A rare disease, a first in virtual clinical trials

How do you test a new medicine for a rare cancer when recruiting patients into the clinical trial will take many years? Like everything after 2020, go virtual.

Roche has delivered another first in oncology. It’s a novel new approach to clinical trials, a by-design solution to the challenge of getting innovative new medicines to patients with unmet needs. And doing so at a faster pace, turning what used to be a marathon into a relative sprint. The project team designed the trial on a fully virtual basis to essentially “take the trial to the patient”, at home.

One of the challenges in recruiting patients into clinical trials fortherapies is the small number of patients; rare diseases are by nature uncommon in the general population. This was especially demanding for the Roche team - key contributors Elizabeth Theogaraj (Global Regulatory Leader), Walter Bordogna (Clinical Science Leader), Bettyna Brivet-Jones (Operations Program Leader), and Jamie Wingate (Global Studies Leader) - looking to test a new molecule for cancers with a specific genomic alteration that is found in a range of tumor types, but only in about 0.2% of all cancers. This extremely low occurrence would mean screening 25,000 patients in order to enroll 50 patients into the trial, an endeavour with a timeline of over 10 years. Patients and families do not have that time.

“We needed to think differently and the virtual/ decentralised trial approach appeared to us the most efficient means to recruit for the rare disease that we were working on. As a result, Roche is conducting the first fully decentralised, home-based trial in the field of oncology. We started planning before the pandemic” says Elizabeth Theogaraj, Global Regulatory Leader, “but the experience of 2020 has led to a great deal of growing interest from regulators in this approach. The from home possibility makes participation easier for patients, and offers new opportunities that were not available under traditional trial approaches”.

A first in oncology

The resulting trial explores a promising molecule in the indicated disease in asetting, that is to say, regardless of the tumor site in the body.

“It has been important to build confidence with regulators that the remote and virtual basis of the trial does not in any way compromise the rigor of the clinical evidence gathering process”, says Elizabeth. An important first, we hope, of many to come.

The ongoing trial is using the full complement of decentralised trial solutions, including virtual Principal Investigator and Study Coordinator via telemedicine, mobile nurse, specialised courier services, and tumor scans at local oncology facilities. Doing so in oncology is a special milestone as decentralised trials in this demanding disease area have not been attempted to date.

A virtual trial approach removes the geographical barriers and allows the patient to take part in the clinical trial, regardless of where they are located. And the trial is better integrated into their daily routine. “All this reduces patient burden and promotes patient engagement and retention”, adds Elizabeth. The trial is centered on the patient in a very real sense, in their home environment.

This clinical trial innovation opens up the possibility of improved healthcare to many who would otherwise miss out. Also of acceleration in patient recruitment into clinical trials, and faster answers to clinical research questions. While a welcome evolution in general, the decentralised approach offers the potential for greater and easier inclusion in particularly underserved communities and to people with rare diseases.

Watershed 2020 and meeting the rights of patients

Decentralised trial approachesBroader adoption of digital technologies enabled a number of ongoing trials to continue, when they otherwise would have been put on hold. The approach also allowed patients to avoid exposure to COVID-19 risks. The pandemic threw into sharp relief the issues of health inequalities. Making it possible to take the clinical trial to the patient, at home, can be an important enabler in solving challenges of reaching traditionally underserved communities.

“Decentralised trials are about the right of every patient to participate in a clinical trial regardless of where they live”, says Michelle Rohrer, Global Head of Regulatory. “With today’s technology and home care, clinical trials can achieve an inclusive diversity not possible in our traditional clinical trial model”.

From the patient perspective, the virtual approach is an exciting, convenient new option. It will be important for the future to ask patients about their preferences and work to incorporate the key dimensions in the design of the trials. “We need to provide patients an easy and simple experience, where they feel respected and have empathic support with meaningful outcomes. Every patient’s experience is unique and our decentralised trials provide fantastic additional options for many who would not be able to join otherwise”, adds Yvonne Ulrich, Regulatory Business Office Lead.

Seeking answers and the best way to find them

Regulators are increasingly open to innovation in the conduct of clinical trials, especially when we engage in early and proactive discussions and the methodologies are robust. There is openness to novel design and new ways of seeking solutions for drug development: “Health authorities are very open to working with us to explore new and more patient friendly approaches”, says Lada Leyens, Global Regulatory Lead, Clinical Trial Innovation at Roche.

Kaspar Rufibach, a Roche Expert Statistical Scientist, observes that sometimes product development teams are trying to guess what regulators want to see, or look only on what was done in the past. “We should rather take an approach of: ‘Here is a scientific question. What is the best way to answer that scientific question?’ If that involves new methods, let’s not worry because if you have a stringent scientific case, you can defend it in front of every health authority. You will be confident enough that the results based on the applied methodology can be approved.”

Change comes slowly, then fast

2020 highlighted just what’s possible in taking clinical trials to patients. Attitudes around telemedicine have eased significantly over the past year. Pre-pandemic use of telemedicine, even in the US, was as low as 0.5%. By the third quarter of 2020, it was at 30%1 . In Europe, there is enormous variability in the readiness of individual countries to conduct decentralised trials which are often subject to local laws and regulations. Yet the past year brought consolidation in some markets, traction in others This more widespread adoption and openness to the approach will further support the acceptance of decentralised trials and healthcare at home.

The experience through the pandemic has caused significant interest from regulators in decentralised trials. “The feedback from regulators that we have spoken to so far has been really positive, they see this as the future, and they see the value to patients”, says Elizabeth. Innovative clinical trials with a virtual/ decentralised methodology can help us put the patient at the centre, increase diversity, and reduce the time it takes to bring promising new medicines to patients. These benefits, over and above the intrinsic healthcare value of the medicine itself, are well worth striving towards, for patients and for society.

A decentralised approach - also known as distributed, home-based or, siteless trials - is a means of getting the trial to the patient at home versus requiring the patient to come to the major clinical facility of the lead researcher. This is made possible through virtual collaboration between the lead researcher and local medical teams, enabled by digital technologies, and facilitated and monitored through home visits.

A decentralised or virtual protocol (procedural method) is a growing innovation in the conduct of clinical trials. It brings many benefits to patients and society, including less friction in accessing healthcare, more potential for health equity and inclusion in clinical trials, greater potential for the conduct of trials in low prevalence diseases, and ultimately, to lower healthcare costs to society.


  1. Galloway, Scott, Post Corona From Crisis to Opportunity, Transworld Publishers, 2020

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