Read the Conversation
EF: What was the mission you were given when appointed last year?
NL: I was appointed in 2019, a transition year for the country with the change of government, to start up operations in Mexico. Ultragenyx had a presence already in Colombia, Brazil and Argentina, as market access is quicker in those countries, and in Mexico it is more sophisticated to get started. Our mission in Mexico is to bring our therapies to patients in need and to this end we are launching two products for rare diseases -our focus-. The first for X-linked hypophosphatemia and the other one for MPS VII. Before COVID the plan was to build a platform to support the diagnosis of these rare diseases. We set up a diagnostic program providing healthcare providers and institutions free testing for suspected cases of MPS VII and since we detected a few patients with a confirmed diagnosis. Due to Mexico’s size, we know there are a lot more cases so we have to put together a medical education program with physicians for them to understand which patients to profile and test. We have also started the regulatory pathway to get approval as orphan drugs designation for the two products, but due to the pandemic most of the government institutions have been stopped or downsized with the stay at home policy. Our biggest challenge is to keep continuity, and our short term objective is to create awareness as even with the pandemic there are other patients that need help. In spite of the uncertainty with COVID we are still building the team –sales reps, etc.-as we know the situation is temporary and we need to be ready with the right people in the field as healthcare must go on. This is why we keep lab testing up, our diagnostics program is still running and if any case needs to be tested our partner in diagnostics, QUEST, makes sure it happens, picking up the test at the patient's home and taking it to the lab. Diagnostics are vital for detecting and treating rare diseases.
EF: What is your personal definition of access?
NL: Every country has a different pathway for access. Specifically in rare diseases, access is very dependent on the healthcare system, so it is important to show the government of the medical value we bring to the healthcare system. In our therapeutic areas, it is about our excellent treatments and management of patients in the long term, providing value to the system. We want the government to understand the importance and value these medicines bring to patients. We offer diagnostics, treatments, therapies and additional interventions needed around patients with rare diseases and it is quite a challenge because the resources are limited and authorities can have other priorities. Some of our therapies are very much needed and means the difference in between life and death for our patients. The discussion is complicated as orphan drugs tend to have higher prices than standard drugs but we try and get the best treatment or patient management possible via the government's possibilities and allocation of resources.
EF: Does rare disease awareness exist in Mexico and what can you do to increase this awareness?
NL: For the last ten years rare diseases in Mexico have been part of the healthcare agenda and that is growing. Patient advocacy groups have driven changes within the regulatory and healthcare agenda to the extent that now there are specific laws and regulations for patients with rare diseases and though there is room for improvement in the processes there has been an evolution in the rare diseases industry over the last years. Today for example in a hematology service all the resident doctors know about Gaucher's disease, whereas 10 years ago they did not even know it existed, so there has been an important journey made through the learning curve. Market access is still a big issue, as it takes 4 to 5 years to get a drug approved for a rare disease due more to market access processes than COFEPRIS and regulatory agents. That gap needs to be closed with the early adoption of innovative therapies. The challenge for increasing medical awareness is to shorten the time of diagnosis. On average a rare disease patient takes 8 to 10 years to be correctly diagnosed taking again on average 10 to 12 specialists before they get an accurate diagnosis. It all boils down to medical education on different rare disease symptoms, how to diagnose and do quick referrals to geneticists or find support with other specialists that can help with the diagnosis. There is a project in the house of deputies and in the senate at the moment to screen newborn babies very easily with a spot of their blood, testing them for multiple diseases. Early treatment changes the outcome of the disease especially when they are discovered at such a young age. There are a lot of gaps to close although the work done by pharma companies, medical societies and patient advocacy groups have shown progress in the healthcare agenda.
EF: What is Ultragenyx’s ambition for Mexico?
NL: Our mission –also our ambition- is to provide needed therapies to the Mexican patients. ULTRAGENYX brings a very interesting portfolio of innovative and gene therapies which are the future of healthcare. Our pipeline is for rare diseases treatments and our long term challenge is to reach every single person in need of treatment in Mexico and give them the adequate care for their condition and to do this we have to work very closely with the government proving our therapies work. Finally we must invest in Mexico. We have a big country with a thriving economy even with the recession that is coming. It is an interesting market and it makes a lot of sense to continue investing here. The first step is to get market authorization and bring the first therapies to market which will be the building blocks to bring future innovation, revolutionary therapies and innovative solutions. After Brazil we are the second biggest market in Latin America.
EF: What is your advice to other managers in regards to managing in times of transition?
NL: My analysis of the sector is that in general healthcare is a very inelastic sector, because healthcare must always go on. Even in times of a pandemic there will still be patients with hypertension, diabetes, with chronic diseases, oncology patients, etc. The allocation of resources is important and a challenge at the moment because with COVID we are not sure how the resources will be distributed so we must fight and negotiate for our patients to bring value to the equation and get treatment for them even when COVID is center stage. General management of the crisis is key as is the management of employees and patients who are trying to reach their physicians and are frustrated in their attempts. I am in constant communication with our team to keep them motivated for the long term with a short term strategy for the crisis trying to understand its impact –we won’t change our long term objectives but we might have to adjust the pathway to get there- this is a temporary situation so you need to continue investing, hiring, launching products, and have continuity of our activities is ensured for the long term objective. It’s a balance between a tactic and strategic approach in a crisis.
EF: What attracted you to Ultragenyx?
NL: I started my career in Jannsen with rare diseases and they have a compassionate philosophy when addressing rare diseases with a very strong social objective -it is not only about selling- and I learned a lot there of the social importance of healthcare. When I came to Ultragenyx I had the opportunity to apply some of Jannsen’s patient driven principles, one of the key pillars of the company is responsible pricing for rare diseases treatments, we balance the right price for the right therapy for the right patient which is a system which has been very well received by the US payer and we are hoping this message will resonate in Mexico and in the region making access a bit easier with our innovative products because the priority is to get the therapies to patients. This is a hugely important point, “walking the talk” with patient centricity and compassionate use programs was the biggest reason I was drawn to work in Ultragenyx as was the opportunity to build a business from scratch in Mexico which is a professional challenge. Creating and helping to materialize what is needed in Mexico; executing and fine tuning our vision for Mexico is very exciting especially as I started from scratch. It’s a motivating and exhilarating challenge for me as well as a big responsibility, it will be hard to align expectations but definitely worth my time.
EF: How difficult is it to hire talent, build a team and retain it?
NL: One of my initial concerns was the capacity of bringing talent to a company which was just starting up but Ultragenyx’s reputation built a strong case in our favor. When I posted a couple of ads to hire people the answers were massive. I put this up to the company’s reputation and the fact that our ´to be´ employees wanted to grow with the company, they are betting on us to grow and progress along with us. This sort of motivation brings real talent and enthusiastic people to the team and I think we are doing a great job right now. I employ 44 people at the moment and plan on hiring 8 more this year with a plan to continue growing responsibly.