Stakeholder Report for Market Access Challenges and Solutions in Cell and Gene Therapy

Project background

The client was planning to launch a new CGT therapy. There is a need to identify Dutch market access challenges and provide solutions with actionable recommendations to unlock access to CGT for Dutch patients who need this transformational promise of innovative treatment.

Challenges faced

In recent decades, cell, gene, and tissue therapies have emerged as innovative treatment options. These potentially curative treatments are associated with large health gains for patients with conditions for which there are often limited treatment options available. Despite the promise of CGT, there are several challenges to ensuring timely and optimal reimbursed access to these innovations. CGTs are complex therapies with relatively high initial costs, and the curative claim is often based on small studies with limited follow-up. The challenges surrounding CGT are widely acknowledged in the scientific literature. International reports and white papers provide recommendations on how to overcome Health Technology Assessment (HTA) challenges and how to improve access to CGT for patients. In Europe, the joint clinical assessment of CGT will start in 2025. However, the remaining challenges in assessing the comparative (cost)effectiveness, and affordability of CGT therapies will require country-specific solutions.

Our solution

A targeted literature review (TLR) was performed to identify market access challenges and solutions for CGT therapies. Challenges were categorized into clinical trial design and evidence base, health economics and reimbursement assessment, funding, and procedural and organizational challenges. Next, semi-structured interviews with stakeholders in CGT were undertaken to build on the knowledge gathered in the TLR. Based on the categories of challenges identified in the TLR, interview questions were framed to be relevant for the Netherlands and grouped as follows: future readiness of the assessment framework by ZIN for CGT assessment, clinical evidence concerning the state of science and practice, HTA challenges, funding and Dutch procedural and organizational challenges. Stakeholders with CGT experience (N=15) were interviewed. The following stakeholders participated in the interviews: healthcare practitioners, hospital pharmacists, a former policy maker from the Ministry of Health, health insurers, patient advocacy groups, manufacturers, the Innovative Medicines Association, and academics with expertise in HTA.

Based on the challenges and solutions revealed by the TLR, various white papers, and the interviews, the following key vulnerabilities for the Netherlands have been identified:

• no shared ownership of future readiness for CGT

• uncertainty in (long-term) efficacy, safety, and cost-effectiveness

• lack of knowledge and experience in contracting agreements for the financing of CGT

• HTA methodological challenges

• no clear procedures and legislations around CGT (in the EU and the Netherlands)

• lack of patient voice in evidence generation

Our impact

Proposed solutions by the TLR and the interviewed stakeholders were consolidated into actionable recommendations for addressing the key vulnerabilities by a multi-stakeholder approach. These recommendations can be the first steps of a united approach to working together and learning from each other to unlock access to CGT for Dutch patients who need this transformational promise of innovative treatment.

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