In rare diseases, true patient engagement is critical to every step of a drug’s development, from clinical trials and regulatory approval, to patient diagnosis and adherence. Why is that? Because patients with rare diseases (and their families) are essentially the thought-leaders of their condition. Usually these patients are very few in number, and have high unmet needs. Management of their condition is often fragmented, with knowledgeable healthcare professionals few and far between.
Putting the patient at the centre is an absolute necessity for pharma companies involved in this space. The key is involving patients as early as possbile throughout all relevant functions wihin the organisation throughout the life cycle, from early R&D, clinical development, approval & market access, and commercialisation & life cycle management.
Patient engagement is not only suitable for rare diseases. And with the patient now moving towards the centre of the value equation, pharma companies have little choice but to embrace a more patient-centric approach. Although patient engagement fits very naturally with rare diseases, there are lessons that big pharma can apply to their more ‘mainstream’ disease areas. The following four steps are integral to succcess:
- Adopt a (compliant) strategy of listening and learning
- Be inclusive and eengaged and be in it for the long haul
- Provide support beyond the pill
- Organise around your stakeholders
