The annual World Pharma Pricing Market Access & Evidence congress was held on 23–25 September 2020 for the first time in a virtual environment, and was attended by members of our Value Demonstration Practice.

This three-day event enabled payers, health technology assessment agencies, pharma/biotech and other market access (MA) stakeholders to exchange information on 10 topics, including pricing and MA, evidence generation and real-world evidence, big data and analytics, health technology assessments and managed entry agreements (MEAs).

A common theme running through this year’s sessions, round tables and interviews was inevitably the impact of the COVID-19 pandemic, but there was still much to be learnt about innovations and best practice in the field.

Pricing challenges for innovative advanced therapy medicinal products (ATMPs)

As in recent years, there was much focus on potentially curative cell and gene therapies, in view of the expanding pipeline and their unique considerations for MA and pricing. We heard from several speakers about the innovative pricing schemes and access models that had been implemented for these therapies to accommodate high upfront costs. There were multiple examples of how payers were driving change in relation to this innovation (e.g. NICE developing guidelines on ATMPs), but there was also discussion on how some were encountering challenges due to the burden of the schemes. Alexander Grosvenor from PRECISION advisors provided some advice for industry in terms of how to succeed with these therapies, including considering the willingness of each payer and the feasibility of such agreements. He also described how the advantage of being first-to-market can be more pronounced with gene therapies than other therapies, owing to the pool of prevalent patients, but how later gene therapy entrants may still succeed if they offer greater value, with physicians and patients potentially waiting for this treatment option.

Valuing patient advocacy is key

The importance of patient involvement in drug development and MA was a key theme during an afternoon session on patient advocacy. Attendees heard from Nadia Bodkin of the Rare Advocacy Movement, a patient advocacy group for rare diseases, of the increasing importance placed on the patient perspective by reimbursement authorities in recent years. Nadia advised industry to establish a corporate infrastructure that values patient advocacy and to consult with advocates to co-create websites/campaigns and design trials.

We also heard how patient advisory groups have interacted directly with payers and collaborated with multiple industry stakeholders to accelerate patient access. Josie Godfrey of JG Zebra Consulting described how, through the patient-led HERCULES project, economic and natural history models, disease-specific PROs and burden-of illness data for Duchenne muscular dystrophy had been generated so as to fulfil payer evidence requirements.

In the same disease area, we also heard from Rob Burley from Muscular Dystrophy UK about how this group has supported access for multiple products. Transparent communication has been their key to working best with industry.

Transparency versus confidentiality – the future for managed entry agreements

Bart Van Den Daele of Gilead Sciences described his experiences of MEAs. MEAs are useful for medicines with existing gaps in efficacy data, and they allow for risk mitigation while enabling quicker access for patients in need. Bart described how MEAs can be most beneficial for smaller countries, where overflow to bigger countries is considered low risk and therefore confidential rebates can allow deeper price concessions by pharma.

The speaker gave caution, however, about public outcries for greater transparency and increased cross-country collaborations, which threaten the perception of confidentiality and may compromise the benefits of MEAs. It was suggested that the system should be updated to enable increased transparency in a controlled way, including confidentiality charters and codes of conducts for cross-country collaborations.

Overall, the first virtual World Pharma Pricing Market Access & Evidence meeting was a great success for the organizers and the HEOR community amidst the adversity of the COVID-19 pandemic, and provided an excellent opportunity for specialists to connect and discuss new challenges in this field and how we can all help to meet these challenges now and in the future.