Unlocking market access success for cell and gene therapies: the vital role of early strategic planning
September 5, 2024 | CGT, Market Access, Strategic planning, cell and gene therapy, payer reimbursement
By Manca Povsic
Unclear vision at product launch, misalignment of functions, no planned flexibility, slow response at key commercialization stages – sound familiar? Strategic planning is talked about a lot but is difficult to get right.
Strategic planning is a cross-functional activity, but what are the specific opportunities and pitfalls for market access functions and how can early strategic planning lead to market access success, particularly for cell and gene therapies (CGTs)?
Strategic planning is an essential step in outlining product vision and direction to optimize market access and reimbursement potential. This is especially important in the cell and gene space, which is an area of great growth but even greater uncertainty, with relatively few precedents and an evolving access landscape.
The challenges for CGTs
| Challenges | What this means for market access |
| CGTs are complex molecules and frequently involve a very intensive research and development process | • High price |
| CGTs have high manufacturing and administration costs because of their complexities | • High price
• Additional costs after reimbursement |
| Many CGT companies are small start-ups, operating with limited funds and launch experience | • Limited manufacturer market access experience |
| CGTs serve focused patient populations | • High price
• Perception of low unmet need, difficult to recruit patients |
| Many CGTs target relatively unknown diseases to both payers and patients | • Perception of low unmet need or disease burden |
| The CGT space is becoming more competitive. Even in markets where there is no direct competition, tightening healthcare budgets mean there is less money to spend on CGTs, which creates unintended competition across disease areas | • Lower price
• Harder to gain reimbursement |
| CGTs frequently represent a high upfront cost despite the uncertainty around long-term data and durability of response | • High price
• Payers less likely to fund CGTs |
| Managed-access agreements can be challenging and time-consuming (eg agreeing a deal, allocating responsibilities, managing access over several years, etc | • Delays in patient access, additional costs after reimbursement |
All these challenges make CGT commercialization high risk, with many products failing to reach target patients. In recent years, we have seen many CGTs successfully navigate the early road to market only to fail at the approval and reimbursement stage – why is that?
Time and again we see market access planning for CGTs relegated to the very last moment, forcing teams to reactively manage the consequences of earlier (in)decisions.
While there are many reasons why market access planning is often delayed, including siloed working and lack of experience and resources, the benefits of getting it right from the start have the potential to more than outweigh the risks. With the CGT environment getting ever more crowded, the competitive landscape is now increasingly nuanced, which makes setting a clear strategic direction to begin with even more important.
What are the key activities that need to be considered early to promote success in the market access space?
- Develop the product profile and its value drivers early
Having a strong value story and being ready for challenges is particularly important for CGTs, where uncertainty is high and there are expectations of life-changing or even curative potential. Developing early product profiles and best-, medium- and worst-case value scenarios allows for clarity on the fundamental drivers of product, patient and societal value.
For example, if your product is primarily going to relieve patients of their weekly injection burden, you should consider this to be your core value driver; it should guide all aspects of your strategy, whether it is clinical trial design and endpoint selection, patient and healthcare professional communications or seeking approval and reimbursement.
Clarifying the strategic vision and target product profile(s) as early as possible allows you to break down your commercialization journey into stages and puts a laser focus on your access needs. Pivoting your strategy at one stage of the journey can be much easier than having to take a different route altogether.
- Consider your trial design carefully
Having a clear end goal starts with being clear on what you want to achieve with your clinical trials. With internal priorities at this stage primarily focusing on whether the product works, other priorities, such as access and reimbursement, can end up getting left behind. Trial design does not often take payer expectations or needs into consideration, but this is critical when planning for successful reimbursement.
CGTs serve focused patient populations with little room to expand to additional indications, so it is crucial that the trial population is well defined in the planning stage and the unmet need is understood. Defining the population includes considering the type of patients likely to be recruited into the clinical trial.
Consider the following:
- Will the patients be children or adults?
- Will they be very ill or have a low burden of disease?
- Are there stratifications in the population, eg progressed/non-progressed?
- Will disease burden/costs and type of population affect the types of outcomes that will be measurable and the time of follow-up?
To achieve successful reimbursement, early planning should focus on the highest unmet need, where the CGT has the best chance of filling the gap and offsetting the cost burden. Defining key populations and understanding what endpoints will resonate with payers across different markets early on will ensure you understand what success looks like in your clinical trials.
