The average time between EMA approval and the moment that patients gained actual access to the six innovative cancer medicines included in the study was 2.1 years. However, variability in time to access was substantial. For one specific medicine, there was an eight years’ gap between the moment the first hospital in the study and the last started treating, prescribing it to patients.
The average time to patient access in the study sample was longest in Hungary and Belgium, followed by Switzerland and the Netherlands. Only one – specialized – hospital (in Switzerland) had all six selected medicines accessible for all selected indications at the time of the study.
'This inequality between patients with the same indication is of course unacceptable,' says Johan van de Gronden, director of the Dutch Cancer Society. 'That's why in 2021 many European cancer societies and the Netherlands Cancer Institute joined forces to form the European Fair Pricing Network, so that we can conduct scientific research to identify the problems and then move the various parties involved to do something about it.'
After EMA approval, a new medicine needs to be approved by national health authorities and included in the national reimbursement list. That's why the researchers also mapped the time between national reimbursement and actual patient access.
The average timespan between national reimbursement was 0.5 years. However, time to access ranged from 6,7 years before national reimbursement to +6,2 years after reimbursement.
‘Differences in access based on countries’ healthcare systems were to be expected,’ says research leader Wim van Harten. ‘But we had not expected the extent of the observed heterogeneity within and across countries, in view of all having access to the underlying information. The differences between specialist and general hospitals may partly be explained by referral guidelines. But it is also known that older patients and those with lower socioeconomic status will be less easily referred to a specialized or academic hospital.’
The study showed that in more than half of the cases first patient access took place in the context of early access programs (EAPs) or off-label use of medicines that are already on the market for other indications. This can lead to unequal access because the prevalence of early access programs (EAPs) varies significantly across and within countries.
These EAPs are mainly requested by specialized hospitals, which, for example, have conducted research on such a new drug and already have many contacts with pharmacists through their clinical trials. Sometimes care is also deliberately concentrated in a few centers to build up experience with the drug. EAPs are often financed by the pharmaceutical companies involved.
‘Early access programs play an important role in granting access to medicines with great benefit for patients in high medical need,’ says research leader Wim van Harten. ‘However, it is important that EAPs are put in place for the right medicines. EAPs for medicines with limited evidence and uncertainty regarding its effectiveness could increase the safety risks for patients. To regulate and harmonize EAPs, and at the same time collect real world evidence, the Drug Access Protocol that has been put in place in the Netherlands, has been an important step.
Main barriers for timely and equal patient access The researchers distinguished a number of factors that may be responsible for delays in access across countries and hospitals. ‘It would be too easy to blame individual actors, such as governments or pharma,’ says Wim van Harten. ‘Our research shows that all parties involved have to do better.’
- Pharmaceutical companies tend to launch a new medicine faster in bigger and richer European pharmaceutical markets such as Italy, France, and the Netherlands, including through EAPs. This leads to delays for patients elsewhere in Europe.
- Due to high medicine prices, negotiations between national health authorities and pharmaceutical companies result in time-consuming processes that hinder patient access. The Transparency Directive (Council Directive 89/102/EEC) limit of 180 days to decide for pricing and reimbursement is often not honored.
- Countries have different ways of containing their health budgets, such as the costs they accept for a life year gained. Portfolio differences related to quality criteria set by governments or insurances may also have played a role in at least one country in the study.
- Hospitals may have different specializations, budgetary constraints, or strategic priorities.