It can take around 15 years from when a new drug is discovered in the lab before your doctor can prescribe it. The length of time and failure rate associated with this process means that developing new drugs costs hundreds of millions of pounds.
And, more importantly, the protracted process of drug approval means that unless patients are enrolled on a clinical trial, they may miss out on getting a treatment that could have extended life or improved its quality.
For this reason, the research community and the government want to speed up getting new drugs to patients with life-threatening diseases like late-stage cancer.
New drugs faster
As part of its Life Sciences Strategy, in 2011 the government asked for feedback on an ‘Early Access scheme’.
Their idea is to develop a system that allows certain groups of patients to access experimental drugs before they are licensed for wider use.
You can read the public consultation documents about the scheme here.
While the scheme offers an opportunity for a limited number of treatments to be offered slightly sooner, we don’t think it will do enough to get more drugs to patients much earlier.
Instead we’re looking for other ways to do this, such as ‘adaptive licensing’, which would operate throughout Europe.
We’ve been involved in the government led Expert Group that was set up to look at both an Early Access scheme and adaptive licensing. This group formed in 2012 as a result of David Cameron’s pledge to investigate access to drugs.
The Expert Group recently published a report making several recommendations to the government that would allow patients to access new treatments more quickly.
Let’s take a look at the findings in this report and some of the unanswered questions about how such drug access schemes would work.
What happens now?
First though, a whistle-stop tour of the system: the existing process for developing a new treatment means the medicine goes through four tiers of clinical trials that gather data about the safety and effectiveness of a new drug.
It can take up to two years for regulators to check the data and license the drug, allowing it to be prescribed. Following this there is an assessment to determine whether the drug is cost effective. In England this assessment is made by NICE. And in England patients can get access to certain treatments before NICE approval through the Cancer Drugs Fund.
The Early Access scheme proposed by the government would allow UK patients with little or no other treatment options to be prescribed a drug once its clinical trials had been completed but before being formally licensed.
This would mean patients could have certain medicines around a year earlier.
Early Access scheme: far enough?
It’s a tricky balance to ensure that we have enough information on the benefits and risks of a drug while giving patients speedy access to potentially effective treatments. We’re glad that serious attention is being given to this challenge.
But there are unanswered questions. Most importantly, who would pay for treatments being provided through an Early Access scheme? This has been a sticking point in discussions by the government.
The Expert Group supports an Early Access scheme, and has urged the government to launch it as soon as possible.
Although the scheme could allow patients to have some treatments slightly sooner, we don’t think it goes far enough to enable promising drugs to get to patients at a much earlier stage. But we do think this could be achieved through adaptive licensing.
Broadly this means licensing a drug early in its development for a specific group of patients and/or treatment settings. The drug’s safety and effectiveness is closely monitored throughout its development and used to adapt the drug’s license.
Adaptive licensing could offer more options to patients over the Early Access scheme because it gives them the chance of promising treatments at an earlier stage in development, before every phase of the clinical trial is complete.
The Expert Group believes that existing flexibilities in European law support the idea of adaptive licensing. But there’s little awareness, especially among smaller companies and academics, about how to use such flexibilities.
The group has recommended that the agency responsible for regulating all medicines in the UK, the Medicines and Healthcare products Regulatory Authority (MHRA), should communicate these legal flexibilities to industry to ensure that they are used.
But we still think more can be done.
New regulatory processes to enable adaptive licensing are being explored by the European Medicines Agency (EMA), the agency that licenses medicines across all EU member states. This could involve the development of new or revised European laws, a process that can be very lengthy.
The Expert Group supports the EMA’s development of adaptive licensing and has recommended that the MHRA ensures UK-based companies are involved in these developments.
We’ve repeatedly urged the UK government to support the EMA’s efforts to bring about an adaptive licensing scheme. We hope with the Expert Group’s recommendations that we will now see a firm commitment from the government to do so.
What does this mean for cancer patients?
Both schemes could benefit many cancer patients; especially those who have rarer cancers when treatment options can be extremely limited. But it’s important to consider that prescribing medicines that are not fully licensed, and therefore not fully assessed, could pose serious risks to patients.
So it’s essential that patients give full informed consent and are monitored closely throughout their treatment. And it’s vital that there is a clear process in place to allow regulators to stop patients accessing a treatment if they later learn that the risks appear to outweigh the benefits.
From talking with cancer patients we know there is a strong desire to try new innovative drugs and to take on the potential risks to a greater extent than many regulatory agencies realise.
That’s why we think the government should support efforts to bring about adaptive licensing in the UK.
Hollie Chandler is a policy advisor at Cancer Research UK