Read the Conversation
Meeting highlights:
- 2025 Objectives for Ultragenyx Mexico: Focus on growing their portfolio of orphan drugs for rare diseases, securing market access and reimbursement, and integrating rare diseases into national healthcare strategies through government purchasing.
- Advancing R&D and Regulatory Collaboration: Mexico’s regulatory landscape and proximity to the U.S. position it as a priority market for orphan drug designation and clinical trials.
- Strengthening Healthcare Data and Diagnostics: Partnerships with private insurers and diagnostic labs address gaps in genetic testing and patient identification, leveraging AI and data analytics to improve disease detection and outcomes measurement.
- Vision for the Future: Ultragenyx seeks to transform the rare disease model in Mexico by expanding access, introducing gene therapies, and reinforcing its leadership in orphan drugs, driven by patient-centred collaboration and systemic improvements.
EF: What are your main priorities for 2025?
NL: 2025 marks a pivotal moment for Ultragenyx Mexico as it continues to develop and grow its portfolio of orphan drugs for rare diseases. This involves regulatory registration, marketing authorization, and a critical focus on market access and reimbursement. Securing inclusion in Mexico’s national reimbursement plans will ensure broader access to these essential treatments. The transition to government purchasing processes for orphan drugs is particularly promising, as the government increasingly incorporates the rare disease community into its healthcare strategies.
Balancing affordability, cost, and access remains challenging, especially for rare diseases. However, Mexico's evolving reimbursement process, supported by state backing, offers a promising shift compared to models in other countries. A patient-driven approach is essential—by addressing the specific needs of a neglected community, the value of these treatments becomes clearer.
Growing physician awareness and productive collaboration with payers and government entities underscore the transformative potential of these medicines. Aligning all stakeholders around patient well-being bridges the gap between innovation, access, and affordability, paving the way for delivering cutting-edge therapies.
EF: You recently attended the US-Mexico Binational Health Development Forum. What were the key outcomes, and how can this collaboration drive innovation in your field?
NL: Internally, we are excited about Mexico's potential, particularly in the regulatory landscape for orphan drugs. As a priority market, we plan to include Mexico in the first wave of submissions for orphan drug designation following FDA approvals, reinforcing its role in our innovation efforts.
Expanding clinical research and R&D activities in Mexico is another critical focus. With its proximity to the US, strong investigator networks, and established reference centers, Mexico offers tremendous opportunities to advance R&D and host more clinical trials. We are already investing significantly on a global scale and see the potential for directing a substantial share to Mexico, fostering local R&D, and driving the development of new therapies.
Orphan drugs play a pivotal role, as Mexican patients have participated in clinical trials, demonstrating how these efforts can transform lives. Beyond direct patient benefits, this positions Mexico as a key platform for innovation. Stronger US-Mexico collaboration will further enhance access to transformative therapies and attract greater investment in the country’s healthcare and research infrastructure.
EF: What are the most critical steps to foster an innovation-friendly environment in Mexico?
NL: COFEPRIS plays a pivotal role in fostering innovation, with its focus on digitalization broadening its scope. A key priority is establishing a fast-track approval mechanism for drugs already approved by reference agencies like the FDA or EMA, particularly for Orphan Drugs.
However, COFEPRIS has room to improve its technological capabilities and streamline processes. In rare diseases, regulatory pathways exist but still include grey areas. Collaborative working groups are addressing these gaps to create a clearer, more efficient framework, reducing submission and uncertainty.
Meanwhile, COFEPRIS also has an opportunity to lead in advanced medicines like gene therapies, which represent the future of healthcare. As these therapies gain global traction, collaboration with regulatory bodies is essential to establish a framework for their introduction in Mexico. Our company, with an extensive gene therapy pipeline, relies on a seamless regulatory process to bring these innovations to patients.
For rare diseases and orphan drugs, discussions are ongoing about differentiated evaluations, now expanding to advanced therapies. Their potential curative nature raises complex questions around pricing, HTA evaluations, and regulatory submissions that need careful consideration.
Finally, Mexico has significant potential to attract R&D investment. Expanding R&D sites would not only drive innovation but also position the country as a leader in developing and adopting cutting-edge therapies.
EF: Do you see rare disease treatment progressing positively, especially with the private sector driving growth in clinical labs? How are you collaborating with them?
NL: The private healthcare market in Mexico remains significant, with private insurance covering around 11 million people, including 5 to 6 million with high-tier platinum policies. This creates opportunities for healthcare providers, even in niche or rare disease areas, as many patients may eventually transition to the public system. To address this, Ultragenyx is partnering with insurance companies to demonstrate the value of ensuring proper care for these patients while still under private coverage.
Additionally, there is a growing demand for diagnostic labs, particularly for molecular and genetic testing, which is not widely available. By partnering with local labs, Ultragenyx is helping fill this gap, enabling more comprehensive testing solutions, including genetic panels and diagnostic tools for confirming medical conditions.
