The World Health Organization’s (WHO) Essential Medicines List (EML) is an important tool for policy makers across member states to guide country-level selection and procurement of essential medicines. A WHO-coordinated international survey conducted at the end of 2021 was the first to evaluate the alignment of EML cancer medicines used by oncologists worldwide, and the extent to which these medicines were accessible in high-income countries, upper-middle-income countries, and low and lower-middle-income countries (LLMICs). Fifteen medicines considered essential by all economic classifications were represented on the EML, despite the fact that only a third of these medicines were universally available in LLMICs, compared to almost 90% for high-income countries (Figure 1).
Despite universal consensus on what constitutes essential cancer treatment, failure to translate this shared understanding into equitable access has highlighted the urgent need for revised policy action and industry cooperation. The latter is particularly important, since LLMICs on their own have struggled to implement relevant frameworks to guide the procurement of cancer medicines, as so pioneered by developed markets in Europe and Australia (Table 1).
Absence of institutionalized HTA for the negotiation of fairer drug prices and discounts
P&R of medicines determined by HTA bodies that assess their cost-effectiveness, clinical differentiation, or budget impact
Limited financial capacity to cover patented medicines in the context of universal coverage
Greater financial capacity to cover reimbursable medicines leading to fewer out-of-pocket expenses
Use of managed entry agreements
No mechanisms to cover drugs whilst managing uncertainty around performance or financial impact
Commonly used, and range from finance-based (price-volume agreements) to performance-based (payment by results)
Specialist cancer drug funding
Budgets are small and constrained to offer specialized funding for cancer drugs
Country-specific funding for new cancer drugs include the CDF (NICE) and the Fund for Innovative Oncological Products (AIFA)
No cross-country policies to facilitate access through centralized HTA or joint price negotiation
Cross-country initiatives established to facilitate pan-European procurement of medicines such as the EUnetHTA and NLF
Table 1. Differences in market access frameworks between LLMICs and developed markets. HTA: Health Technology Assessment; P&R: Pricing & Reimbursement; CDF: Cancer Drugs Fund; NICE: National Institute for Health and Care Excellence; AIFA: Italian Medicines Agency; EUnetHTA: European Network for Health Technology Assessment; NLF: Nordic Pharmaceuticals Forum
As such, differences in frameworks between LLMICs and mature markets have left industry with fewer incentives to launch in developing nations, so much so that then-Bayer CEO Marijn Dekkers admitted that the launch of its renal cell carcinoma medicine Nexavar was not intended to reach patients in LLMICs such as India:
“We did not develop [Nexavar] for the Indian market, let’s be honest. We developed this product for Western patients who can afford this product”.
Such an admission drew vast criticism from patient advocacy groups, which denounced the current biopharmaceutical business model for failing to properly address the issues of patient access beyond the ‘best 10%’ of the world.
Creating an industry-led patient access programme does not mean that it works
The most obvious place to start for industry would be to implement more sustainable pricing models such as equity-based tiered pricing, which aims to adapt the prices of cancer medicines to the purchasing power of consumers in different countries. Novartis Access (NA), a recently established Novartis programme for non-communicable diseases (NCDs) including breast cancer and diabetes, looks to offer EML medicines at a monthly price of just 1 USD to LLMICs, with Kenya the first LLMIC to receive the programme. However, after its first year, researchers from Boston University assessed the impact of NA in Kenya, only to find that the programme has had little-to-no impact on patient access so far.
Novartis’ price reductions were not enough, as limited understanding of Kenya’s healthcare system and pricing guidelines meant that prices for essential cancer medicines were still higher than available treatments, with most NA medicines still considered unavailable for the poorest households. Failure to address contradictions between Kenya’s national EML and standard treatment guidelines (STGs) resulted in limited household uptake of NCD medicines across the NA portfolio. Indeed, NA medicines were only made available at subdistrict hospitals as per Kenya’s EML, and not lower-level facilities despite their role in the management of cancer and other NCDs. The outcomes of NA so far suggest that a functional patient access programme requires far greater knowledge transfer between industry and LLMICs, a lesson that Novartis will surely learn from as NA continues to expand to other LLMICs in Africa and central Asia.
Access to patients in LLMICs requires direct collaboration with stakeholders, starting with the patient
Patient access requires far more industry involvement with stakeholders to understand how much a patient is really able to afford fundamental cancer treatment. This has been exemplified by the use of patient assistance programmes (PAPs), which have been increasingly used by industry to guide the procurement of high-cost specialty drugs in developed markets. Importantly, there is no reason why the use of PAPs cannot be extrapolated to LLMICs for the procurement of essential cancer medicines.
Lessons can be learned from Takeda, which is currently implementing a more sustainable, intra-country tiered pricing strategy for its innovative lymphoma therapy Adcetris, by addressing accessibility on a patient-by-patient basis. Through Takeda’s PAP, patients from LLMICs first apply to participate in a socioeconomic evaluation using a validated Patient Financial Eligibility Tool (PFET) administered by Axios, an independent Takeda-enlisted specialist. Axios then determines the amount of additional support needed for patients to pay their course of Adcetris. Accounting for a number of socioeconomic metrics, the PFET is a versatile tool that can be adapted to each LLMIC’s economic context, and has been subsequently used by Takeda and Axios to facilitate access to Adcetris in several LLMICs, including Thailand, Indonesia, and Kenya.
The best way for pharma companies to learn could be from each other
Industry leaders have so often worked in silos to integrate more sustainable approaches to patient access, but what if there were opportunities to exchange best practices between each other? On the 22nd of May 2022, the Union for International Cancer Control (UICC) announced the launch of the Access to Oncology Medicines (ATOM) Coalition, marking a major step change to global industry cooperation. Led by the UICC and its partners, the ATOM Coalition is a new, international partnership that looks to bring industry together to improve access to essential cancer medicines in LLMICs. The Coalition aims to achieve this in two ways:
In addition to more sustainable pricing models, meeting ATOM’s second objective will likely require the additional implementation of public-private partnership approaches, in order to maintain the capacity of LLMICs to handle essential cancer medicines appropriately:
Most importantly, the Coalition brings together several country-level and global public and private sector organisations currently implementing access programmes in LLMICs to exchange best practices and enhance existing international initiatives. Notable ATOM members that have joined the Coalition so far include the American Society of Clinical Oncology, the UN-backed Medicines Patent Pool, and a list of cancer drugmakers including AstraZeneca, Novartis, Roche, and Sanofi. The establishment of the ATOM Coalition will give industry leaders the chance to better reflect on the efforts of their counterparts, and exchange best practices to facilitate access to essential cancer treatment.
Moving forward, how the Coalition will decide on which LLMICs to cooperate with first, and whether we will see enhanced coordination between industry leaders will be of major interest as the partnership continues to gather momentum in its early stages.