How Patient Engagement Can Improve Patient-Access – a real-life case study

Meaningful patient-industry collaboration supports society to obtain insights and evidence on the burden and severity of the disease for use in patient advocacy and inclusion in regulatory and HTA processes. For example, gathering patient-reported outcomes (PROs) on the respective patient-relevant unmet need addressed through surveys/technology/wearables can highly illuminate the disease burden.

 

In one case so far, the PROs admedicum collected among patients via our specific survey methodology made the basis for an EMA decision to maintain the orphan drug status of a highly patient-relevant treatment for myotonia.

 

 

Another way patients can be involved is by involving patient experts for co-creation of clinical trial design. Expert patients or patient advisory boards can significantly help define the most patient-relevant endpoints and should be consulted before the trial begins. They help define the clinical trial strategy in regards to primary and secondary endpoint according to patients’ relevance and benefit.

 

Furthermore, these endpoints need to be calibrated and validated on patients’ capacities, and not only what established tests can measure.

 

Patient engagement in HTA also means designing the measurement of patient-reported outcomes (PROs) subject to patients’ priorities, including:

  • Adverse events
  • Treatment adherence
  • Treatment satisfaction
  • Patient preferences
  • Health-related quality of life

 

By emphasizing the importance of endpoints through the measurement of patient preferences, e.g. on health-related quality of life, specific endpoints like fewer adverse events could be higher within the HTA assessment process.

 

Finally, patients can be involved in HTA by establishing a network with other patient organizations for the disease and obtaining their statements on patient relevance for sending to the HTA agencies.

 

Looking for assistance in HTA with your company? Here is how admedicum can help.

 

 

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