Fair Pricing for Rare Diseases

Director , Syenza


Welcome back, in this update we continue with a review of the WHO’s 4th Fair Pricing Forum, a key event focused on making medications and treatments affordable and accessible to everyone. In this update, we’re reviewing takeaways from parallel session 6 on fair pricing for rare diseases which we recently attended.

The UK’s Innovative Approach

Let’s start by focusing on the UK’s approach to addressing the challenge of pricing and financing medications for rare diseases. It’s a demonstration of ingenuity and adaptability. First off, the UK’s shift from an end-of-life criterion to incorporating a severity modifier in their pricing strategy is nothing short of a masterstroke. Why? It reflects a deep understanding of the nuanced nature of rare diseases. The severity modifiers, with multipliers ranging from 1.2 to 1.7, push the willingness to pay per quality-adjusted life year to between £35,000 and £51,000. This isn’t just about opening the purse strings wider; it’s about recognizing the profound impact of severe conditions on patients’ lives and adjusting the economic model accordingly.

Managed Access Agreements and Economic Flexibility

Managed access agreements are used routinely for diseases between payers and pharmaceutical companies. They allow for the collection of real-world evidence in the context of uncertainty. Yet, they place a heavy burden on healthcare professionals, time-wise. One can’t help but wonder, is the quest for data turning healthcare providers into data entry clerks? The flexibility in the cost-effectiveness threshold for ultra-rare conditions is also noteworthy, with the willingness to pay threshold soaring to between £100,000 and £300,000 per quality-adjusted life year.

Financial Strategies and Challenges

Then there’s the financial firepower through the Cancer Drugs Fund and the Innovative Medicines Fund, each budgeting an additional £340 million annually. It sounds impressive, but is it enough to keep pace with the ever-increasing costs of new treatments? Or are we just plastering over the cracks in a system struggling to keep up with the rapid advancements in innovation?

Malaysia and India: Fair Pricing for Rare Diseases

Now, let’s glance over at Malaysia with its Rare Disease Trust Fund and India’s digital portal for crowdfunding. Malaysia started the Rare Diseases Trust Fund in 2021 but it begs the question – why is there a need for such a fund in the first place similar to the UK? And India’s approach to crowdfunding and voluntary donations for patients with rare diseases is a reminder that civil society and businesses can play a direct role in financing different treatments. India is also considering removing custom tax and sales tax from imported medicines for rare diseases.

Conclusion: A Continuous Journey

In conclusion, the UK’s innovative approaches to drug pricing and financing for rare diseases, from severity modifiers to managed access agreements, show a willingness to adapt and explore new strategies. Yet, beneath the surface of these sophisticated models lies an uncomfortable truth – the struggle to balance innovation, affordability, and access is far from over. Navigate this landscape is a complex task, one thing becomes clear: the journey towards truly fair drug pricing is a marathon, not a sprint.