Brazil Celebrates First Year of Cladribine Access for Multiple Sclerosis, Calls for Expanded Leverage
São Paulo, Brazil – A neuroimmunology meeting in São Paulo this week marked the first anniversary of cladribine’s availability through Brazil’s public healthcare system for the treatment of highly active relapsing multiple sclerosis (MS). The event, attended by international experts including Gavin Giovannoni, highlighted both the progress made and the need for broader access to this potentially transformative therapy.
Cladribine’s Impact and Current Limitations
Cladribine, an oral medication, has been approved in Brazil for use in patients with highly active relapsing MS. However, its current indication is limited, categorizing it as a third-line disease-modifying therapy (DMT). This restricted access prevents many individuals with MS from benefiting from treatment, particularly those in early stages of the disease or those with more advanced forms of MS. Currently, Brazil’s regulatory framework classifies MS into three distinct diseases – relapsing remitting, secondary progressive, and primary progressive – and lacks approved therapies for progressive forms like siponimod for secondary progressive MS and ocrelizumab for primary progressive MS.1
Recommendations for Optimizing Cladribine Use in Brazil
Experts at the meeting, including Professor Giovannoni, outlined several key recommendations for Brazilian health authorities to maximize the benefits of cladribine:
Aligning with WHO Essential Medicines List
The World Health Organization (WHO) has included cladribine on its Essential Medicines List (EML) due to its potential to transform MS management in resource-constrained settings, mirroring the realities of Brazil’s public healthcare system. With cladribine now off-patent or nearing the end of patent protection in many markets, the anticipated arrival of generic versions promises to significantly reduce the drug’s cost – potentially by 90% over the next 3-5 years.1 This cost reduction supports broadening the indication to include both active and smoldering MS.
Revising the Classification of MS
The current classification of MS into distinct subtypes is increasingly viewed as scientifically flawed, driven historically by regulatory incentives rather than biological understanding. Modern research suggests MS is a single disease with overlapping biological mechanisms. Focus should shift towards identifying and treating active inflammation (responsible for relapses) and addressing smoldering MS-associated worsening (SAW), measured as progression independent of relapse activity (PIRA).1
Understanding Cladribine’s Mechanism and Dosage
Brazilian authorities need a deeper understanding of how cladribine works, differentiating it from other DMTs. Cladribine is not a chronic immunosuppressant; its immunosuppressive effects are mild, short-lived, and front-loaded, allowing for full immune system reconstitution post-treatment. This profile minimizes the risk of opportunistic infections and progressive multifocal leukoencephalopathy (PML).1 cladribine’s selectivity for memory B-cells and its rapid action offer distinct advantages. Contrary to some perceptions, cladribine demonstrates high efficacy comparable to other leading DMTs like alemtuzumab and natalizumab.1 Its oral administration, short courses, and minimal monitoring requirements enhance adherence and accessibility, particularly for patients in remote areas.
Addressing Barriers to Adoption
Incentives within dual healthcare systems (public and private) can sometimes hinder the adoption of convenient, easy-to-administer therapies like cladribine. Healthcare facilities may benefit financially from treatments requiring more intensive administration and monitoring. Addressing these perverse incentives through policy changes is crucial to promote wider adoption of cost-effective treatments like cladribine.1
The Role of Compassionate Use and National Registries
Experts advocate for a national compassionate use program allowing patients with progressive MS to access cladribine while awaiting the availability of affordable generics. Establishing a national registry to track outcomes in patients treated with cladribine would provide valuable data to support broader access and inform future economic evaluations.1
Economic Considerations
A recent cost-utility analysis of drugs available in Brazil for relapsing-remitting MS found that teriflunomide and alemtuzumab were dominant in the analysis, with an incremental cost-effectiveness ratio of $8231.87/quality-adjusted life-years.4 The analysis highlights the importance of considering cost-effectiveness when making treatment decisions.
Looking Ahead
The first year of cladribine availability in Brazil represents a significant step forward in MS care. However, realizing its full potential requires a concerted effort to address regulatory limitations, promote education among healthcare professionals, and incentivize wider adoption. Collaboration between healthcare authorities, clinicians, patient organizations, and the pharmaceutical industry will be essential to ensure that all individuals with MS in Brazil have access to the most effective treatments available.