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EF: In the post-Covid times, executives must deal with complex multi-faced economic and political scenarios; even some big companies are restructuring and reassessing their footprint. How do you visualize 2023 for Ultragenyx; do you see it as a challenge or an opportunity?
NL: I see it as an opportunity; Mexico is yet to establish a stable purchase process, particularly for the government sector; a few strategies have been attempted creating a gap in formal procedures, especially for new market therapies. It has been challenging for innovators and new products to access the healthcare system, understand how it will be implemented, and incorporate innovation into the public sector. The good news is the interest from some stakeholders within the public sector to advance in adopting new therapies. Even companies bringing established medicines face issues, and for new therapies, it is even more complex. We are in a niche market, and our patient population is very small, so we can bring in orphan drugs as a US pack, avoiding challenges in addressing demand and production. The demand is easier for us to predict based on the small number of our patients and vials needed; however, a clear pathway to the patients is what we strive for. Previously, we presented a dossier with our realistic expectations for the market and received authorization, arriving at our goal: the patient. Innovation should be facilitated by agencies such as COFEPRIS or the Consejo de Salubridad General in order to go to the Mexican patients needing it.
The government is again approving rare disease therapies. Recently, there has been some action on rare diseases in Mexico; the last Consejo de Salubridad General meeting approved the reimbursement for a key therapy. Four to six new orphan drugs have been incorporated into the Orphan Drug Rare Disease List, which is very short and limited, signaling the government is retaking interest in rare diseases and orphan drugs for a usually neglected population. It is great to see some traction on reimbursement processes and the update of the rare disease list.
EF: Last year, when we spoke, you mentioned recognizing innovation's value and shortening diagnostics times. How do you assess progress over the last year, and is Mexico moving in the right direction regarding two issues?
NL: Innovative therapies give patients real solutions for previously unmet needs. Life-changing therapies are well-perceived by the government and the payers, especially when documented and upheld by the outcomes confirming the value of innovative treatments. We still have to fight for budgets, allocations, and resources; we even have to fight for the patients initiating therapy earlier in their disease. We are constantly against the clock as we navigate a very complex system in the most efficient way possible to shorten time to patient access. As for earlier diagnostics, rare diseases still face the same challenges, but there has been progress in some disease areas. Since a couple of big educational centers where rare diseases are diagnosed are available in Mexico, there has been a learning curve in some recognized pathologies resulting in more timely diagnoses. There also is a better offer of diagnostics and molecular and genetic tests, and prices have fallen, making access easier. Still, there is a lack of genetic and molecular testing within Mexico; many tests must be sent abroad. Ideally, testing could be provided by the Mexican health system and available broadly and not have to be set up differently.
EF: With six gene therapy treatments in the pipeline, Ultragenyx, like other companies active in the same space, is hoping for a clear regulatory path for the approval of gene therapies. How do you anticipate introducing them in Mexico, and what will their impact be on the Mexican population?
NL: Gene therapies are next-generation treating genetic diseases; the science is complex, and we are still working on clinical studies. Our first therapies will initially be marketed in the US and then expand into more regions. The pathway is still under discussion, and companies with gene therapies in their pipelines and AMIIF are developing a regulatory proposal to present to COFEPRIS. We are still working at a stakeholder level, figuring out the best way to do it, and taking ideas from ANMAT, ANVISA, and other successful regulations already in place. The established orphan drug pathway in the COFEPRIS guidelines could be an alternative for new therapies that address orphan diseases and the path to getting market authorization. However, the big question to be yet answered is reimbursement. We will discuss this topic with the Consejo de Salubridad General because having innovative market access strategies with payers, etc., needs building. The broad discussion has not happened yet, and we need to bring more stakeholders to the table to be ready and better sooner than later, as the therapies are around the corner. Hence, the discussion needs to be accelerated. It is a matter of balancing our mid-term priorities versus immediate priorities, but the regulatory framework is a must because they are the next generation of medicines. The same parallel can be drawn when we migrate from chemical drugs to biotechnology, with the regulatory system and ecosystem adapting to the change.
