PD Research to clinic/The route to market
In a 2011 article Michael Kelly described  how few Parkinson’s drugs make it through the clinical trials pipeline. Arguably all that the last ten years have brought is improvements to existing treatments, this despite all the breakthroughs in understanding and possible treatment avenues that have opened up. It is salutary to reflect that levodopa, often referred to as the gold standard treatment for Parkinson’s, has been available for forty years.
Delays in getting new drugs to market are particularly relevant to people with Parkinson’s. The degenerative nature of the condition means that every year that passes without effective treatments to stop or slow the progression is a year lost. In time, as people with the condition can attest, many of today’s drugs become part of the problem rather than part of the solution. In the opening session of the Worldwide Parkinson’s research congress in Glasgow in 2010 Bryn Williams spoke about ‘the fierce urgency of now’ in the search for a cure. Where will this urgency come from?
Action for improving the process
Drug repositioning – the use of drugs developed for one purpose being used to treat another – is one opportunity. An example is Exendin-4, a drug developed for the treatment of diabetes that is currently being tested as a Parkinson’s medication. The fact that a drug has already been through the clinical trials process means that it can be tested relatively easily as a treatment for another condition and, if successful, deployed comparatively quickly. Exciting though this is it may be unrealistic to expect that existing drugs will be able to modify cells in the complex way that the latest research suggests will be necessary.
Although no-one wants to take risks with unknown drugs there is a growing feeling that the current clinical trials procedures are overly bureaucratic and can actually prevent the development of new drugs. Pharmaceutical companies may conclude that there is no point devoting the time and money to clinical trials when they have to wait such a long time to receive any payback. There are signs that the problem has at least been recognised. Cancer Research UK has pointed out that since the Clinical Trials Directive came into effect in 2004 the UK’s share of trials across the world has dropped from 6% to 2%.The fact that Pfizer has closed its research and development facility in Kent is another illustration of the problem, and the sort of thing that makes Government take notice. The pharmaceuticals industry is very important to the UK economy and anything that drives work away is to be taken seriously.
The response in the Strategy for UK Life Sciences is encouraging. One of the key actions is for the Medicines and Healthcare products Regulatory Agency (MHRA) to “bring forward proposals for a new ‘Early Access Scheme’ to increase the speed and efficiency of routes to market approval for innovative, breakthrough therapies.” This initiative is to be welcomed, and could potentially offer access to new drugs before they have been formally licensed. Unfortunately the scheme that was proposed is rather unambitious, envisaging that only one or two medicines might qualify each year. Furthermore, it would only reduce the time to market by about a year. A public consultation on this proposal has taken place and the next steps are awaited. A panel of experts has been tasked to come up with recommendations.
In the US the Food and Drug Administration (FDA) regulates the testing of new medicines and the same criticisms apply to the time taken to get drugs to market. Here a more radical approach has been mooted. This would involve patients taking a greater risk by using new drugs while they are in development, but only after full disclosure of all the risks and benefits. Here again Parkinson’s patients may be more willing to contemplate this strategy when they weigh up the risks against the knowledge that their condition will deteriorate over time. While this approach does have its merits it would be difficult to implement as it may contravene international agreements about the ethics of conducting medical research among human subjects.
What can be done to encourage the development of new drugs and get them to market quickly? It is crucial to take this opportunity to influence the decision makers who are reviewing the clinical trials process. This underlines the importance of the campaigning work that organisations like Parkinson’s UK and the Cure Parkinson’s Trust carry out. Keeping abreast of what is going on in the UK and Europe and lobbying to influence legislation is critically important. The Parkinson’s community is not alone here and can work with other charities that have an interest in speeding up the route to market for new drugs.
Raising awareness of clinical trials
In the short term a more achievable objective is to raise awareness of, and participation in, clinical trials. This is something that all the neurological charities that have an interest in research are working on. In this respect a welcome development from the National Institute for Health Research (NIHR) is the Clinical Trials Gateway website. This enables patients to identify trials that are taking place in their area, together with the recruitment criteria, and enables them to make contact with the research team. Initiatives such as this, together with a simplification of the clinical trials process, should enable more treatments to get to market in less time. That is what we all want.
Collins, Francis S. “Reengineering Translational Science: The Time Is Right.” Science Translational Medicine 3, no. 90 (July 6, 2011): 90cm17. http://stm.sciencemag.org/content/3/90/90cm17.full
- Kelly, Michael: “Why do so many drugs fail at the last hurdle? Is there a serious procedural flaw in drug research?”, Spring Times, April 2011. File details:
- Williams, Bryn: Opening ceremony, World Parkinson Congress, Glasgow, September 2010.
- Adams, Stephen: “EU Red Tape Making Drugs trials impossible” Daily Telegraph, February 13th, 2012.
- “Strategy for UK Life Sciences” published by the Department for Business Innovation and Skills, December 2011.
- MHRA website: http://www.mhra.gov.uk/Howweregulate/Medicines/MISGNewTechnologiesAdvisoryPanel/Earlieraccesstonewmedicinesintheuk/index.htm
- Madden, Bartley: “More Choices, Better Health – Free to choose experimental drugs” published by The Heartland Institute, Chicago, 2008.
- Clinical Trials Gateway website http://www.ukctg.nihr.ac.uk/default.aspx