Case:
Introducing Rare Disease Gene Therapy in Denmark

Introduction

“How do you introduce a multi-million kroner treatment when there are only a handful of patients?” This question, posed by our client, a global pharmaceutical company, encapsulates the paradox of rare disease therapies in modern healthcare systems. Their groundbreaking gene therapy promised to halt a devastating genetic disorder – but first, it had to navigate the intricate channels of the Danish healthcare system. Here’s how we turned historical data into a roadmap for the future.

Challenge

Our client, a global pharmaceutical company, was in the preparatory phase for launching an innovative advanced therapy medicinal product (ATMP) designed to treat a rare disease.

While the potential for this groundbreaking therapy was immense, the company faced several significant challenges:

1. Economic Implications:
   ATMPs for rare diseases often come with hefty price tags, reflecting years of research and development and the small patient populations they serve.
   
   
2. Limited Clinical Data:
   Given the nature of rare diseases and the novelty of ATMPs, robust clinical data can be scarce. This shortage of evidence can make it difficult to demonstrate value to health authorities and payers.
   
   
3. Hospital Budget Constraints:
   hospitals operate under strict budgetary limits. Introducing an expensive new treatment can be a significant financial undertaking, often requiring special considerations.
   
   
4. Complex Decision-Making Process:
   The Danish healthcare system, with its multiple stakeholders and rigorous evaluation processes, presents a unique landscape that must be carefully navigated.
   

Recognizing these challenges, the client sought our expertise to gain insights into how similar innovative products had fared in the Danish market. They needed a clear understanding of the potential bottlenecks and key decision points in the market access journey.

Mikael Bergholdt Asmussen, Chief Advisor for Market Access at Signum, explains the situation: “When launching ATMPs, it’s often a challenge due to the economic consequences and limited clinical data. Our client needed to understand how these factors had played out in previous cases to better prepare for their launch.”

Solution

To address our client’s needs, we devised a comprehensive approach leveraging our unique access to data and deep understanding of the Danish healthcare system:

1. Historical Case Analysis:
   We conducted an in-depth examination of similar cases where innovative products for rare diseases had been introduced to the Danish market. This involved: a) Reviewing publicly available information on previous ATMP introductions. b) Analyzing the decision-making processes of key stakeholders, including the Danish Medicines Council and Amgros (the pharmaceutical procurement service of the Danish regions). c) Identifying common concerns and decision points in these cases.
   
   
2. Stakeholder Mapping:
   We provided a detailed overview of the important stakeholders involved in the introduction of ATMPs in Denmark, including their roles, influences, and key considerations.
   
   
3. Process Deep Dive:
   Leveraging our exclusive access to pharmaceutical sales data and insights from the Danish Health Data Authority’s Secure Research Platform, we reconstructed the journey of similar products from application to patient treatment. This included: a) Timelines for various stages of the approval process. b) Key decision points and their outcomes. c) Pricing and reimbursement negotiations.
   
   
4. Bottleneck Identification:
   By combining our historical analysis with current market insights, we pinpointed potential bottlenecks in the healthcare system that could impact the introduction of our client’s ATMP.
   
   
5. Comparative Analysis:
   We provided a detailed comparison of how products within the same pharmaceutical modality as our client’s innovative therapeutic had been introduced and received in the Danish market.
   
   

Mikael Bergholdt Asmussen emphasizes the value of this approach: “By unwrapping the setting using similar cases, we were able to provide our client with valuable insights into the processes, concerns, and decisions made by the Danish Medicines Council and Amgros. This comprehensive view, combined with our unique access to sales performance data, allowed us to paint a clear picture of what our client could expect.”

Results

Our analysis provided the client with a wealth of actionable insights to inform their market access strategy:

1. Decision-Making Insights:
   We offered a clear understanding of how the Danish Medicines Council and Amgros had approached similar ATMPs in the past, including their key concerns and evaluation criteria.
   
   
2. Timeline Expectations:
   By analyzing previous cases, we provided realistic timelines for each stage of the market access process, allowing our client to plan accordingly.
   
   
3. Potential Hurdles:
   We identified several potential bottlenecks in the healthcare system, allowing our client to address these issues in their market access strategy.
   
   
4. Stakeholder Engagement Plan:
   Our stakeholder mapping allowed the client to develop a targeted engagement strategy, ensuring they could effectively communicate their product’s value to the right decision-makers.
   
   
5. Pricing and Reimbursement Insights:
   By examining how similar products had been priced and reimbursed, we gave our client valuable context for their own negotiations.
   
   
6. Hospital Readiness Assessment:
   Our analysis highlighted the need for new setups at treating hospitals for ATMPs, allowing our client to factor this into their launch preparations.
   
   

“The insights we provided went beyond understanding the process,” Mikael Bergholdt Asmussen notes. “We provided our client with a comprehensive view of the market access landscape. This allowed them to anticipate challenges and opportunities, strengthening their preparatory strategy for the product launch.”

By providing a detailed roadmap based on historical data, market insights, and a deep understanding of the Danish healthcare system, we empowered our client to approach their ATMP launch with confidence and a clear strategy.