Inequalities across disease areas can be addressed through innovative market access approaches
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A focus on:
Despite accelerating therapeutic innovation, inequalities in access to novel treatments have persisted. This is due to a combination of factors within the market access process and is particularly evident in rare diseases.
Our vision:
Market access policies which aim to reduce inequalities in access to novel therapeutics, particularly those concerning medicines for rare diseases.
Looking to the future:
Incorporate the use of innovative reimbursement mechanisms to realign incentives between payers and providers of medicines around patient and population health to reduce inequalities in access to therapies between those with rare diseases and those without.
Grace Mitchell
Market Access Specialist
Health inequalities are growing despite the increasing complexity of medicines and advances in technology
The drivers of health inequalities are complex and multi-faceted, as outlined in this report. However, a contributing factor is the differential time it takes for new medicines to be approved and become available for use. Different market access factors combine to create a complex environment with multiple opportunities for delaying the delivery of effective medicines to patients. This delay in access is exacerbated in rare diseases since the reimbursement market is not optimised to account for the uncertainty and risk associated with the medicines designed to treat them.
In the case of rare diseases, only around 5% have a licensed treatment. For those conditions which do have a therapy approved, the average time taken for the treatment to be reimbursed in the EU is much longer among orphan medications than the EU average (636 days vs 511 days), owing to complex market access dynamics (Figure 1).
Factors contributing to the uncertainty associated with the approval and reimbursement of rare disease treatments and orphan drugs include:
- The rarity of the disease means there are fewer data points across clinical trials, corresponding to lower certainty of results and/or a longer time duration being required.
- Innovative medicines targeting rare diseases are inherently expensive, meaning the pricing and reimbursement negotiations often take longer (Figure 1), while the uncertainty and therefore risk to payers are typically higher.
- There is often no standard-of-care medicine to compare against the efficacy of the novel intervention in a clinical trial setting making it harder to assess the medicine.
- Innovative medicines aim to offer long-term responses or cures. The novelty of rare disease interventions means long-term data to assess this is lacking.
- Payers may limit the patient population eligible to receive the drug to those most likely to respond in an effort to curb costs and reduce the risk of the drug not proving clinically effective (Figure 2).
Figure 1: Rate of availability: Percent of drugs available to patients in European countries as a proportion of EU approvals
Figure 2: Orphan rate of availability (%, 2017-2020)
Figures 1 &2. IQVIA, EFPIA Patients W.A.I.T. Indicator 2021 Survey. 2022.
Figure 1: Rate of availability: Percent of drugs available to patients in European countries as a proportion of EU approvals. Availability is defined for most countries as the point the product gains access to the reimbursement list.
Figure 2: A breakdown of the availability of Orphan medicines, by European country as a proportion of EU approvals.
A more flexible market accommodates innovation, prevents delays in patient access, and reduces inequalities more effectively.
Market access policies can help reduce inequalities in access to therapeutics
Currently, the pace of drug development exceeds that of changes to policy and market access which can act as a barrier to patients receiving innovative medicines. A more flexible market accommodates innovation, prevents delays in patient access, and reduces inequalities more effectively.
Some efforts to streamline the patient access pathway have been adopted, with examples including the Cancer Drugs Fund and Project Orbis. However, without embedding a commitment to reducing health inequalities at the outset, such projects may have the potential to exacerbate inter-indication inequalities. The inclusion of rare diseases in such efforts should be ingrained in policy decisions as a method for reducing inequalities in patient access to orphan drugs.
Innovative reimbursement mechanisms, such as managed entry agreements and outcomes-based contracts, pose significant potential for increasing access to orphan drugs by mitigating financial risk and uncertainty between payer and provider. Indeed, we are seeing a steady increase in the use of such mechanisms as countries are adapting to novel practices. However the rate of increase is slow and far outpaced by the number of innovative therapies entering the market. The market must be better adapted to use novel pricing mechanisms in the battle against inequalities.
The opportunities of real world data, as outlined in chapter 3, offer an additional value element for innovative reimbursement mechanisms. Our work on the clinical and financial design of implementable innovative reimbursement mechanisms has highlighted the potential for using the real world data collected through these schemes to identify inequalities in market access approaches at a high level of granularity, recognise trends in prescribing habits over time, and monitor patient uptake in close to real-time. These insights can lead to open conversations with prescribers to understand treatment patterns and highlight areas of inequalities.
Calls to action to address inequalities in unmet need
The market access environment must adapt in response to the dynamism of innovation in order to reduce inequalities in access to treatment. There are several ways in which relevant stakeholders can contribute to this change:
- Life sciences companies should develop cohesive and continued communication across all stages of development. This would ensure that drugs meet the appropriate data requirements for regulators, as well as relevant outcomes for pricing agreements. This approach ensures that the data is tailored to payer and regulatory requirements to make drugs available to patients in a more timely manner.
- Life sciences companies should harness the power of real world data through innovative reimbursement mechanisms. Innovative reimbursement mechanisms have the potential to transform the way we measure outcomes to facilitate faster and more equitable access to patients. Leveraging real world data, such as PROMs, through innovative reimbursement mechanisms is therefore critical in filling the data gap and measuring the value of rare disease therapies whilst reducing uncertainty.
- HTA bodies need to actively support a broader approach to evidence generation and reimbursement options for orphan medications. In the case of rare diseases, the ability to robustly demonstrate efficacy to the levels required to support reimbursement is unachievable for many patient populations. Allowing alternative evidence approaches and being open to more dynamic pricing mechanisms that reimburse proven patient outcomes are needed to accelerate patient access to these treatments.