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EF: What is your review of 2023, and what are the new opportunities and priorities planned for 2024?
NL: From Ultragenyx's standpoint, this year had two noteworthy aspects. The General Health Council approved three therapies we submitted for reimbursement. It is commendable that the government is taking the lead in advancing innovation for communities affected by rare diseases, especially considering the time usually required to navigate permissions in the HTA environment with gatekeepers like the General Council. The fact that major government decision-makers acknowledge the value innovation brings to patients with rare diseases within the healthcare system is encouraging. Government institutions are voting in favor of assisting the medical community in providing patients with cutting-edge and new therapies.
Another noteworthy milestone is the reimbursement for one of the primary X-linked Hypophosphatemia (XLH) disease products. When therapy becomes available, payers are willing to pay for it and treat patients, which has the power to significantly improve the patients' lives.
The innovation and value proposition of offering transformative treatments to targeted communities has been positively received. Nevertheless, it has not been easy. Participation has been limited to individual patients or hospitals, and having a small team makes it difficult to follow up on each case. The reimbursement model remains complex, causing delays in patient access. We are doing our part by compiling these pharmacoeconomic models and value proposition propositions and regularly forwarding them to the physicians, hospitals, and payors. As long as everyone performs their duties, innovation can reach patients even though it requires many steps.
EF: What are your thoughts on nearshoring in terms of R&D and potential investments that can be made in the country to foster innovation?
NL: Near-shoring R&D in Mexico is a significant direct investment opportunity for the country. With the proximity to the United States, a hub for major R&D initiatives, collaboration among all stakeholders is crucial for promptly introducing innovation to Mexico.
The R&D companies are in conversations with the government stakeholders to increase the potential for larger R&D investment in Mexico. The country offers a significant possibility because of its size, the caliber of its healthcare workers, and the prestige of some of its major institutions. The debate focuses on streamlining our R&D study submission process to ensure faster data dissemination without overlooking any details.
A lot of companies are planning to open R&D sites in Mexico, increasing investment. However, comparing timelines with Argentina and Guatemala poses challenges. Our aim is to help governments, organizations, and medical facilities seize opportunities. The first location to open will benefit from a first-come, first-served policy.
We will have to see whether COFEPRIS' new digital initiative accelerates protocol submissions for our research and development. Authorities recognize our appetite and the opportunity we represent.
EF: In the context of big pharma moving into niche products, what advice or guidelines would you offer business executives seeking to create a value-based product line?
NL: Encouragingly, major pharma companies are shifting towards niche therapeutic areas and rare diseases in their R&D and product lines. After 20 years in this field, it is good to see that their entry positively impacts funding, policies, and access processes. Diagnostic programs are a part of this value proposition, as frequently discussed with the General Health Council and payers in Mexico. In rare diseases, ensuring proper diagnosis, biomarkers, and specific mutation tests is crucial. Stakeholders need to understand the value proposition, especially considering the substantial investment per patient.
Patient Support Programs (PSPs) aid in monitoring patients during their hospital stay, ensuring therapy adherence, dose consistency, and accurate result measurement. These programs act as extensions for doctors and institutions to track and analyze patient progress comprehensively.
Discussions also involve gene therapies incorporating devices, delivery systems, and pharmacogenomics, which link gene variations to medication metabolism rates. Our approach encompasses holistic healthcare solutions from diagnosis to treatment; we do not just sell medicines.
Because rare diseases and high-specialty cases are typically fatal, degenerative, or chronic, early diagnosis is crucial and makes medical education even more important. We lose valuable time if the diagnosis is delayed. By consistently educating and raising awareness of diseases within the medical community, we can provide additional value to the patients, the community, and the system as a whole.
EF: How do you envision the future of diagnostics, and how have you expanded your collaborations in Mexico to keep growing access to diagnostics?
NL: The diverse genomic variability across countries is attracting new health-related businesses to Mexico and Latin America. Certain communities with higher rates of specific diseases were previously overlooked. However, the well-established rare disease market in Latin America in the past 20 years has drawn attention to these communities.
More players contribute to more learning opportunities. The first approach is to invest in medical education because more individuals are becoming interested in certain diseases. We are currently discussing rare diseases with pediatric residents. After witnessing patients receiving care in places like the National Institute for Pediatrics or the Children’s Hospital Federico Gómez, these medical professionals will return to the small towns with more knowledge, establishing the foundation for future efforts to increase awareness of rare diseases.
The major challenge lies in enhancing the availability of diagnostics. Although the costs have been reduced, genetic tests still range from $500 to $1000, making them unaffordable for many in Latin American countries. Expanding local labs with sequencing technology could bridge this gap and provide genetic testing locally. The plan is to integrate this test into the healthcare system, serving as a crucial step towards early diagnostics with additional resources.
Regarding digitalization and AI, the pharmaceutical industry has embraced technology more slowly than other sectors. However, AI models have shown promise in breast cancer diagnosis, improving accuracy with each iteration and highlighting its potential for early detection of rare diseases. Mexico could still improve in this segment. When integrated with medical records, AI could revolutionize diagnostics, especially for rare diseases. Algorithms can analyze patient data from electronic records, aiding in early diagnosis based on signs, symptoms, and test results.
Social media is another tool that has developed throughout time and helps patient communities connect with one another. As individuals began asking questions on blogs or other platforms, several patient associations started to form. Acknowledging that even in the absence of an electronic record, everyone has access to smartphones, we need to adopt new technological assets. A new generation of early rare disease diagnosis may be possible if we learn how to use these instruments.
Moreover, high-risk population screening, conducted in places like dialysis centers or renal clinics, proves effective in diagnosing certain rare conditions affecting specific groups. Additionally, for X chromosomal conditions, family testing becomes essential to identify undiagnosed relatives lacking access to early detection.
EF: Which skills are you searching for in new hires, and how do you attract and retain them in your organization?
NL: We aim to find a versatile skill set covering various access and reimbursement procedures within the complex Mexican healthcare system. Instead of traditional roles, we seek a high specialty representative with a broader scope that goes beyond physicians or adapting to hospital policies—a more holistic strategy.
This hybrid role blends representative and KAM responsibilities, collaborating closely with MSLs and key account managers to devise a broad and comprehensive medical and institutional engagement. This approach has proven effective in our small business, where job roles are less defined due to our size. We are seeking individuals committed to a comprehensive approach capable of seamless transitions between functions and avoiding silos. Our attention is on the patient.
EF: What final message would you like to share for 2024?
NL: Election years typically present significant financial issues for the healthcare system's administrative procedures. It is crucial to collaborate closely with healthcare facility doctors and administrative staff to ensure patients maintain continuity of care.
We anticipate portfolio expansion in 2024 and HTA approvals for reimbursements, enabling treatment for new diseases. Changes to the purchasing system or the implementation of IMSS-BIENESTAR might lead to significant adjustments that can have an impact on patients, therapy continuity, and reimbursement processes. Stabilizing the system is crucial for all healthcare professionals and organizations. It is my request that the outgoing and incoming government administrations properly understand the rules of engagement and refrain from making significant changes every year, as this causes severe disruption. Despite potential challenges, I think 2024 will be a good year for healthcare in Mexico.
