Digital therapeutics are on the rise and have reached another milestone with the introduction of the DiGA Fast Track in Germany. However, the requirements for the admission in the DiGA directory are high and clinical evidence in form of a study plays a crucial role in proving the healthcare promoting effects of the application.
Product evidence is not only needed for the DiGA admission itself but also a key success factor within the German healthcare system, especially when it comes to convincing doctors, patients, and potential collaboration partners of your solution. That’s why manufacturers should always take into account that the evidence provided in the form of a meaningful study, in addition to a positive approval decision, will have an impact on following price negotiations as well as marketing and sales activities. Therefore, it is even more important to always think one or more steps ahead when planning your DiGA study.
The requirements for a clinical study are described in the DiGA guidelines, but are supplemented by preferences and lived practice of the BfArM. For example, retrospective comparative studies, case-control studies, retrospective cohort studies or intraindividual comparisons are possible design options according to the guideline. Nevertheless, the randomized controlled trial (RCT) has established itself as the gold standard for providing evidence in the past years. Additionally, the size of the study population and the intervention period of the so far published studies are similar and based on practical experiences. For the comparison groups of the study, the options of standard care or non-treatment are mostly used. Only very rarely, control apps (e.g. with limited functionality) are the comparison tool of choice when it comes to providing evidence for a DiGA.
The decision on the study design should therefore be well-considered – even before the application is submitted or the study is started. In the best case, the study design should be coordinated with the BfArM from the beginning of the process. Therefore, the following crucial aspects should be taken into account:
- Objectives: It is important to clearly define the positive effects of care that are aimed to be demonstrated before the start of the study. The DiGA Fast Track offers two different ways to demonstrate those effects: a) The medical benefit or b) the positive structural and procedural improvement (pSVV) of the digital solution. The medical benefits refer to the classic endpoints such as improvement in health status or quality of life, as well as a reduction in disease duration. The pSVVs represent a new form of evidence and include endpoints such as an improvement in health literacy, patient sovereignty or adherence. Experience shows that the majority of DiGAs consider evidence of medical benefits. The only exception so far is the oncology DiGA Cankado, which is evaluating only the improvement of health literacy in its current clinical trial. In theory, a combination of medical benefits and pSVVs is also conceivable.
- Patient population: Suitable patient groups are a crucial success factor of your study. In this context, subgroups should not be disregarded, as experience has shown that significance must also be demonstrated in e.g., male and female subgroups. This requirements should be considered, especially with regard to the size of the study population.
- Study design: Some lessons learned from practice can already be taken into account when it comes to the design of the study. For DiGA manufacturers, there is no way around a randomized controlled trial for successful admission or retention in the directory after the trial phase. This type of study can be supplemented by further evidence such as meta-analyses. There are also initial indications that, after successful admission, real world data can be used to enable application to further disease patterns or patient groups.
- Time schedule: Realistic time schedules are particularly important for preliminary listed DiGAs. According to the DiGA Fast Track, manufacturers have 12 months to conduct their study and submit the results to the BfArM. That this is a small timeframe becomes clear when looking at the preliminary admissions to date: Even after more than two years of DiGA Fast Track, no manufacturer has managed to complete its trial phase within 12 months. Most of them had to extend their trial phase by 3-12 months to successfully submit their results. However, there is no guarantee of an extension, as the negative decisions of the diabetes DiGA ESYSTA and stroke DiGA Rehappy illustrate.
Our experience at Flying Health shows clearly: a comprehensive and early analysis of the requirements and potentials of a study can have a decisive impact on the future success of a DiGA. Not only when it comes to the DiGA Fast Track, but also for potential investors and customer groups. The concept of your study should therefore always be bullet-proof before taking up the challenge of the demanding DiGA admission process.