated despite budgetary constraints. The initial experience in the Netherlands has been somewhat mixed due to low patient numbers and patchy data collection, but Dr Goettsch believes that with suitable adaptations, managed entry schemes provide a way forward for integrating cost-effectiveness and innovation in publicly funded national healthcare systems.

Reference

1.   Wilking, N. and B. Jönsson, A pan-European comparison regarding patient access to cancer drugs. Karolinska Institutet report., 2009.

Speakers

Lothar Bergmann, University Hospital, Frankfurt, Germany

Jan H M Schellens, Netherlands Cancer Institute, Amsterdam, the Netherlands

Kathy Oliver, International Brain Tumour Alliance, United Kingdom

Wim Goettsch, European network for Health Technology Assessment (EUnetHTA), Diemen, The Netherlands