Impact of Patient Engagement on Market Access and Services
admedicum is pleased to announce a blog series on topics in patient engagement relevant to those in the pharmaceutical industry. This is the fifth part of the series.
Part 5: Impact of Patient Engagement on Market Access and Services
The impact of patient engagement on market access
Patient involvement is also of great importance for the marketing authorization and benefit-risk assessment procedure. In some jurisdictions, patient involvement is an indispensable part of the market access procedure (e.g. usability tests for drug/device combinations). As an example, the demonstration of a patient-relevant benefit in early assessment according to German law § 35b SGB V is a prerequisite for subsequent market access in Germany. The German Institute for Quality and Efficiency in Health Care (IQWiG) assessing such patient benefit has defined patient relevance in its methodology guidelines.
If the demonstration of additional benefit has already been designed and implemented "by the patient" right from the start, it significantly strengthens the case in favor of genuine patient-relevant innovation. This is especially true for assessment procedures where patient representatives are regularly involved, such as with FDA, EMA, NICE, and GBA.
It is important to involve people who are personally affected, be they patients or relatives (“subject-specific patient representatives”), to take into account those benefit dimensions that are not currently sufficiently recognized. Examples are oral liquid drug formulations for drugs that are currently only available in solid dosage forms for diseases associated with severe swallowing difficulties, or the importance of even small functional improvements like better finger control in neuromuscular disorders that help to control the joystick of a wheelchair.
The widespread use of mobile devices among society, and of course patients, offers an ever-increasing opportunity to identify and realistically record patient-reported outcome criteria, (PROs) in a way we would have never anticipated 10 years ago. These can be collected in conventional study settings, particularly in randomized controlled trials (RCTs), but also in "real-world settings". The FDA has recently provided an open-source platform for digital applications in clinical development as guidance for companies.
Digital data collection can significantly strengthen the evidence-base of a drug. This is also true when product modifications have an influence on long-term therapy adherence or long-term outcomes. This can be important for e.g. changes in the dosage form or the frequency of application, or for the fixed combination of drugs in complex pharmacotherapy.
Beyond-the-pill services: Offers beyond the medicinal product
More and more pharmaceutical companies are recognizing that, in addition to the "hardware" of the product itself, it is necessary to offer services related to the drug in order to make its application safer, more effective and more patient-friendly. Such offers beyond the drug may range from information on the disease to assist in coping with disease-related limitations. They may also consist of patient support programs including such programs that involve third parties, e.g. home therapy providers, to assist in the use of the drug. Additional medical technology products offered against payment can also be part of these services. These kinds of services are often referred to as "beyond-the-pill services".
What these services have in common is that they will only have a patient impact if they provide solutions to real problems. This can only be achieved if a detailed understanding of the patient journey (treatment path) and different patient typologies is given. Such understanding cannot be obtained if companies try to work only with physicians (whose role remains important) since physicians don’t live with the disease and often cannot know or judge why certain decisions are made by patients in daily life. The patient journey and what is relevant to them can only be systematically determined when it is done together with affected patients.