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Novel endpoints in the digital age

Redesigning the goalposts

Novel endpoints

Technology is changing how we define success in drug research according to experts who say the digital age is facilitating use of novel endpoints in trials.

Every clinical trial has an endpoint, a measure used to determine if the drug in question provides therapeutic benefit. And all endpoints are a reflection of patient response. From obvious examples like survival to metrics like quality of life, endpoints have, and will always be, focused on the patient.

In recent years, however, the assessment of endpoints and the types of endpoints used has started to change. For example, while traditional patient-focused goals like symptom reduction are still used, the manner and the level of detail in which they are measured has changed.

In addition, advances in digital monitoring technologies are allowing more protocols to include novel endpoints that previously could not be measured or were considered too burdensome to the patient.

Industry’s desire for greater patient-centricity is driving the change, according to Jean Paty, vice president of patient-centred endpoints at IQVIA.

“Capturing the patient experience with both disease and treatment and integrating that feedback into trial design is essential for the biopharma industry.

“This direct-from-patient data through novel endpoints allows researchers to address patients’ concerns about their disease experience, which can increase the likelihood of drug approval and reimbursement, allowing patients access to life- saving therapies.”

Dr Paty added: “Novel endpoints may also add differentiating value for sponsors, who can use this data to shape their drug discovery and development strategies, win regulatory support and engage with healthcare providers.”

Novel regulations

Regulators have also welcomed the pharmaceutical industry’s willingness to assess therapeutic benefit using new technologies and novel endpoints.

A European Medicines Agency spokesperson said: “Novel endpoints have the potential to be more precise and even more inclusive, meaning patients who would otherwise have been unwilling to enrol in a trial may now choose to do so.

“They could also aid in retaining patients in a trial if the novel endpoints reduce the burden on patients, compared to previously invasive endpoints.

“These qualities of novel endpoints would lead to more efficient development, with shorter trials or with fewer participants required, and the trial results may be more robust and generalisable.”

Despite these potential benefits, the EMA says drug companies should not underestimate the work involved in using a novel endpoint.

The spokesperson said, “For novel endpoints to be established and be useful for regulatory decision-making, they need dedicated scientific development activities and evidence from being tested in a controlled trial.

“There is no perfect overlap between clinical assessments used for guiding the treatment of a patient in a clinical care setting with those that can be used for regulatory purposes to measure safety, efficacy and clinical benefit of a medicine in a trial. These are some of the reasons why we offer support for such dedicated development activities through scientific advice for novel methodologies.”

The EMA’s support is provided via its initiative for qualification of novel methodologies, which is designed to aid drug firms with protocol design.

One key consideration all developers should keep in mind is that even the most innovative trial endpoint is only useful if the data generated can be processed effectively.

“For all novel endpoints it is important that the methods used to analyse them lead to robust conclusions, and the development of novel endpoints should always be done with consideration as to how they are going to be analysed.”


Greater patient-centricity is facilitated by digital monitoring technology, according to Pamela Tenaerts, executive director of the CTTI (Clinical Trials Transformation Initiative).

“When endpoints are gathered from participants in clinical trials, assessments typically take place in healthcare settings rather than in the context of patients’ daily lives. In addition to being limited in terms of their objectivity, these measures also may capture only brief ‘snapshots’ of the participant’s functionality and/or disease burden at a given point in time.

“Mobile technologies, however, offer new ways to capture objective measurements as clinical trial participants go about their daily lives. They also reduce barriers to participation, thereby making more generalisable, patient-centric assessments possible.”

The CTTI is a US public-private partnership focused on helping industry make trials more efficient and of higher quality. Encouraging and supporting the appropriate use of novel endpoints is part of this effort.

Tenaerts cited a CTTI case study outlining the case for developing novel endpoints generated using mobile technology in Parkinson’s disease (PD) as an example.

“It explains that the field of PD is well positioned to develop a novel endpoint that, either alone or as part of a basket of endpoints, improves upon existing gold standard PD endpoints.

“’Number of episodes and total duration of bothersome tremor’ is an example of a measure that could address unmet need for the assessment of an aspect of PD and realise the benefits of passive, continuous objective data capture in this population,” she added.

A closer look

New technologies are also being used to look at established endpoints in a novel way, according to Jean Paty from IQVIA.

“Pain has been historically measured using a 0-10 scale at specific times of a day, across an entire day, and sometimes across
a week or more at a time. New technologies are allowing us to measure pain at different points for that patient to get a better sense of the full picture.

“Imagine a patient has an accelerometer that’s monitoring heart rate or blood pressure and it detects something irregular. You can then ask the patient for their pain level at that time. This is a novel endpoint because you’ve captured data about the person’s pain level in a completely new way and can potentially tie it to a specific setting in ways that we have not been able to do previously.“

Gareth Macdonald is a journalist specialising in the life sciences industry

Article by
Gareth Macdonald

16th December 2019

Article by
Gareth Macdonald

16th December 2019

From: Research



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