Reflections on the World EPA Congress

As we are now a month on from attending the World Evidence, Pricing and Access Congress, it has been useful to reflect upon the presentations shared and resulting discussions, in order to ascertain what are the major underlying trends and themes.

One key trend that has been building is the focus on rare diseases, which have moved from the exception to the mainstream focus of many companies. There is a large unmet need with only 3% of rare diseases currently have an approved treatment, but with over 300 million people worldwide.

Developing treatments for rare diseases present a number of challenges around economic sustainability, sometimes limited evidence, trial design, evidence maturity and uncertainties on long term outcomes, price density, value assessment as well as price negotiation, and ultimately the challenge of equity and ethics.

There were a number of presentations, roundtables and panel discussions that explored these challenges, as well as ideas to address them or improve access. We see the following as growing trends:

• Addressing uncertainties through contracting, such as managed entry agreements
• Growth of non RCT data use, and different approaches to RWE, data capture and evidence development
• Sustainability and affordability, and equity concerns
• Initiatives to build on current parallel procedures and consultations

Uncertainties for rare disease treatments, gene therapies will continue and our key take-aways focused on the discussions around managed entry agreements (MEAs):

MEAs come in many forms, and can combine multiple elements such as risk-sharing, outcomes based payments, incorporating value-added services, result-based reviews, and patient access schemes. They help moderate payer uncertainty and aim to allow faster access to new innovative healthcare technologies.

• MEA strategy should be considered and planned for early in development. It is predictable (prior to phase 3), and will also take time to optimise. So start early.
• Early external engagement is important, to ensure that the real problem and uncertainties are being focused on, and that any solutions can be implemented. Do not forget to build in infrastructure and practicalities.
• MEAs have to be considered as pan – country. Alignment of affiliate teams and needs, together with an assessment of each country’s gaps should be considered. There is an initiative within EU for joint commercial agreements that will change the landscape. Increasing joint consultations are likely.
• Follow through on commitments, and build trust for the long term.
• Keep it simple, success will lie in how both parties ensure that the key components, including easily trackable and objective endpoints, how any agreement is managed and updated. As the initial raft of previously agreed MEAs draws to a close, it will be interesting to see how schemes are simplified.
• Data, evidence and the increasing use of registries as a key factor in tracking outcomes, and use of registries for such access schemes. Expect to see key initiatives like DARWIN EU (catalogue of RWE and data sources) making a difference as well as developments of the Autorisation Temporaire d’Utilisation (ATU) in France.
• Flexibility on pricing, and equity issues will become an increasingly important debate. With an increasing push for price transparency. Of note were:

The achievable price is highly variable. For example, in China, a 95% discount was required for Spinraza to achieve NRDL inclusion.

Of the major seven markets, confidential discounts were proposed to be at their highest in the UK, whereas Germany has no hidden discount.

Our conference team - David Alderson, George Hale, Abbey Child and Mark Orchard

Central to all these trends is the rapidly evolving gene therapy space with Zolgensma performing ?very well commercially, Luxturna received highly positive appraisals in key EU markets, and discussions of ?ex-vivo vs in-vivo gene therapy, with the more personalised process of the former being noted as increasing complexity and cost. With cost and complexity being a key issue when it comes to viability (as seen with Zynteglo).

#marketaccess #pharma #rarediseases #managedentryagreements