I have been working as a haematologist for more than thirty years in Hospital Universitario La Fe in Valencia in Spain. I’m mainly devoted to the care of patients with myeloid dysplastic syndromes and also lead the bone marrow transplantation unit at our hospital. I also lead in Spain the Spanish Cooperative Group on Myelodysplastic Syndromes that is currently the largest registry on MDS in the world with more than 40,000 patients in our database. I was one of the founding members of the HARMONY alliance and now I am playing in the Executive Committee, I am the co-Chair of the public side, let’s say, of this public-private partnership.
How do you think the HARMONY approach will effect clinical treatment?
Really HARMONY is a win-win project in several aspects because this has one thing that makes HARMONY absolutely different to any other research project in haematology in Europe and in the US. It’s that all the different stakeholders involved in the treatment of patients with haematological malignancies are in the consortium. For every one of the different stakeholders this project has substantial benefits starting, for instance, for the regulatory agencies because when we approached for the first time the European Medicines Agency they told us that a project like this was very well perceived because sometimes, and I would say most of the times, when a pharmaceutical company approaches them in order to get a new drug approved for the treatment of patients with haematological malignancies they have only to trust in the data that were coming from the clinical trial that that specific pharmaceutical company developed. This is because there was no benchmarking available which you can compare with. HARMONY solved this issue; HARMONY we will build a very, very large, big database with high quality data into it and they will have in the coming years a real picture of what is happening with the new drugs when they enter the market, when they are outside the clinical trials. So, for them, this is an advantage.
Also for HTAs, the health regulatory agencies, obviously would like to compare the new innovative drug that is entering the market with the current treatments you have for that particular disorder. Again, HARMONY will allow them to know which is the cost, which is the effectiveness, which are the secondary effects in real life, not in a clinical trial. Because you know the people entering a clinical trial are absolutely different from the people that in real life are receiving treatment.
Coming next to the scientific, this is obvious. We are sure, we are fully sure, that we are going to develop first class science especially in the field of genomics. We think that with this enormous database we are going to build it will be possible to test the potential co-occurrence and its effects on the outcomes of the patients. So for us it’s obviously a project that is going to give us new insights into our knowledge of these malignant disorders. For the pharmaceutical companies obviously there are many advantages. The first is that they think that inside HARMONY with all the different stakeholders in they will be able to reach a consensus on new endpoints, new outcomes, that can substitute the ones that we are using in clinical trials. For instance, in most of our haematological disorders the main clinical outcome that is sought by the repair medicine agencies is overall survival. This implies that you need a lot of years to perform a clinical trial that gives you meaningful results and you are able to demonstrate a clinical benefit over conventional treatment. If we are able to see and to find new biomarkers or new short-term surrogates of overall survival for the different haematological disorders the pharmaceutical companies will be able to have their innovative drugs in the market in a more reasonable time.
Last, but not least, the ones that will benefit most are the patients and one should never forget that HARMONY is a patient-centred alliance. For the patients what we think we are going to get is new innovative treatments in a faster time and knowing in the real world the efficacy and also the clinical benefit and the potential adverse events of these new therapies. So this is the concept of HARMONY, this is the kind of project that we think that will be signalling the future in research in every not haematological or oncological disease but in any disease because if we want to have personalised medicine and efficient medicine and also we are very much worried about the increasing costs of drugs we need to create platforms like HARMONY in the different disorders. In fact, in the US there is the Vice-President, Joe Biden, in the last ASCO meeting addressed this question and said the US oncologists should look to HARMONY because this is the project I would like to have in the US. That is real and I think this time, maybe for the first time, we are in advance than our American colleagues.
What are the main challenges that need to be overcome?
The first that we did not anticipate and is probably one of the major reasons why we are going not as fast as we should is the fact that in Europe we are 27 countries and this implies that for building a database, such a huge database, we need to share data and we should consider in sharing this data two aspects that are crucial, that is privacy and safety of the data of our patients. In the US it’s very easy, it’s very easy because probably they have only one law for all the country. In Europe it is different and, as you know, the regulations at the European level then are transposed to the different European countries. This results in several aspects that differ between countries. So when you want to build this project you have to consider all those differences and make this possible. This is taking much more time than we thought but now I think that we are in the right way because one of the things that we should not forget is that as a pioneer project HARMONY will pave the way to other projects in healthcare that will be developed in Europe and will follow our line. That is why we must be very, very careful and be completely sure that the way that we are assessing data privacy and data safety are the best for the benefit of our patients.
So you’re sharing the expertise gained from HARMONY with other projects of this nature?
Yes, in fact one anecdotal thing is that in the BD4BO, the Big Data for Better Outcome umbrella of projects of the IMI, there is one project that is called coordinating and supporting action that was supposed to give guidance to the different projects that were under the umbrella of this BD4BO project, especially in terms of ethical and legal issues. But we started before them and now it is the opposite, we are in some way giving them the guidance and they are going the way that we are going.
Is there anything you’d like to add?
HARMONY is a big opportunity for everyone and what we should try is to in some way profit from the advances in technology that we have now. There is no human being that is capable of storing the amount of knowledge in medicine that we have, we need machines and we need artificial intelligence. The failure of some systems like Watson, for instance, has been that they have been unable to put together computing and expertise. That’s what in the next future we think that the HARMONY project will be able to achieve. That’s our final goal.