Dr Chris Jackson: Cancer sufferers in dark over drug

Successful overseas trials of Pembrolizumab in fighting melanoma have increased calls for the drug to be funded in New Zealand. Photo / Getty Images

Opinion

New Zealand has the highest rate of melanoma in the world yet we have no effective therapy for advanced melanoma. Pharmac's decision to not fund the "wonder drug" branded Keytruda makes me wonder: has Pharmac made the right decision?

Everyone familiar with the drug, properly called Pembrolizumab, agrees it is effective. The survival rate for patients after one year is around 70 per cent when treated with this drug, or its near identical unregistered competitor Nivolumab, compared to 25 per cent first-year survival for untreated patients.

It is also comparatively safe. Every cancer drug has side effects, but the government's drug safety agency, Medsafe, says the benefits outweigh the risk. Yes, it is affordable. Pharmac does not spend its entire budget, returning funds to other health sector services, so almost certainly has enough money.

Even without long-term data, the results are sufficiently compelling to convince funders in the UK, Canada, Australia and many other countries to make treatment publicly available. The question is whether it is effective enough to justify the high cost, compared to other priorities?

We don't know if Pembrolizumab is good value for money because Pharmac won't divulge cost effectiveness, citing commercial sensitivity. The UK's evaluation agency releases limited cost-effectiveness analysis, while some Canadian reviews are released in full. Professor Tony Blakely said the cost effectiveness calculations stack up compared to other decisions. Having some indication of cost-effectiveness and comparisons to other products would lead to a more informed debate.

Along with cost effectiveness, Pharmac doesn't list its priorities so we don't know how Pembrolizumab ranks against other drugs. We are asked to trust Pharmac with no shared information even when its decisions differ from those of similar countries.

We have no effective treatment for melanoma. This offends our Kiwi sense of fairness and has led many to call for politicians to step in. But we don't want politicians deciding what drugs to fund - there are other solutions.

Drug effectiveness should be considered separately to cost. We should be told the outcomes of this, otherwise confused debates about effectiveness will continue. We could implement a "drug scorecard" using a validated scale like the European Society of Medical Oncology's Magnitude of Clinical Benefit scale - this would give more transparency and lead to a greater sense of fairness. Without this, claims of a "wonder drug" can go unchallenged.

We need goal posts for funding. This could be a set cost-effectiveness point, otherwise we would risk writing drug companies a blank cheque. It may be reasonable that the budget could grow if additional health gains are "bought". If a drug falls short, then there would be little grounds for complaint.

With no end in sight, we need agreed timeframes for assessing funding applications. Patients are forced to weigh-up whether to foot the bill for treatment themselves or wait for a Pharmac decision with no guarantee of a positive outcome.

Some areas are missing out - we need to recognise that. Fairness is important, as well as raw incremental cost-effectiveness ratios.

Where there is early but compelling data for an area of high unmet need, such as melanoma, a new category of "breakthrough therapy" could permit temporary funding to be granted with the collection of use, effectiveness and toxicity data. It would better inform decision-making while allowing patients, who would otherwise have nothing, to receive treatment. To the best of my knowledge, Pharmac has not done this before.

We need to account for the savings drugs can bring - patients may avoid the need for further radiotherapy or surgery if drugs work well. A period where we collected local data would help accurately inform the true cost and benefit of a drug in our entire health care system.

A temporary funding category would also give time for the near-identical drug, Nivolumab, to enter the market, and Pharmac could use that to lever the price down. This would mean greater cost initially, but bigger savings in the long run.

Currently patients feel that their voices are not being heard by those deciding their fate. Without transparency and accountability, they are left feeling cheated.

Pharmac is good, but not perfect. Without change, we are doomed to have this debate again.

Dr Chris Jackson is medical director of the Cancer Society of New Zealand.