I have recently updated the clinical trials section of our website with a couple of MND clinical trials that are currently recruiting. This has led me to think about the adaptive licensing discussions that have been taking place recently and how this relates to these MND clinical trials.
Adaptive licensing is an idea. An idea that has seen increasing media attention over recent months and years. Adaptive licensing aims to see the licensing of drugs somewhat earlier than they currently are at present, particularly with regards to those living with diseases like MND. We, the MND Association, therefore encourage further exploration into the idea of adaptive licensing including as to how it may work.
The current situation
In order for a drug to be licensed in the UK it needs to have been shown to be both safe and beneficial by means of a clinical trial. Clinical trials are considered the ‘Gold standard’ for drug testing in humans and consist of four main parts:
- Phase I Testing the safety of a drug for the first time in healthy people
- Phase II Testing the optimal dose, safety and tolerability of a drug in people living with the disease
- Phase III Testing if a drug is effective (beneficial) at treating a larger group of people living with the disease
- Phase IV Testing and monitoring a drug (including side effects) once it has been licensed for use
After phase III testing the drug, and all of its clinical trials data, is reviewed by the appropriate licensing body before the drug can be licensed for use. If a license is given, then data will be continuously collected over a longer period of time, which is known as phase IV testing.
It is important to know that some drugs, which show promise in the lab, are shown not to be effective in a large phase III clinical trial (particularly with diseases like MND). This is why clinical trials are needed. Drugs, which are not beneficial or may have harmless side effects, need to be fully tested so they are not given to people unnecessarily.
These strict guidelines for clinical trials are in place to protect patients and to ensure that people living with MND are not given treatments that may be harmful or offer no benefit. For more information about clinical trials see our information sheet
The adaptive licensing idea
The European Medicines Agency (the European drug licensing body) describes adaptive licensing as a system that, “seeks to maximise the positive impact of new drugs on public health by balancing timely access for patients with the need to provide adequate evolving information on benefits and harms.”
Clinical trials take time and the aims of adaptive licensing are for ‘more drugs to be available to more people and more quickly’ which we heartily agree with.
But, we do not know how an adaptive licensing approach may work. As it is still currently just an ‘idea’ there are lots of questions that need to be answered. Including; who would administer the drug, and how would their effects be monitored? Who would be responsible – the doctor or the drug company? Would there be a ‘control or placebo group’? What would happen to clinical trials?
This is why the Association encourages answers to these questions by further exploration into the ideas of adaptive licensing including as to how it may work.
Our campaigns team have written a series of blog posts, which explain more about adaptive licensing and what it means for people living with MND;
- Adaptive licensing and the MND Association’s position An overview of how a model of adaptive licensing might help to get more drugs to people with serious illnesses like MND more quickly.
- Recent developments in legislation and policy This post explores the various policy initiatives, including adaptive licensing, that have been set up to try to get new medicines to patients earlier.
- Challenges of adaptive licensing, and implications for people with MND This post looks at some of the unresolved issues that must be addressed before we know whether adaptive licensing will provide some of the answers we would like to see.
Progress in MND research
Our understanding of MND has progressed immensely over recent years. Twenty years ago we only knew one of the genes (known as SOD1) behind the rare inherited form of MND. Today, we now know 12 of them. With research into MND growing more and more every year we are hopeful that this research will lead to the likelihood of new drugs and treatments.
While we agree with calls for more drugs to be available to more people and more quickly, achieving this in practice is not easy – if it was, it would have been done by now.
This is why the Association encourages further exploration into the idea of adaptive licensing.
I think tthis is wonderful news and very much overdue. If something which has demonstrated efficacy in the lab and safety in phase 1 could be made available to patients willing to sign disclaimers that would be a major breakthrough. If it has proved safe then a patient may ultimately be disappointed but at least gets a chance to treat the illness possibly 2 to 3 years sooner than might otherwise be the case which could well, with mnd, prove to be the difference between life and death.
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