Saturday, 10 September 2016

CTAP - what's the big deal?

Earlier this week the Cystic Fibrosis Trust announced its Clinical Trials Acceleration Platform (CTAP), a new initiative to increase capacity for CF clinical research in the UK and make it easier for people with CF to find out about and take part in research if they choose to do so.
    
This is an initiative I have been working on with the CF Trust for the last 3 years because I believe passionately that the current system for doing CF clinical research in the UK is inadequate (and not really a system at all) and that people with CF deserve better. Many people living with CF, CF consultants and others who do research agree and want to help build a better system. 

I am very excited to see CTAP moving forward but it won’t work unless the whole UK CF community comes together to make it work. I hope you all take a moment to find out about CTAP and think about what you might be able to do to help make it a reality for the benefit of those of who live with CF today and future generations.

As we know, research is needed to evaluate the safety and efficacy of new medicines, to compare existing treatment options to make sure we are using them as effectively as possible and to improve the quality of clinical care. Getting involved in research is also a clear statement that we will not settle for the status quo.

Taking part in clinical research, either as a participant in a specific clinical trial or by giving input into the design and running of better quality, more relevant, more user-friendly clinical trials through Patient & Public Involvement (PPI), is something we can all do to really make a difference. 

Today clinical trials are only generally available to those people with CF who happen to be at certain centres. These tend to be the larger CF centres but everyone with CF deserves the option of taking part in clinical trials and we should all have easy access to the information we need to make informed decisions about participating.

This is why the need for action is URGENT:

·         Despite huge advances over the last 50 years, people with CF are still dying far too young;
·     The lives of people with CF and their families are still severely impacted by the disease and the burden of treatment;
·         We are reaching a critical juncture with numerous new therapies requiring evaluation;
·         A bottleneck is emerging where the US clinical trials network and large CF centres in the UK are having to turn new trials away because they are already saturated and have no spare capacity;
·         We do have the European Clinical Trial Network and 7 of our UK CF centres are members of that but, despite a clear collective interest, there is no coherent system for CF clinical research in the UK. We have lots of CF centres, other groups, individual researchers and others doing great things but they are largely doing their own thing. There is no central co-ordination or management. This results in inefficiency and an overall situation in which resources are wasted despite the best intentions of the very dedicated and talented individuals who are involved in CF research in the UK;
·         Global interest in CF research is currently high as a result of Ivacaftor showing: a) that it is actually possible to restore CFTR function; and b) that when CFTR function is restored, the health of people with CF improves in a way that significantly improves health outcomes and actually makes people feel better;
·         While this level of Pharma interest exists, it is essential that we organise ourselves as a community to make the most of the pipeline and translate it into maximum benefit for people living with CF. If we don't do it, Pharma interest will fade away and the pipeline will collapse under its own weight.
CTAP seeks to address all these things and create a system for UK CF research which can help relieve the global bottleneck, push new drugs through the pipeline and give people living with CF in the UK a better chance to access them.

At the moment, only about 1 in 10 people with CF in the UK has taken part in a clinical study or trial. Only 55 patients in the UK (out of 1,100 globally) took part in the Phase 3 trials for Orkambi. Based on the number of people with CF in the UK, you would expect at least twice as many UK patients to be in the trials like this. If we can go from 10% of people with CF taking part in studies/trials to 20%, it will have a very significant impact.   

I also believe that a more effective system for CF clinical trials will help to reduce drug development timelines and boost competition which will reduce Pharma’s pricing power and make it easier for us to access new therapies in the clinic.    

While new treatments are exciting it is also really important to make sure we understand the evidence for existing therapies and optimal combinations of therapies (such as IV antibiotics) and know that we are using them as effectively as we possibly can. If we don’t do this, we will be accepting that people with CF will suffer unnecessarily and we will not be in a position to capitalise on new therapies, which will only work properly when there is a strong foundation in terms of the standard of care. Luckily, CTAP is looking to help make these kinds of studies easier too.

It is really important that people living with CF help to shape and manage CTAP to make sure it fulfils its potential. Please register your interest by clicking on the links below. As CTAP takes shape, you will also be able to help by volunteering for specific clinical studies/trials, getting involved in PPI and just talking to your CF team about opportunities to take part in clinical research. Don't wait to be asked. If your CF centre does not do research, please ask them why.  

Here are two really important PPI roles to consider applying for:

PPI Coordinator (closes 12th Sept 2016): CTAP PPI Coordinator Role
PPI Advisor (closes 30th Sept 2016): CTAP PPI Advisor Role 

Here is some more information on CTAP: Announcement of CTAP

Here is where you can register your interest in being informed of developments and potentially getting involved in the future: Register your interest

Please check it all out and get involved if you can.