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Letters to the Editor
References 1 Korman TM, Turnidge JD, Grayson ML. Vancomycin vintage: my favourite DRESS. Intern Med J 2015; 45: 233–4. 2 Young S, Ojaimi S, Dunckley H, Douglas MW, Kok J, Fulcher DA et al.
Vancomycin-associated drug reaction with eosinophilia and systemic symptoms syndrome. Intern Med J 2014; 44: 694–6. 3 Kardaun SH, Sidoroff A, ValeyrieAllanore L, Halevy S, Davidovici BB, Mockenhaupt M et al. Variability in the
Access to ‘investigational’ cancer drugs: perspective of a trainee The thoughtful article by Lewis et al.1 would have drawn the attention of oncology trainees who face the dilemma of discussing cancer drugs not yet subsidised by the Pharmaceutical Benefit Scheme (PBS) and remain ‘investigational’. These drugs are expensive, accessible only through enrolment into clinical trials open for recruitment or patient access schemes offered by pharmaceutical companies. They can be categorised into two distinctive groups, as Lewis et al.1 eluded to: one, the truly investigational drugs for which the evidence supporting their use was based only on early-phase clinical trials, and the other, the drugs that had been investigated in phase III randomised controlled trials, defining their roles over the current standard of care and may have already gained regulatory approval by the Therapeutics Good Administration (TGA). Nivolumab is one drug that falls to the first group which mandates the ethical considerations discussed by Lewis et al.1 when their compassionate access is first being initiated and later being sought by patients. However, for the second group of drugs, availability of patient access schemes becomes crucial, being the only means to access the drugs for most patients. As an oncology trainee, the lack of access to treatments with a high level of supportive evidence can be frustrating. The nanoparticle albumin-bound paclitaxel Abraxane (Specialised Therapeutics Australia, STA) exemplified such need for patient access schemes. Based on the pivotal phase III MPACT trial that demonstrated a significant improvement in the overall survival (OS) over the standard of care (hazard ratio 0.72; P < 0.001) in the treatment of metastatic pancreatic cancer,2 Abraxane
References 1 Lewis JR, Lipworth W, Kerridge I, Doran E. Dilemmas in the compassionate supply of investigational cancer drugs. Intern Med J 2014; 44: 841–5.
clinical pattern of cutaneous side-effects of drugs with systemic symptoms: does a DRESS syndrome really exist? Br J Dermatol 2007; 156: 609–11.
gained TGA approval in March 2014 but would become PBS-subsidised after 1 November 2014, an 8-month delay. STA has been providing Abraxane on a costsharing scheme to patients who would fulfil the inclusion criteria of the MPACT trial, and after the announcement of confirmed PBS listing in September 2014, STA began supplying Abraxane to patients who met the established PBS criteria to access it free of charge. Trastuzumab emtansine is another example of cancer drugs that has gained TGA approval, but its subsidy is being met only by the cost-sharing scheme. This is again a drug investigated in a phase III clinical trial to have demonstrated a superior OS benefit over the standard of care in the treatment of metastatic breast cancer.3 If no patient access scheme existed, this drug would be financially out of reach for the majority of patients eligible for it. From a personal perspective, the potential loss of opportunity to treat patients with an effective, regulatory-approved cancer drug is at the time being prevented by the presence of compassionate access schemes. While decisions regarding compassionate supply of cancer drugs need to be carefully made, compassionate access schemes have nevertheless an important role in facilitating access to important cancer drugs. The dilemma in the setting where compassionate access to cancer drugs is available then becomes one of whether to discuss it or not, and whether it is justifiable not to discuss it. Received 21 October 2014; accepted 27 November 2014. doi:10.1111/imj.12666
J. C. Kuo Department of Medical Oncology, The Canberra Hospital, Canberra, Australian Capital Territory, Australia
2 Von Hoff DD, Ervin T, Arena FP, Chiorean EG, Infante J, Moore M et al. Increased survival in pancreatic cancer with nab-paclitaxel plus gemcitabine. N Engl J Med 2013; 369: 1691–703.
3 Verma S, Miles D, Gianni L, Krop IE, Welslau M, Baselga J et al. Trastuzumab emtansine for HER2-positive advanced breast cancer. N Engl J Med 2012; 367: 1783–91.
