Validation of DTx

Nel dibattito sui metodi di riprova clinica delle DTx, un possibile approccio è considerare il software come "principio attivo" e l'interfaccia utente come "eccipiente" da adattare al contesto socio-culturale dei pazienti

Analyzing data from clinical trials to determine the effectiveness of different therapies and interventions.

In a study just published in The Lancet Regional Health-Europe, a team of Finnish researchers has shown that a smartphone application is three times more effective than using written material in reducing the likelihood of a smoker kicking the habit.
To prove the validity of this solution, the authors conducted a randomized controlled trial on 200 smokers stratified by age, gender and years of smoking, in a 1:1 ratio between experimental arm and standard of care: essentially the gold standard of clinical validation.

However, the need to apply this type of clinical approach to digital therapies is an idea that is not yet universally accepted.

What kind of validation?

Although not all digital health technologies necessarily need clinical trials, digital therapies (DTx), which by definition deliver therapeutic interventions to treat a medical disorder or disease, require medical evidence and real-world results to be validated. However, some authors urge to consider ‘digital medicines’ with a different approach.
For instance, in 2018, an editorial published by The Lancet itself evoked a kind of ‘digital exceptionalism‘, according to which digital medicine should be considered differently from standard medicine and can afford significantly facilitated pathways.

Digital products inherently collect a large amount of data in real time and other methods of evaluation might be more suitable for this field.

The Lancet

An anomaly not limited to this field: in surgery, the ‘difficulty of conducting randomized trials, also due to the strong influence of individual ability’ has long been described, which led to the creation of the IDEAL recommendations, a specific tool for evaluating surgical innovations .

“It is evident,” the editorial concluded, “the need for similar standards, not only for safety and data utilisation, but also for clinical effectiveness and cost-efficiency of digital medicine.

The danger of digital exceptionalism

The author of the editorial, however, did not blindly adhere to the idea of an unconditional approach by clarifying that ‘without a clear framework to differentiate effective digital products from commercial opportunism, companies, clinicians and policymakers will struggle to provide the level of evidence required to realise the potential of digital medicine. The risks of digital medicine, in particular the use of AI in healthcare interventions, are worrying.

Continuing to support digital exceptionalism and not robustly evaluating digital health interventions poses the greatest risk to patients and health systems.”
Many experts share these concerns.

Gualberto Gussoni, President of the RIDE2Med Foundation, states in an article in Makingpharmaindustry, for example, that “we must not forget that we are talking about devices for therapeutic purposes, and it therefore seems reasonable that clinical investigations specific to these products, and carried out in support of their certification and authorization (and perhaps even reimbursement by healthcare services), should allow their efficacy and safety profile to be adequately and uniformly documented, in a manner at least similar to that of drugs.

The excipient in DTx

With a view to suggesting an effective approach to the scientific validation of digital therapies, an article published in Healthcare Informatics Research suggests an interesting analogy with traditional drugs.
The ‘active ingredient‘ of DTx,’ the authors explain, ‘is the software component that shows a therapeutic effect, and for this part it is necessary to use standard clinical evaluation methodologies to validate its efficacy. The ‘excipient’, on the other hand, is the user interface, which maximises the efficacy of the active ingredient.

A special point about DTx products is the fact that they require more active engagement on the part of patients than drugs, which is why the importance of the ‘excipient’ cannot be overlooked: ‘In other words, in order to achieve the desired therapeutic effects, the socio-cultural background of the patients who will be using DTx must be taken into account.

For it to receive global approval, it is necessary to establish global standards for the clinical evaluation of the efficacy of the ‘active ingredients’, while at the same time taking local ‘excipient’ considerations into account’.