For many biopharma companies, running an expanded access program (EAP or Compassionate Use Program, CUP) is an extremely important component of bringing early treatment options to patients with high unmet medical needs, yet it is understood that offering expanded access requires certain financial commitment from the business. The ultimate goal is to provide much-needed treatment to patients who have an urgent need, but companies must find balance in managing program costs. While costs are associated with planning, designing and implementing an effective EAP, there are opportunities to offset these costs through charging for access in certain situations.
In order to make the most of this opportunity, biopharma companies should consider reaching beyond their primary commercialization target countries and consider the potential demand for their products in other markets.
What is sometimes overlooked in the initial planning of expanded access strategy, is that many countries permit charging or formal reimbursement for access to unlicensed drugs before local regulatory approval. While not always appropriate, the charged access model can be successfully integrated as part of an overall global EAP strategy. In some well-known commercial markets, the regulatory mechanisms for charged expanded access are robust and well utilized.
For example, in France, pharmaceutical products that do not yet have marketing authorization or are not being investigated in clinical trials can be offered to patients for early access via a Temporary Authorization for Use (ATU) mechanism. This regulatory pathway is aimed at medicines that treat rare or serious diseases, particularly when there are no other treatment options available for patients or when treatment cannot be delayed. Manufacturers can supply products under the ATU status free of charge or on a charged-for basis, at an agreed rate.
Importantly, there can be many alternative funding pathways for unlicensed medicines offered via EAPs other than private pay by the patients. Countries such as the U.K,, Germany and France have established payment mechanisms through public and hospital sick funds; various payors can be involved, with the exact strategy dependent on several factors related to the particular unlicensed drug and extent of unmet medical needs.
Challenges and Considerations for Compassionate Use Programs
Although opportunities for charging for access during EAPs do exist, careful planning around regulatory considerations should be taken into account during early planning stages to ensure the access strategy is fully aligned with the commercial objectives of the particular drug asset.
The regulatory mechanisms governing expanded access and compassionate use programs vary country by country; even jurisdictions such as the European Union lack a unified regulatory framework. Therefore, it should be expected that each country will have unique requirements for providing charged-for early access to products that have not yet been approved locally or formally reimbursed. For biopharma companies lacking in-house expert EAP resources, planning and navigating charged-for access opportunities can be a challenge. Importantly, strategic and regulatory decisions can have far-reaching future implications related to commercialization and reimbursement.
Sponsors considering offering an unlicensed drug via a charged EAP should address the questions on when and where it is appropriate to charge for access, how a pre-commercialization price can affect future commercial pricing levels, the ethical aspects of fair and equitable access to treatment for patients, commercialization footprint and implication of charging in future commercial markets versus those without commercialization intent, and the drug’s current regulatory status.
All these areas play an important role in planning a successful strategy, which can, in turn, help offset the financial burden of running an EAP while allowing broader access to patients in need. With the right EAP partner, these considerations and other nuances can be accounted for in the planning process, reducing the risk of unexpected future issues.
Plan for Your Expanded Access Program to Succeed
At Durbin, we recognize that biopharma face budgetary constraints in the pre-launch phase of a product. When partnering with a sponsor to design and deliver their EAP, we focus on creating strategies for sustainable expanded access programs.
To do this, we comprehensively review the sponsor’s current drug development status and commercialization plan and build a tailored, expanded access strategy around each specific drug asset. As part of that process, we assess the countries in which permitting access to the drug may be most appropriate, considering regulatory, medical and commercial factors. We also identify the countries in which there is ability to charge for access programs, including routes such as government funding initiatives for locally unlicensed products.
The strategic planning process results in recommendations for markets in which it may be preferrable to supply the drug free of charge, and where access can be charged for, and at what time. Interestingly, many expanded access programs are either cost-neutral, or revenue generating for the sponsor.
While addressing unmet medical needs with potential to generate early revenue are two of the benefits, there is also a substantial opportunity for biopharma companies to collect Real-World Data (RWD) much earlier than usual and build global awareness of the product ahead of local commercial launch. Both opportunities can aid the overall success of the drug’s launch.
The challenges outlined earlier should be addressed through careful planning. For many biopharma companies, in the absence of substantial in-house expertise on global EAP regulations and best practices, one approach is partnering with a full-service EAP provider, such as Durbin. From our deep experience in designing and delivering global EAPs, we are equipped with the regulatory expertise to design an optimal access strategy and provide hands-on support globally.
Through our sophisticated wholly-owned distribution centers located around the world and knowledgeable and flexible team resources, we provide pharmaceutical organizations with complete transparency and control during the entire expanded access journey.