EF: What were the most important or interesting takeaways from Innovation Week for an innovation-driven company like Ultragenyx?
NL: The pandemic was conquered due to innovation and rapid associations between small development companies and big pharma organizations, accelerating bringing of the vaccine to the people in an affordable fashion. The industry has this capability; we can work together and scale up when necessary to provide solutions. There has also been innovation regarding patient centricity, using phone application models and functions during the pandemic to broaden patient services. There are applications for medicine-taking, treatment adherence, hemophilia to manage the disease at home, and many others that make life easier for patients. As mentioned at Innovation Week, Mexico has the opportunity to participate and become a hub for clinical research. In the specific rare disease world, many rare diseases have been recognized in different communities and patients diagnosed. Pharma companies are interested in having Mexican sites participate in face-to-face trials, -a huge opportunity- and rare diseases only need a few people. We must implement an agile system to bring more R&D to Mexico. Many centers drive innovation here, but we must support and bring the pieces together to make Mexico attractive. I refer to research, developing, investing in opening sites, and working with investigation hospitals. Considering our neighbor to the north, it is a kind of R&D nearshoring; many clinical studies and branches could be opened just south of the border. It could be a direct investment in the economy; we are working to bring R&D directly to the country.
EF: You manage Mexico and Central America; how do you assess the regulatory perspective in the region?
NL: Thankfully, it works for Mexico's orphan drug designation process. The regulatory entity that assesses orphan drugs has worked within the timeline. I know there have been some challenges in other areas, but in our case, they have supported bringing innovative orphan drugs to the country. COFEPRIS is a reference for Central America, as is the FDA and INVIMA of Colombia, so there are many opportunities in Central America to rely on other agencies. But there is market access and the regulatory aspect, so it is good to have alternative pathways for orphan drugs. Because these drugs are specific, it is good to have a different path, a good practice that has been taken from the FDA, Europe, MENA, and Australia.
EF: If you had to create your own startup company tomorrow in the Mexican healthcare sector, what would it be and why? The hypothetical question is to understand where the opportunities lie in Mexico.
NL: Based on my experience and what I see as a healthcare gap, I would continue working on orphan drugs. Many opportunities and innovations are not considered in Mexico due to the country's complexity, the fragmented system, and all the market access steps between the regulatory and the end patient. There are opportunities to bring additional therapies to rare disease patients, and it is not like competing in diabetes, where there are a lot of big players already in a well-covered market. In rare diseases, some patients have nothing, not even in the pipeline for their diseases. I would continue on the same pathway because of the opportunities and because there are many patients to be served.
EF: Our feature is called "Road Map to the Future," if you had to create a road map for the healthcare industry in Mexico or Central America, what would be your three base pillars?
NL: Mexico needs to regain an open dialogue with the government. There are still many healthcare system issues; the pharma sector is seen as a stakeholder, but we need to engage in a constructive dialogue on building the future together. It is important to work on this; as an industry, we can bring value, innovation, and solutions. That would be the angular stone of my Road Map to the future. Secondly, we are in the 5th year of the current administration we urgently need clear processes and rules of operation. We need clear, straightforward, transparent rules with clear step-by-step procedures. Last but not least, I should have started with this pillar, as it must be a priority of all stakeholders and is what the patient needs. The gaps in the system must drive the budget, purchases and innovation, and even the clinical trials. Patients that need care and are not getting it, we need to work around budgets for their actual needs starting with R&D and bringing mild innovation to underdeveloped markets. The market is the patient, and patient centricity is more than a slogan.
EF: Is there any final message you would like to share?
NL: We are trying to build partnerships; we have been working on partnerships with payers, hospitals, state health secretaries, and IMSS that are open to dialogue on a broader level to bring solutions to healthcare in general. The current administration is in its final phase, the transition is coming, and we must act now. We are only a couple of months away from the presidential campaign kickoff, and many healthcare issues will not be solved by the end of this administration. We must find a way to work with the candidates thinking of the near-term future addressing healthcare topics, such as supply chain shortages, coverage of the present needs, access reimbursement, etc.