Another important consideration for early planning is endpoint design. Clear and early focus on patient-perceived benefits is key, especially in disease areas where there are many endpoint measures available (or, equally, very few). Endpoint and comparator selection is an important part of your early strategy, so consider it well ahead of trial commencement, including planning for validation steps with patients, regulators and payers, particularly if surrogate endpoints are being considered. This will help to expedite approval and reimbursement discussions later.
- Know your payer uncertainties
Strategic planning is all about anticipating the steps needed to achieve your overall goal, which cannot be realized without considering how your desired approval and reimbursement outcomes will be achieved. A clear outline of the key uncertainties and challenges your CGT will likely face at health technology assessment (HTA) will make this part of the journey easier to navigate.
Uncertainties may include the following:
- Efficacy: CGTs frequently come to market with only early phase trial data in a small patient population. Even if trial data show curative effects, the uncertainty of efficacy in a real-world population over a longer time period is high
- Safety: lack of long-term follow-up increases the uncertainty surrounding long-term or repeated use of CGTs, particularly in young patients who may use CGTs for the rest of their lives
- Cost-effectiveness: efficacy and safety data gaps lead to large uncertainties in the value of CGTs, particularly if the therapies rely heavily on extended data extrapolation. This can lead to budgeting uncertainties, especially when the therapies are expensive and there is a lack of clarity on patient numbers
- Managed-access: lack of clarity around the value and price of CGTs can lead to payer hesitation to fund managed-access agreements, as well as the complexities of who will run them and how the long-term operation will be overseen can see managed-access programmes struggle to get off the ground
- Manufacturing/administration: most CGTs require specialized manufacturing and storage and administration processes. It is not always clear what infrastructure will need to be developed before patients can access these therapies and how this will be financed
Defining uncertainties that may be faced at launch allows for planned flexibility to be built into the product strategy. This is especially important in the CGT space, where achieving patient access early can mean the difference between cure and lifelong treatment.
- Plan stakeholder engagement
We have all seen the news around lengthy HTA assessments and long manufacturing times for CGTs, while eligible patients wait to access these therapies. This is why a key aspect of early strategic planning is expediting patient access wherever possible. Once you know and plan for your uncertainties, the decisions that need to be made to ensure patient access should be clear. Outlining the timeline and requirements for these decisions will allow you to benchmark your progress against clear goals.
You may not know all the answers or even all the decisions facing your product. This is why it is vital to plan collaborations with stakeholders at the right points in your journey, which may include the following:
Clinical trial planning
- Patients and patient organization involvement will shape therapy development, clinical trial design and real-world implementation
- Early engagement with healthcare professionals, payers, regulators and policymakers ensures alignment and buy-in before you invest in expensive trials
Launch planning
- Payers may lack familiarity with CGT indications, so planning for advocacy and education is essential. Engaging HTA experts and patient organizations helps to bridge this gap
Approval planning
- Patient access schemes and registries must be planned well in advance; involving patient organizations and HTA stakeholders ensures their requirements and costs are considered ahead of approval
Patient access planning
- Early planning allows for the incorporation of patient-centric approaches to reduce the patient burden of administration and hospital visits
- Involving HTA stakeholders and payers ensures any additional costs of CGT use/administration are considered well before therapies are made available so they can be built into budget models
To complicate matters, many of the critical decisions may be interconnected. This makes it vital to collaborate, not just externally but internally, across teams and functions to work towards a common strategic goal. You can’t guarantee that you will choose the right path every time, but plotting a clear journey from the outset will allow you to take an agile approach and renavigate if necessary.
Four steps to put you on the road to market access success
- Understand your therapy’s challenges and opportunities
You don’t need to consider every pitfall right away, but you do need to plan for the key obstacles and milestones, particularly if you are entering a crowded marketplace or a focused patient population.
- Define your biggest reimbursement uncertainty
Fostering strategic thinking and clear, simple solutions from the start can yield big rewards at approval and reimbursement stages. Focus on the big uncertainties first and build your plan around them.
- Design a trial that’s future-proofed for market access
Spend time upfront considering how your trial design can help you gain reimbursement and patient access, not just show efficacy. Focus on realistic trial populations and meaningful (and payer-endorsed) endpoint selection and incorporate patient perspectives throughout.
- Engage with reimbursement stakeholders early
Understanding your reimbursement stakeholders is key for a successful route to market. Outline when seeking stakeholder advice will help expedite your access plans and differentiate between earliest/latest and best time for engagement.
There’s a lot to consider on your journey to market access success, and we would love to help you get there. Contact Manca Povsic (manca.povsic@amiculum.biz) to learn more about how we can support your early market access planning for CGTs and beyond. For more insights, visit the AMICULUM News and Insights page.
This content was provided by Amiculum
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