Originally appeared in Internal Medicine Journal of the Royal Australasian College of Physicians 2013, published by The Authors
Co-funded expanded access programmes for new oncology drugs: creating a two-tier system for Australian cancer patients?
We read with interest the recent article by Tie and Gibbs in the Journal that describes utilisation of the cetuximab Interim Access Programme (IAP) in Australian patients with colorectal cancer.1 Patients enrolled in this programme were initially required to self-fund cetuximab (phase I) but thereafter received treatment free of charge (phase II). A large number of patients (479) was funded in phase II of the IAP, and we applaud Merck Serono for supplying these patients with cetuximab before it attracted a Pharmaceutical Benefits Scheme (PBS) reimbursement. However, an analysis of the demographics of patients enrolled in this programme is most revealing. Tie and Gibbs note that Australians treated in public hospitals and from lower socioeconomic backgrounds and regional areas were markedly underrepresented in the IAP. In the absence of a more plausible explanation, we would assume that many of these patients were unable to enrol on the cetuximab IAP as they did not have the financial capacity to self-fund phase I of the programme.
With this in mind, we note the ever increasing numbers of Australian drug access programmes that require patient copayments. For instance, patients with metastatic renal cell cancer can access the mammalian target of rapamycin inhibitor temsirolimus through the Pfizer EMBRACE programme if they fund alternate 4-weekly cycles. Similarly, women with locally advanced or metastatic breast cancer can access the oestrogen receptor antagonist fulvestrant through an Astra Zeneca access programme that requires 3 months of treatment to be patient funded. We also note that in a forthcoming Janssen access programme, men with metastatic prostate cancer will be able to receive treatment with the novel hormonal agent abiraterone provided that they fund alternate monthly cycles. None of these agents is currently covered on the PBS, and based on the findings of the cetuximab IAP, we remain very concerned about the potential for inequitable enrolment to also occur on these access programmes.
In highlighting this situation, we are not advocating that patients with sufficient financial resources be denied the opportunity to pay for drugs that are Therapeutic Goods Administration-approved but not PBSreimbursed. Nor are we denying or discouraging the generosity of pharmaceutical companies for assisting some Australians to gain access to highly expensive drugs that prolong median overall survival in randomised phase III studies.2–4 However, because of the requirement for partial self-funding, the aforementioned expanded access programmes may have unwittingly contributed to the development of a two-tier pharmaceutical system that selectively favours Australians of higher socioeconomic
status. We believe that this is not consistent with Australia’s proud history of providing universal access to healthcare through programmes such as Medicare and the PBS. Therefore, we implore governmental and regulatory bodies to ensure that in the future, all Australian cancer patients are given a ‘fair go’ by instituting measures that help all Australians to access novel oncology drugs in expanded access programmes.
1 Tie J, Gibbs P. Treatment with unfunded drugs in oncology: the impact of access programmes and clinical trials. Intern Med J 2013; 43: 23–31.
2 de Bono JS, Logothetis CJ, Molina A, Fizazi K, North S, Chu L et al. Abiraterone and increased survival in metastatic prostate cancer. N Engl J Med 2011; 364: 1995–2005.
3 Karapetis CS, Khambata-Ford S, Jonker DJ, O’Callaghan CJ, Tu D, Tebbutt NC et al. K-ras mutations and benefit from cetuximab in advanced colorectal cancer. N Engl J Med 2008; 359: 1757–65.
4 Hudes G, Carducci M, Tomczak P, Dutcher J, Figlin R, Kapoor A et al. Temsirolimus, interferon alfa, or both for advanced renal-cell carcinoma. N Engl J Med 2007; 356: 2271–81.