This week our team attended the World EPA Congress in Amsterdam, reporting on the key discussions and trends coming out of the congress. Here, Jake James, Analyst and Eleanor Butler, Consultant, recap on the three day meeting.

Day one – Tuesday 23 March

Transforming healthcare delivery with data and evidence

Representatives from AstraZeneca discussed the global burden of long-term conditions in the context of utilising data to transform the delivery of healthcare.

50% of disease burden comes from 5 key long-term conditions; Asthma; COPD; Heart failure; T2D; CKD disease and variation in global care further adds to this. A focus should also be placed on making relevant clinical data public and granular to aid medical decision making. Further barriers to care include long timelines associated with clinical guideline implementation. Remote patient services and monitoring (“value-added services”), can act to significantly reduce disease burden and patient mortality however, there is a need for an in depth understanding of the care pathway to capitalise on the potential benefits of such systems. Furthermore, such services provide the opportunity for additional feedback loops enabling manufacturers (and payers) to access greater levels of data to inform R&D.

Key note panel: Navigating the Market access, RWE and pricing landscape in a pandemic: overcoming challenges, embracing opportunities and looking to the future

Representatives from Bayer, J&J, AstraZeneca, NICE, and Horizon Blue Cross Shield of New Jersey discussed key challenges and opportunities that the pandemic has presented for industry and how these learnings can be optimised to provide faster access to innovative therapies in the future. Key themes discussed included:

• Likely shortened time to approval for new therapies, and the need for HTA agencies to catch-up and keep-up with this trend
• Previous developments in the R&D landscape supported the rapid development of the COVID-19 vaccines through optimisation of existing processes and practical data sharing in a collaborative, multi-stakeholder and mutli-technology environment
• RWE can be used to bolster ‘imperfect’ clinical evidence packages when manufacturers cannot create a ‘gold standard’ RCT
• Development of methods to contain cost and predict budget impact will be important to promote sustainable access to therapies, these could include outcomes-based pricing, staggered price decreases, and ‘subscription models’ for treating patients
• In emerging markets, there should be a focus on finding ways to structure access and health systems that can accommodate innovative (high cost) therapies

Jake and Eleanor also attended the round table on Harmonisation of regulation across Europe: Establishing a common approach to drug assessment and approval

Key themes discussed were:

• Standardisation of data and outcomes collection
• Harmonisation of the assessment of clinical data and timings between European markets
• Consensus on priority therapeutics (pulling on experiences from successful collaboration in the pandemic)
• Standardisation of payment and financial models, and the data collection requirements, to pay for high-cost innovative therapies

Day two – Wednesday 24 March

Challenges in obtaining patient access for orphan drugs

The morning plenary for day two addressed the challenges and megatrends in obtaining patient access to orphan drugs. Only 3% of rare disease populations have an available therapy for their disease, and not all of these patients have access to these within their home country.

In some geographic areas patients may have a delay in access to innovative medicines of up to 2-years or longer which far exceeds the EMA target for national reimbursement within 4-6 months. Germany is currently the only market that meets this goal with access on the day of launch. China and Spain typically have a lower willingness to pay; reimbursement of orphan therapies over the cost of $40,000 per treatment is uncommon in China, which can lead to ‘cherry picking’ within market launch strategies if pricing negotiations cannot result in a price within the acceptable pricing corridor. The economic evaluation requirements within the HTA processes in the UK and Spain may also result in slower times to access due to strict ICER thresholds.

The discussion continued around what strategies can be used to facilitate patient access to orphan drugs. Current trends include:

• The need to increase pricing transparency
• Inclusion of the patient perspective from early within the clinical development process
• Increased use of RWE to drive decision making, with reference to the DARWIN EU project
• Reforms to existing national P&R policies, including the recent updates to the AIFA guidelines, NICE HTA methods and the new CEPS-LEEM framework agreement
• Highlighting the need for greater acceptance of managed entry agreements, includingRWE generation, to mitigate payer uncertainty, with reference to outcomes-based payments
• Utilisation of novel payment models such as annuity payments to mitigate financial risk

Roundtable: Pricing models for cell and gene therapies: What have we learned?

Jake James attended this roundtable, where the current issues regarding the pricing of ATMP products were discussed. One particular theme that arose was the lack of willingness for nations to adopt an annuity payment system for high cost, innovative therapies. Other key discussion points were:

• Changing stakeholder thinking to view a cell/gene therapy as a holistic process rather than simply a treatment (e.g., putting an emphasis on patient diagnosis, and the associated costs, through to patient follow-up and collection of RWD)
• The need for manufacturers to ensure they have a robust natural history model in cases where it is difficult to identify an appropriate comparator therapy
• The need for commercial teams to engage with the ATMP development process and HTA advice procedures earlier within the clinical development pathway to ensure successful timely launch

Day three – Thursday 25 March

Updates on the parallel HTA pilot in the Netherlands

A representative of the Medical Evaluations Board (MEB), discussed updates from the pilot examining the parallel marketing authorisation and HTA procedures. This procedure has been used to evaluate extramural products and expects to accelerate patient access by 90 days compared to the current sequential procedure.

The pilot has presented successful results, with three of the seven applications having been completed, two ongoing, and the other two expected to start in 2022. Early contact between Zorginstituut Nederland (ZIN), MEB, and the manufacturer is critical. The process is unchanged, but starts earlier, requiring significant alignment between stakeholders.

For future submissions, the MEB is encouraging manufacturers to consider the parallel procedure, and state that other types of applications could be possible, such as extension of indication and accelerated registration procedures. The parallel procedure is a forerunner in Europe, and the MEB remain open to sharing information with HTA agencies and other partners to expand the experience across other markets.

The benefits of biosimilar uptake on stakeholders

The COVID-19 pandemic has disrupted healthcare systems worldwide, causing a rise in healthcare expenditure.  Global healthcare spend rose in 2020 by €120Bn, largely due to large scale uptake of antivirals, vaccines, and telemedicine. As a result of this, payers need to regain financial control of their healthcare budgets.

The uptake of biosimilars presents opportunities to many stakeholders, including:

• Payers, who can manage their healthcare budgets by introducing biosimilars, with many biologics losing exclusivity between 2024 and 2029. This could allow re-allocation of budgets towards novel therapeutics like cell and gene therapies.
• Patients, who are more likely to access potentially life-changing medicines
• Physicians could utilise the value-added services that increased competition can bring

To incentivise biosimilar uptake, gain sharing agreements can potentially be implemented. A recent example was seen in Germany, where physicians were compensated €10 per quarter per patient initiated and maintained on biosimilar medication. This led to significant uptake of biosimilars of infliximab.

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Article published 25 March 2022.