Gene and Cell Therapies
Cell and gene therapies provide a unique new opportunity to effectively act on diseases in an unprecedented way.
The concept of “one and done” treatments has gone from theory to practice with the first approvals in the field, and many more are expected to come.
On one hand, these new approaches are rapidly transforming and reshaping the clinical landscape of the indications that they seek to treat. By changing the natural course of the disease as we know it, with patients becoming healthier and their medical needs shifting, they create novel opportunities for companies to build franchises based on these evolving needs.
On the other hand, cell and gene therapies also come with challenges, including the high COGS and uncertainty around sustainability of the therapeutic effect which ultimately translate into complex market access and pricing discussions with Health Authorities. Also, to speed approvals and facilitate evaluation of these therapies in the short-term, companies have explored novel and surrogate endpoints, further increasing uncertainties around clinical trial design.
01. Go-to-Market Model
- Opportunity assessment
- Commercialization strategy
- Guidance on sites (capacity, location) to deploy
- Strategies to optimize patient detection and referral to centers of excellence
02. Pricing and Reimbursement
- Price and pricing model for value optimization
- Guidance on risk-sharing and innovative contracts
03. Policy and Advocacy
- Stakeholder engagement strategy (e.g., payers, physicians, patient organizations)
- Raising awareness and advocacy (e.g., with payers, patients, regulators)
04. Market Access
- Who are the key stakeholders/ bodies involved in CGT market access? What are the country-specific processes? Is my CGT eligible for cross-country funding (i.e., S2 pathway)?
05. Patient Support Program
- How to best support patients throughout their journey and differentiation from the competition?