Watch Prof Bowen Video on MDS Clinical Trials
In the second video of this series, Professor David Bowen, Consultant Haematologist at St James University Hospital, Leeds, explains why clinical trials are key in the development of new treatments for MDS.
Prof Bowen is an honorary professor of Myeloid Leukemia Studies and Consultant Hematologist at St. James’s Institute of Oncology, Leeds, in the United Kingdom.
He is chief and co-investigator for several national MDS clinical trials and a member of the NICE Appraisal Committee evaluating cost-effectiveness of newly licensed drugs for potential use within the UK NHS. He also co-chairs the Steering Committee of the EUMDS Registry Trial.
Why are clinical trials needed?
We usually do Phase III clinical trials where we are testing a new drug against whatever is considered to be standard or a dummy - a placebo.
The only way that we can assess whether a drug is effective is to work out whether it works, whether it does what we hope it would do, while at the same time not having too many toxic effects.
The typical situation in that context, in Phase III trials, is that the drug has modest exposure, in terms of the numbers of patients, and it looks promising, in terms of the fact that it works and it appears to be safe. But until you expose a lot more people to it, you don't know how effective it's going to be, nor how safe it's going to be.
And so you need to explain to the patient that just because it looks like a drug is going to be effective in the first instance, it does not necessarily mean to say that when larger numbers of patients are treated, it will be both effective and safe. And therefore, testing it against a dummy drug is a logical thing to do.
Currently recruiting MDS clinical trials
"Most of the patients are suitable for at least the Registry Clinical Trial"
We are a major centre for MDS research, so we are very focused on clinical trials here in Leeds. Most of our patients are suitable for at least the Registry Clinical Trial.
The Registry is essentially an opportunity to collect information about every patient's type of MDS and to use that information going forward, to document how these patients' MDS develops, what sort of treatment they have and what their response is to treatment.
That is a trial, a Registry Trial, and it is very important as it generates data that we can call "real world data" or "community based data" to record information about patients who are not in clinical trials of new drugs, but are nevertheless managed using conventional treatments.
We need to know what the outcome for patients in day-to-day practice is so that we can make a comparison between that and treatments that are developed in therapeutic trials.