Early access schemes
PHARMAC has strong interest in funding promising new medicines with emerging evidence (so-called ‘early access’), but the timing and extent of such decisions are always a careful judgement.
While recognising the challenges faced by individual patients, and their understandable desire to try new treatments, our job is to look at all the evidence and make a decision that is in the interests of all New Zealanders.
Clinical evidence is a key input into PHARMAC’s funding decisions, and can take many forms. Ideally the funding applications PHARMAC reviews should be supported by long-term data that establish the long-term benefit of a treatment.
PHARMAC sometimes receives funding applications that haven’t developed a full (and in some cases sufficient) evidence-base to support claims of people being cured or living longer. When this occurs, we have to consider the benefits of funding a treatment with high uncertainty about its results, with the reality that it would take funding away from other more proven treatments.
In the context of new medicines with emerging evidence, some countries run ‘early access’ schemes, an initiative that some people periodically suggest should operate in New Zealand. The idea behind such schemes is to allow funding for a new or promising medicine based on what the early research is showing.
However, even evidence of the best quality requires careful consideration of its application to decision-making. A meta-analysis(external link) showed that results of first trials that present large treatment effects often dissipate as new evidence accrues.
In addition to considerable policy assessment work internationally, there have been a number of international commentaries(external link) that have discussed such schemes, and which provide good material to inform ongoing consideration of the right pharmaceutical funding policies for New Zealand. Relevant issues include:
- when early access schemes are ring-fenced, how the boundaries for those schemes are defined, and whether it is possible to have effective and durable definitions of what should be ‘in’ and ‘out’ (such as having a dividing line between preventative and therapeutic treatments)
- how promising a new treatment is relative to existing treatment options, and potentially other new treatment options
- whether, when thinking of all people, earlier access to medicines for one group when evidence is just emerging (which is what early access schemes provide) is better than providing earlier access to different medicines for other groups (where the evidence is stronger)
- perception risks of what might be seen as speculative investments in health treatments versus applying funding to treatments with more chances of success (and, when considering all patients, potentially choosing the most speculative of all possible uses of public money available for providing earlier access to medicines)
- the incentives created on suppliers of medicines with regards:
- generating evidence of efficacy if a lower evidence standard is acceptable (such as them having less incentive to conduct clinical trials with acceptable quality)
- their pricing of medicines for a targeted pool of funding, and
- the shifting of, in effect, the risks and costs of product development – ordinarily borne by companies to prove the products and services they offer – on to another party (in this case, a funder of medicines)
- the ability to sustain operation of a stand-alone scheme for new (early access) medicines if better health outcomes could potentially be achieved from using the funding in other ways
- whether or not an early access investment can be easily changed (increased, reduced or stopped) based on improved evidence.
PHARMAC understands that patients, their families and whānau, and clinicians ideally want the newest medicines in the hope they will provide the best possible health result. PHARMAC, too, wants to invest in new medicines to improve the range and efficacy of medicines available to New Zealanders.
When it comes to ‘early access’ where evidence is still emerging, PHARMAC needs to take a careful and robust approach. This includes being open-minded to new funding arrangements, provided these have been shown to be better for New Zealand’s health outcomes over the long term.
In the USA, the Food and Drug Administration (FDA) has a fast-track scheme for drugs to enable early regulatory approval. Similarly in the United Kingdom, the Cancer Drugs Fund (CDF) was established to give patients access to medicines that don’t meet the cost-effectiveness thresholds used by the National Institute for Health and Care Excellence when assessing medicines.
There are various articles that detail their experience with early access schemes:
- Cancer Drugs Approved on the Basis of a Surrogate End Point and Subsequent Overall Survival: An Analysis of 5 Years of US Food and Drug Administration Approvals(external link)
- NHS increases budget for cancer drugs fund from £280 million in 2014/15 to an expected £340 million in 2015/16(external link)
- The Cancer Drugs Fund - Benign or malignant?(external link)
Other early market entry approaches used by the industry include product familiarisation programmes (PFPs):
Analysis from the European Medicines Agency's approvals for cancer drugs between 2009-13 showed most entered the market without evidence of benefit or survival gain. More than three years afterwards, there was still no conclusive evidence that lives were extended or improved for most cancer indications, and when they did, these were often marginal.
- Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13(external link)
More information on trials and evidence
- Testing treatments(external link)
- Maintaining confidentiality of interim data to enhance trial integrity and credibility(external link)
Last updated: 5 November 2017