There are 6,000-8,000 rare diseases
At least 75% of rare diseases affect children
As the focus on improving outcomes and the pressure of pay-for-performance increases, survival in the shifting healthcare landscape requires the reshaping of traditional operating models. This reality is even more present and acute in rare and orphan diseases due to the high price of treatments, a small evidence base and often high engagement from patient and caregiver groups. By 2020, the market for rare and orphan disease treatments is estimated to grow to $178 billion – amounting to 20% of the total prescription drug sales.
As a result, pharma companies are increasingly seeking to shift from the traditional role of a supplier of medicines to become a more active, patient-centric partner in healthcare. Although the potential business benefits are extraordinary, this transition from a transactional mindset to a more solutions and services-oriented perspective is not without challenges, both internally and externally.
Drawing on discussions from an expert panel at a big pharma conference in Barcelona PA facilitated, we explore how trust can be built in rare and orphan diseases.
Among others, the panel included:
Building trust in rare and orphan diseases
Rare and orphan diseases offer two significant challenges to the pharma industry. On the clinical side, the biggest challenge of orphan drug development is robust evidence generation due to the inherent difficulties in identifying and diagnosing patients, as well as a lack of validated endpoints. To further fuel this challenge, endpoints have to be accepted by regulators, payers, physicians and patients.
From our discussions, four main areas emerge for the key industry stakeholders to focus on:
The way forward
Trust starts with transparency and delivering this transparency will require more innovative approaches than pharma have traditionally used. Integrated Healthcare Solutions, when they are truly patient-centric, can deliver real value to patients and contribute to the body of evidence that will drive better outcomes and more informed clinical, regulatory and reimbursement decision making.
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