PARIS - Ensuring equal access to the latest cancer medicines is one of the most pressing challenges facing OECD health systems today. Despite the emergence of new oncology drugs, disparities in patient access – particularly through clinical trials and early access programs – remain a critical issue.
Rising costs are also straining even the most affluent healthcare systems, making affordability a concern for all.
This paper examines various aspects of inequalities in access to cancer medicines, covering key stages in a medicine's life cycle, from marketing authorisation to reimbursement decisions and uptake in clinical practice.
Executive Summary
Inequalities inpatient access to cancer medicines and ensuring access to new cancer medicines are paramount challenges for healthcare systems. This working paper focuses on various aspects of inequalities in access to cancer medicines, covering the key stages along the life cycle of a medicine. The analysis draws mainly on findings of the section of the 2023 OECD Policy Survey on Cancer Care Performance relating to cancer medicines.
While more oncology medicines are being developed and approved, access inequalities persist
The number of approved cancer medicines has steadily increased over time.Between 2004 and 2011, the average annual number of approvals was close to four in the European Union and European Economic Area (EU/EEA)whereas 2021 and 2022 saw the approvals of 17 and 15 new cancer medicines, respectively.
In addition, the number of approvals of extensions to new indications of existing medicines outnumbered the number of approvals of new medicines in recent years.The research pipeline in oncology signals that this development will probably continue in the near future.
Clinical trials and early access schemes offer patients an opportunity to receive new cancer medicines before regulatory approval and/or reimbursement approval.
However, the number of clinical trials in oncology ranges from fewer than 2 trials to more than 10 trials per 100000 inhabitants across the EU, with lower access in Eastern and Central European countries. Many countries use early access systems to improve access to novel cancer medicines despite limited/immature evidence on efficacy.
Countries with early access schemes most often provide access on a named-patient basis, which typically only benefits a small share of clinically eligible patients.
Pricing and reimbursement arrangements significantly impact access to oncology medicines
In recent years, the rising prices of cancer medicines have led to increased spending on cancer care, posing affordability challenges even for affluent countries with publicly funded health systems.
The budget impact of new medicines has become a significant factor in public coverage and reimbursement decisions, with most countries indicating its growing importance due to higher drug prices and the increasing number of new medicines. This makes it difficult for some countries to provide fast access to oncology medicines.
In addition, many products come to the market with limited/immature evidence on efficacy and cost-effectiveness.
Policies such as managed entry agreements(MEAs)to address uncertainty in coverage and/or pricing decisions for new cancer medicines/indications are growing in importance.
The reimbursement of cancer medicines with a high clinical benefit varies substantially. Of a sample of indications with high clinical benefit in breast and lung cancer, Germany reported that all indications were covered, followed by the Netherlands (92%),and Bulgaria and Sweden (both 85%).
Malta reimbursed no indications and Cyprus and Latvia reported small proportions of indications covered (both 31%). The time from European-wide marketing authorisation until national reimbursement/coverage ranged from around 100 days or less in Germany and Sweden to over three years in Cyprus, Latvia, and Lithuania.
The reimbursement of a companion diagnostic is not coupled to the reimbursement of a matching medicine in most countries. This can lead to a paradoxical situation in which a medicine is reimbursed but not its companion diagnostic, necessitating patients to pay out-of-pocket or rely on funding from pharmaceutical companies.
The lack of a joint reimbursement process for a medicine and its companion diagnostic may slow down the uptake of “personalised medicine” in clinical practice.
Policies to encourage the utilisation of generics and biosimilars vary across countries.
Reference price systems for both generics and biosimilars are in place in a majority of countries, which can contribute to securing lower prices.The adoption of generics and biosimilars in clinical practice might be somewhat hampered by policies that do not allow or encourage substitution for reference medicines.
A majority of countries do not mandate substitution for generics and few countries mandate it for biosimilars. This might deprive countries of achieving efficiencies in the rational use of medicines and sustaining medicine budgets.
Several policy options are available to aid payers and national competent authorities in enhancing patient access to cancer medicines
Healthcare systems need to weigh the costs from investing in cancer medicines against the potential improvements in patient outcomes. An increased focus on spending on effective and cost-effective medicines should be considered.
A formal health technology assessment (HTA), assessing both effectiveness and costs, should be conducted for all new medicines and extensions to new indications.
In addition,the assessment of new medicines with a potentially high relative clinical benefit could be expedited. Not all new medicines are equally effective.
Value frameworks, such as the ESMO-Magnitude of Clinical Benefit Scale, have been developed to support the process of HTA and to assist in rationalising reimbursement decisions. Managed Entry Agreements (MEAs)may help patients to gain faster access to new cancer medicines despite limited/immature evidence on efficacy. Performance-based MEAs may seem more appealing because of their ability to link health outcomes to payments.
However, their routine implementation for a wide range of medicines/indications is hampered by the additional administrative burden and/or lack of appropriate IT systems and staff. Functional systems where relevant data can be easily extracted from medical records and processed by healthcare payers would be needed.
Purely financial MEAs might be the only feasible option for most countries and the majority of cancer medicines in the foreseeable future.Addressing potential barriers that impede patient access to already reimbursed medicines and new cancer medicines is vital to enhance the quality of care.
This includes, amongst others, ensuring adequate public budgets for reimbursed medicines, joint reimbursement decision of a medicine with its companion diagnostic, regular update of local clinical guidelines and protocols, continuous training of clinical staff, and adequate infrastructure and staff for diagnostic testing and treatment administration.
Early access schemes using a named-patient approach create administrative burden to handle cases on a one-on-one basis and may only benefit well-informed patients or patients treated by certain physicians. A switch to a population-based system could be explored as it could contribute to reducing inequalities in access.
Increasing efficiency in the use of cancer medicines deserves greater attention. More effective measures are needed to stimulate competition between producers of generics and biosimilars and to control prices of reference medicines after loss of market exclusivity.
This may create substantial budget headroom, which can be reinvested to increase access to new cancer medicines
To download the full paper, visit: https://www.oecd.org/content/dam/oecd/en/publications/reports/2024/09/access-to-oncology-medicines-in-eu-and-oecd-countries_6cf189fe/c263c014-en.pdf
