Reshaping clinical trials in the age of personalised treatment


Personalised healthcare is at the core of everything we do, and this approach directly informs the design of our clinical trials.

Colorectal cancer (CRC) is one of the most common cancers worldwide, with approximately 1.3 million people diagnosed with this disease every year. It is most common in developed countries where lifestyles tend to be less active and rates of obesity are higher, and there also seems to be an increasing incidence of CRC in rapidly developing countries, such as China, India and Brazil. These facts combined mean that CRC remains a key global healthcare challenge.

Unfortunately, due to its ambiguous symptoms, many patients are diagnosed at a late stage, when the cancer has already spread (metastasised) to other parts of the body. At this point, the cancer is more difficult to treat and the prognosis for patients is significantly poorer.

Fortunately, the treatment of metastatic CRC has advanced significantly over past decades, with new diagnostic tests and treatment options available, resulting in improved management of the disease. Patients can now expect to live significantly longer than they would have only a decade ago. Treatment of metastatic CRC today generally involves a combination of different therapies to attack the tumour from various angles, including surgery, chemotherapy and targeted therapies.

Like many other types of cancer, we know that CRC is a highly heterogeneous disease. This means that even if two people are diagnosed with CRC, their tumours will not behave in the same way. Understanding this crucial point is important from the overall treatment viewpoint, and is particularly important when considering the use of some of the targeted therapies available to patients. One person may benefit from a certain targeted therapy, whereas another person may not, which has a real impact on providing personalised patient care.

We now understand that cancers are actually made up of multiple subtypes driven by different genetic mutations at the molecular level. Even if patients’ tumours look the same under the microscope, the molecular alterations that lead to these cancers may be very different. This knowledge is driving a real change in the way tumours are diagnosed and treated.
Thomas Buechele, Head of Global Medical Affairs, Oncology

Helping match treatments to patients’ tumours

As we are learn more and more about the biology of cancer, we use this invaluable information to continually inform and optimise the design of our innovative and patient-centric clinical trials. One such trial, MODUL, is hoping to provide patients with metastatic CRC with improved outcomes through a highly personalised treatment approach.

In the trial, patients’ tumours are analysed for biomarkers and the results of this testing help define which treatment might be most beneficial to them. For instance, the MODUL trial is currently evaluating a novel drug combination in patients with tumours in which a protein called BRAF is mutated. In patients whose tumours are not BRAF mutated, in contrast, the trial is assessing immunotherapy and a current standard-of-care combination.


A key feature of the MODUL trial is the ‘adaptable’ design that allows more effective implementation of novel biomarker-targeted treatments. As such, by the end of 2016 two new treatment groups will be opened. In one of the new groups, the patients with amplified HER2 tumours will receive a novel combination of HER2-targeted agents, whereas the other cohort will study a novel immunotherapy-containing combination. In all groups, the experimental treatment is directly compared to the current standard of care and it is hoped that the patients who receive innovative treatment approaches will have improved outcomes.

It is envisioned that the trial will continue to evolve over time to allow speedy implementation of novel biomarker-driven groups. For patients participating in the trial it means that very latest treatments tailored to their specific tumour molecular profile could be available.

Educating patients and informing our approach to tackle CRC

Helping patients understand their disease and supporting them as they take part in a clinical trial is a Roche commitment to putting patients first and personalising patient care. In order to help patients navigate their journey within the MODUL trial, a comprehensive patient website is available for patients to understand what CRC is, what treatments they might receive, how biomarker testing is carried out and what their results mean for their journey.

In addition to helping direct targeted therapy choices, analysing patients’ tumour samples is hugely informative to our overall understanding of colorectal cancer and hence also to our ongoing research and development programme. It may help us better understand how and why colorectal cancer acts differently in different people, develop or improve tests that aid the detection of colorectal cancer, and increase our understanding of which patients benefit from which treatment, amongst many other possibilities.

Ultimately, the same level of innovation that is used in the development of pioneering new medicines also needs to be applied to the way we trial those medicines in patients participating in our clinical trials. The MODUL trial is one example of how we are looking to do just that at Roche.

Tags: Personalised Healthcare