A Brighter Future for Orphan Drugs in Spain: Progress, Challenges, and Ways Forward

This week we continue our look at orphan drugs and turn our focus to a specific area: market access for orphan drugs in Spain. Traditionally, Spain hasn't exactly been known for swiftness in granting access to these critical therapies, leaving patients waiting in limbo. However, recent years have brought a glimmer of hope. Let's delve into the positive strides being made, along with the remaining roadblocks that need to be overcome. We wrap up this article with suggestions for drug developers on how to proceed.

Faster Approvals, More Financed Drugs: A Cause for Optimism

There's good news to report! According to Spanish Association of Laboratories for Orphan and Ultra-Orphan Medicines (AELMHU), assessment times for pricing and financing orphan drugs in Spain shrank from 34 months in 2022 to 23 months in 2023 (1). This translates to a significant reduction in the wait times patients endure before accessing these treatments. 

Additionally, the number of orphan drugs receiving financing has seen a welcome increase. 2023 marked a record year, with Spain financing 21 new orphan drugs, compared to 9 in the previous year. This signifies a growing recognition of the importance of timely access for these therapies.

However, it's important to acknowledge remaining hurdles. Firstly, while the reduction in the time for the reimbursement determination is welcome, much more must be done to reduce this to acceptable levels. Secondly, 49% of all drugs that are financed by the health system have restrictions (either to their indications, or indications that are not reimbursed). Finally, no advanced orphan therapies e.g., orphan cell or gene therapies, received financing in the last two years. Only 5 such therapies are currently reimbursed by the health system.

Adapting to Change and Overcoming Regional Hurdles

The challenges facing market access for orphan drugs in Spain, was recently discussed in a round table event hosted by Diariofarma, Chiesi, Sobi and BioMarin (2) and included experts from leading Spanish organisations. Many issues that were discussed touched on issues that are also seen in other countries, including:

• The need for patient involvement: The voices of patients and patient associations are crucial. They understand the true impact of access delays on their quality of life. Currently patients have little involvement in the financing decisions in Spain.
• Real-world evidence: Orphan drugs often have limited clinical trial data due to the rarity of the diseases they target. Incorporating real-world evidence, data collected from patients in real-world settings, can strengthen the case for access by complementing traditional clinical trial data.
• Innovative Payment Mechanisms: Uncertainties surrounding the long-term benefits of orphan drugs can pose challenges in traditional pricing and reimbursement assessments. Exploring innovative payment mechanisms can offer creative solutions to overcome these hurdles. As an example, the Spanish authorities adopted novel payment mechanisms for treatments approved for Spinal Muscular Atrophy (3). These include budget limits, treatment protocols, payment by results and price volume agreements. Such approaches help define areas of common interest and can ultimately enable access.

The experts also highlighted specific challenges for Spain:

• Integrating the Joint Clinical Assessments (JCA): This pan-European initiative designed to streamline assessments, requires careful adaptation of Spain's existing Pricing and Reimbursement (P&R) process. This will be particularly important for the Therapeutic Positioning Report (Informe de Posicionamiento Terapéutico, IPT), which plays an important role in pricing and reimbursement decisions. 
• Access at the Autonomous Region level: Even after receiving national financing approval, delays in reimbursement by individual Spanish regions can significantly disrupt treatment access for patients. Spinal Muscular Atrophy (SMA) serves as a stark example. Despite national financing approval, delays in individual regional reimbursement or complex processes to access treatment, created frustration for patients with this debilitating disease.

By strategically addressing these areas, Spain can improve orphan drug access, thereby getting therapies to patients faster and improving incentives for orphan drug developers.

Navigating the Spanish Market: A Guide for Drug Developers

Here are some key pointers for drug developers aiming to improve market access for orphan drugs in Spain:

1. Engage Early with HTA Bodies: Early engagement with the Spanish Health Technology Assessment (HTA) bodies is crucial. This allows for discussions about the evidence base that will be used to evaluate the drug. For example, ensuring clinical trial designs meet not only the needs of the regulatory agencies, but also the payers as well.
2. Embrace All Relevant Data Sources: Consider all relevant data sources, including real-world data, pre- and post-approval, to demonstrate value and bolster your case for market access
3. Partner with Patients: Engage with patient associations and advocacy groups. Understanding patient needs and perspectives is vital for successful market access.
4. Explore Novel Payment Mechanisms: Be open to exploring innovative payment mechanisms, such as performance-based models, if there are uncertainties in the clinical data.
5. Don't Forget the Regions: Market access isn't just a national issue. Work with the Autonomous Regions to understand their needs and minimise potential delays or constraints upon access.

References

1. Resumen Ejecutivo Informe Anual Acceso 2023 de los Medicamentos Huérfanos en España, AELMHU, Jan 24
2. La gestión de los datos, clave para afrontar los retos de P&R en los medicamentos huérfanos, Diariofarma, Apr 24
3. Acceso a medicamentos huérfanos para el tratamiento de la atrofía muscular espinal en España, García-Parra et al, Rev Neurol, Nov 2022

If you are interested in discussing any of the issues above further for your company/drug development program, please contact me through my LinkedIn profile. Please note, I have no conflicts of interest in the production of this article.