We also collaborate with labs to collect testing samples, which are sent to various labs worldwide. These labs have extensive data and work with us in an academic and scientific capacity. The amount of data, diagnostic labs gather, poses a good opportunity to analyse and shorten the diagnostic journey of rare disease patients.
With the explosion of AI solutions, we will need to wait a few years to see which ones bring real value. While there is excitement, we aim to focus on long-term value for medicine, healthcare, genomics, and analysis. We are also exploring AI applications in study design and molecular development, with potential impact in areas like protein conversions and analysis. In R&D, AI certainly has the potential to make an impact.
EF: How can Mexico harness data as a resource to drive growth and make a meaningful impact on health?
NL: There are significant opportunities in this area. Recently, there was communication about digitalization in healthcare, particularly in public healthcare, highlighting the importance of electronic medical records as a critical first step toward building a robust knowledge base of medical data across institutions. One major challenge in Mexico is the lack of connectivity between health systems. For example, when someone moves from IMSS to IMSS Bienestar or ISSSTe, after losing their job, their medical records do not follow due to the absence of a unified system. A project to implement digital records in the public sector, integrated with the private sector, would address this issue.
AI tools can add significant value here. Algorithms can analyse data to identify potential rare diseases by flagging patterns in symptoms, enabling timely referrals or specialized care. Diagnostics often burden healthcare systems, but electronic records combined with AI analytics could expedite the process, leading to earlier and more accurate interventions.
The key is to start with the basics: a universal, portable digital health record system in Mexico, owned by the individual, ensuring continuity of care across healthcare systems. Robust data would allow for effective outcome measurement.
When we discuss payer investments, it's not just in pills, biologics, or infusions—they are investing in outcomes and value. Proper data enables accurate measurement of these outcomes, ensuring alignment with Phase III trial results or the proposed value of a drug.
This data-driven approach validates treatment effectiveness and confirms that promised outcomes are being delivered, which is crucial for maintaining trust and demonstrating value in healthcare.
EF: What are the top three emerging trends shaping the future of healthcare sustainability in Mexico?
NL: Sustainability is a major concern, requiring the resolution of key macroeconomic and systemic challenges. One of the biggest issues is Mexico’s historically low healthcare investment as a percentage of GDP, among the lowest in OECD countries. This underinvestment creates a significant resource and capability deficit. Addressing this requires medium- to long-term planning to secure more robust healthcare funding and broader access to services.
Efficiency is another critical factor. The current system has numerous regulations that, if navigated effectively, could lead to substantial savings and better resource allocation. Optimizing the existing framework without introducing new processes or regulations can unlock value and maximize the impact of limited healthcare expenditure.
A third challenge is the fragmentation of Mexico’s healthcare system. The multi-institutional design often creates inefficiencies, such as patients needing to travel long distances for specialized care. Greater integration and universal coverage could streamline resource management and improve accessibility, contributing to the system’s long-term sustainability.
EF: Why is investing $1 in rare diseases in Mexico more valuable than in other countries in Latin America or globally? What makes Mexico the ideal destination for such investments?
NL: The Mexican healthcare system is evolving and increasingly addressing patient needs, particularly in the rare disease space. With a large population of rare disease patients, Mexico is an ideal location for investment and development.
Mexico's rich genetic diversity provides a unique advantage for clinical trials and studying rare diseases. Third-level hospitals and specialized care centers, such as those focused on cardiology or nutrition, serve as hubs for identifying and managing patients with rare conditions. These facilities enable better mapping of patient populations and offer structured pathways for addressing rare diseases.
Another advantage is Mexico's centralized healthcare system, which, despite challenges, offers a clear framework for engaging with government entities on health technology assessments (HTA) and reimbursement negotiations. This approach is similar to models like the UK’s NHS or Canada’s CADTH, enabling innovation to reach a large patient population through streamlined government collaboration.
Mexico’s strategic relationship with the U.S., and stable economic growth, position it as a key player in the region. While growth may be steady rather than rapid, it remains closely tied to the U.S., offering a reliable investment environment.
EF: Looking back, what would you consider your greatest achievement? Looking ahead, where do you envision the company five years from now?
NL: One of our greatest accomplishments has been reinventing the rare disease and orphan drug model in Mexico. We focused on building partnerships with patient communities, healthcare practitioners, payers, and the government to shift the conversation toward value and align efforts to provide proper patient care.
Previously, the conversation around orphan drugs often centered on pricing, small patient communities, and legal disputes over treatment access. We took a different approach by adopting a constructive, patient-centric model. Over the past five years, we've brought three therapies to market with full access, reimbursement, and inclusion in the National Medicine Compendium. This pace has exceeded the norm, reflecting our team's dedication.
Looking ahead, we aim to continue growing in the orphan drug space and solidify our leadership position in Mexico and globally. Our goal is to be recognized as the company that transforms care for rare diseases, focusing on innovation and differentiation. A key priority will be introducing gene therapies to Mexico, expanding our portfolio and pipeline, and ensuring no patient is left behind.
Ultimately, our mission is to serve patients and collaborate with stakeholders to ensure all those in need of treatment in Mexico have access.
