Expertise

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.

Projects in This Space

01. CDMO Selection

  • Landscape overview
  • CDMO partnership support for the pre-clinical, clinical, and commercial stage

02. 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

03. Pricing and Reimbursement

  • Price and pricing model for value optimization
  • Guidance on risk-sharing and innovative contracts

04. Policy and Advocacy

  • Stakeholder engagement strategy (e.g., payers, physicians, patient organizations)
  • Raising awareness and advocacy (e.g., with payers, patients, regulators)

05. 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)?

06. Patient Support Program

  • How to best support patient throughout their journey and differentiation from the competition?

Case Studies

Payer Advisory Board+

A company developing a gene therapy wanted to validate the pivotal clinical trial design recommended by the EMA and investigate payer interest in different payment modalities.

Bionest organized a payer advisory board with representatives from EU5 countries (payers and KOLs) including development of a payer brief, moderation of the session, and individual follow up.

Payers recommended a different trial design from the EMA and highlighted preferred country-specific payment modalities.

Cell Commercialization Roadmap+

A company wanted to understand the main challenges associated with adoptive T-cell therapies from development through commercialization in the US and EU5.

Bionest analyzed several cell therapy analogs and held a working session with the company key stakeholders to identify main roadblocks along T-cell development (including manufacturing, supply chain, medical, regulatory, market access, commercial) and how to overcome these.

Key deliverables included a detailed commercialization roadmap (i.e., activities, responsibilities, timing, and KPI) for the company’s program, highlighting success factors and pitfalls to avoid.