Kielan Arblastar, our Policy Involvement Assistant, discusses the implications of the US Government’s Right to Try bill
On May 30 2018, President Donald Trump signed a bill which will give terminally ill patients the right to try clinical treatments not yet approved by the US Government.
The Bill prescribes patients with the right to try investigational drugs which only require approval from their physician and the drug manufacturer.
Quicker access to new clinical treatments is fundamental for improved survival rates for brain tumour patients. Only 25% of patients diagnosed with a glioblastoma, the most aggressive and common high-grade brain tumour, are expected to survive past a year and only 5% will live past five years.
The announcement could offer hope for brain tumour patients who have exhausted all other treatment options, empowering them to choose potentially life-saving drugs which have yet to secure regulatory approval.
How it works
The Right to Try Bill (RTT) attempts to circumvent the Food and Drug Administration’s (FDA) application process, where prescription of experimental drugs is only processed under “compassionate use”.
It will safeguard both companies and doctors from legal challenges derived from prescribing unapproved treatments to patients.
Historically, in order for treatment drugs to be approved by the FDA, drugs must go through a series of clinical trial phases.
Firstly, a drug’s manufacturer must demonstrate it is safe for human use whereby trials can have as little as 30 patients as test subjects.
30% of all clinical drug treatments do not move to the next trial stage.
The second phase tests how effective the drug treatment is for the condition it is designed for. It is of paramount importance this stage phases out drugs which have severe or permanent side-effects.
Only an apparent 33% of drugs reach the last stage, where drug manufacturers must test the product’s viability on 300 to 3,000 patients. This last trial process may take up to four years.
There is disagreement over the Bill’s necessity, however. The FDA approved 99% of patient applications for access to clinical drug treatments since 2009, where requests are usually granted within a matter of a few days.
Proponents of RTT argue, however, the FDA’s compassionate use exceptions are only granted to a fraction of those who need it – 1,200 patients a year – because the application process is expensive, time-consuming and complicated.
If there is a promising treatment available, it can take more than 10 years for it to pass the clinical trial process and become available for patients.
Is it the UK’s turn?
Given both RTT’s potential advantages and shortcomings, is it time for the UK to have the right to try?
The UK already has something similar to RTT in place already – the Accelerated Access Pathway (AAC).
From April 2018, five medical devices or drugs will be selected for a fast-track process each year, meaning clinical drug treatments will be more readily available for uptake on the NHS.
The hope is twofold; it is intended to help provide clinical treatments for diseases such as dementia and cancer but also to put the UK on the trial location map.
One pressing issue is the accelerated process will only apply to the NHS and not the regulatory parameters surrounding new drug treatments.
There will still be in place a lengthy process of securing regulatory approval from either the European Medicines Agency or the UK Medicines and Healthcare products Regulatory Agency, both of which require the completion of a clinical trial process for safety and efficacy purposes.
Whether the UK should decide to press on with the AAC or develop a new initiative of accelerated clinical drug treatments remains a debate to be had, but time is limited – the NHS and regulatory bodies must weigh up the harm to patients from unexpected side-effects versus the ever pressing need to implement access to potentially life-saving treatment.
What is clear is that patient voice should be at the centre of any debate on this.
Image courtesy of righttotry.org