Andrew Mumford
Chief Executive Officer, Initiate Consultancy Limited
Securing funding approval for a rare disease treatment can pose unique challenges. Andrew Mumford, head of an agency supporting pharmaceutical companies throughout this process, discusses these issues and how to address them.
What are the main challenges in gaining approval for rare disease treatments?
Rare disease treatments often have an ‘evidence gap,’ where the small population of patients means there is scarce data or evidence regarding a drug’s safety and efficacy. This poses unique challenges to making informed decisions on whether or not to approve a drug and may force pharmaceutical companies and regulatory agencies to compromise on their preferred processes and outcomes.
What consequences does this evidence gap pose for patients?
Delays to drug approval greatly impact patients waiting for treatment access. This is a particularly acute issue in the rare disease space, as many of these diseases are life-limiting and have no approved therapeutic options.
What specific evidence gaps are there in the rare disease space?
Although the evidence gap is a problem for all aspects of the approval process, it is especially troublesome when it comes to economic data. Rare disease treatments are typically expensive due to research, development, and manufacturing costs.
Additionally, data on their costs to the healthcare system and long-term impact on patient quality of life is often sparse. Consequently, demonstrating a drug’s cost-effectiveness can be difficult, and healthcare systems may be reluctant to ‘reimburse’ (pay for) them.
Pharmaceutical companies’ collaboration with
patient groups, as well as with healthcare providers,
policymakers and specialist agencies,
is crucial to the approval process
How important is collaboration to gaining treatment approval?
The perspectives of patients and their families shed light on unmet needs and can help to impress the urgency of a drug’s availability. As such, pharmaceutical companies’ collaboration with patient groups, as well as with healthcare providers, policymakers and specialist agencies, is crucial to the approval process.
What does the future look like for rare disease treatments?
The pharmaceutical community is investing more time and money into solving evidence-related issues. Advancements in precision medicine, gene therapies, AI and real-time data collection will help fill the evidence gap. However, it is only by collaborating with stakeholders and expert solution-finders that optimal outcomes can be achieved for rare disease patients.