© 2015 Royal Australasian College of Physicians
235
Letters to the Editor
References 1 Korman TM, Turnidge JD, Grayson ML. Vancomycin vintage: my favourite DRESS. Intern Med J 2015; 45: 233–4. 2 Young S, Ojaimi S, Dunckley H, Douglas MW, Kok J, Fulcher DA et al.
Vancomycin-associated drug reaction with eosinophilia and systemic symptoms syndrome. Intern Med J 2014; 44: 694–6. 3 Kardaun SH, Sidoroff A, ValeyrieAllanore L, Halevy S, Davidovici BB, Mockenhaupt M et al. Variability in the
Access to ‘investigational’ cancer drugs: perspective of a trainee The thoughtful article by Lewis et al.1 would have drawn the attention of oncology trainees who face the dilemma of discussing cancer drugs not yet subsidised by the Pharmaceutical Benefit Scheme (PBS) and remain ‘investigational’. These drugs are expensive, accessible only through enrolment into clinical trials open for recruitment or patient access schemes offered by pharmaceutical companies. They can be categorised into two distinctive groups, as Lewis et al.1 eluded to: one, the truly investigational drugs for which the evidence supporting their use was based only on early-phase clinical trials, and the other, the drugs that had been investigated in phase III randomised controlled trials, defining their roles over the current standard of care and may have already gained regulatory approval by the Therapeutics Good Administration (TGA). Nivolumab is one drug that falls to the first group which mandates the ethical considerations discussed by Lewis et al.1 when their compassionate access is first being initiated and later being sought by patients. However, for the second group of drugs, availability of patient access schemes becomes crucial, being the only means to access the drugs for most patients. As an oncology trainee, the lack of access to treatments with a high level of supportive evidence can be frustrating. The nanoparticle albumin-bound paclitaxel Abraxane (Specialised Therapeutics Australia, STA) exemplified such need for patient access schemes. Based on the pivotal phase III MPACT trial that demonstrated a significant improvement in the overall survival (OS) over the standard of care (hazard ratio 0.72; P < 0.001) in the treatment of metastatic pancreatic cancer,2 Abraxane
References 1 Lewis JR, Lipworth W, Kerridge I, Doran E. Dilemmas in the compassionate supply of investigational cancer drugs. Intern Med J 2014; 44: 841–5.
clinical pattern of cutaneous side-effects of drugs with systemic symptoms: does a DRESS syndrome really exist? Br J Dermatol 2007; 156: 609–11.
gained TGA approval in March 2014 but would become PBS-subsidised after 1 November 2014, an 8-month delay. STA has been providing Abraxane on a costsharing scheme to patients who would fulfil the inclusion criteria of the MPACT trial, and after the announcement of confirmed PBS listing in September 2014, STA began supplying Abraxane to patients who met the established PBS criteria to access it free of charge. Trastuzumab emtansine is another example of cancer drugs that has gained TGA approval, but its subsidy is being met only by the cost-sharing scheme. This is again a drug investigated in a phase III clinical trial to have demonstrated a superior OS benefit over the standard of care in the treatment of metastatic breast cancer.3 If no patient access scheme existed, this drug would be financially out of reach for the majority of patients eligible for it. From a personal perspective, the potential loss of opportunity to treat patients with an effective, regulatory-approved cancer drug is at the time being prevented by the presence of compassionate access schemes. While decisions regarding compassionate supply of cancer drugs need to be carefully made, compassionate access schemes have nevertheless an important role in facilitating access to important cancer drugs. The dilemma in the setting where compassionate access to cancer drugs is available then becomes one of whether to discuss it or not, and whether it is justifiable not to discuss it. Received 21 October 2014; accepted 27 November 2014. doi:10.1111/imj.12666
J. C. Kuo Department of Medical Oncology, The Canberra Hospital, Canberra, Australian Capital Territory, Australia
2 Von Hoff DD, Ervin T, Arena FP, Chiorean EG, Infante J, Moore M et al. Increased survival in pancreatic cancer with nab-paclitaxel plus gemcitabine. N Engl J Med 2013; 369: 1691–703.
3 Verma S, Miles D, Gianni L, Krop IE, Welslau M, Baselga J et al. Trastuzumab emtansine for HER2-positive advanced breast cancer. N Engl J Med 2012; 367: 1783–91.
© 2015 Royal Australasian College of Physicians
235
