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EF: Looking at 2022 in the context of the past two years, 2020 was the year of diagnostics and prevention, and 2021 was the year of vaccines; what do you think 2022 will bring?
NL: We are seeing a return to the care of chronic disease patients in 2022. During the pandemic, patients didn't see their doctors for fear of getting Covid or because the hospital services were redirected to Covid. There is now a transition back to care, and there has been a progression in the diseases. The biggest challenges we face in rare diseases are an additional delay and backlog in the initial rare disease diagnosis and a rebalancing of resources for funders. The healthcare systems are still massively vaccinating people with second, third, or fourth doses, and, even so, resources for healthcare and chronic diseases must be allocated. Having to stretch the resources for more needs is an extra challenge, this is where we are.
EF: What do you think about the advancements that are being made in rare diseases in terms of approval?
NL: There is very exciting news. The FDA announced a program to handle medicines for rare diseases. This announcement will be a fast track for rare diseases, giving us more focus, momentum, and investment. In the near future, 30 or 40% of the medication coming to market in Mexico will be for rare diseases, ultra-high speciality drugs, or new age therapies such as gene or cell therapy. This healthcare niche is receiving wide attention as traditional big primary care players shift to high speciality drugs and therapies -specializing in oncology, immunology, and rheumatology. Innovation is moving from high-speciality to even higher speciality and markets such as rare diseases; gene and cell therapies are the new future of medicine. Rare diseases have been a neglected area in healthcare, so it is good news, and the shift is exciting. Physicians don't know how to diagnose rare diseases in a timely manner, and even though awareness has gradually grown, diagnosing is still complicated and a challenge. The journey to diagnosis is long, and many pharma companies have not invested in therapeutic solutions because of the small number of patients. The FDA's initiative creates incentives for investments, development, and innovative therapy programs for diseases and for the people who have been without any solution whatsoever; it also highlights the value of innovation and the capacity of the pharma sector. There is the question of how to replicate this initiative in the region and reflect the acceleration in COFEPRIS, using reference agencies that have done a good job evaluating and bringing the therapies quicker to the country with this validation. Funders must then adopt the technologies and bring them to the patients as soon as possible. I hope there will be more attention and focus on our community to replicate solutions and make innovative therapies come sooner to the patients.
EF: What is needed to strengthen the focus on rare diseases in Mexico?
NL: We have to bring value to the discussion and forget about the individual pricing that has always been a challenge in rare diseases. The individual cost of treating a patient is still a big barrier for funders as they don't understand the value innovation brings to the rare disease community. Rare diseases mostly have genetic origins, affecting maybe one or more persons in a family, and they are usually progressive and debilitating. The patients need caregivers, and more often than not, a family member must quit a job to care for the afflicted person. Due to genetic factors, unfortunately, the low-income population is the most impacted. Considering all these factors, we offer the patients the alternative of having normal lives and being productive, allowing the caregivers to return to work, bringing value to the equation, and changing things completely. Diagnosis remains the biggest challenge for the patients; it can take seven to eight years to diagnose a rare disease, seeing eight to ten different specialists until patients are correctly diagnosed. There is a lack of deeper education within the healthcare community, and there are ways of battling this. Newborn screening in our public institutions to diagnose early has a completely different outcome: avoiding the complex eight-year journey of a progressively deteriorating disease until diagnosis. The two key factors we need to work on in Mexico are i) the value of innovation and ii) the shortening of the time to diagnosis.
EF: As a business leader, what innovative strategies are you working on to add value to the business?
NL: At Ultragenyx, we put patients first; it is our DNA and is part of the added value we offer. The discussions with the institutions and payors, regulatory agencies, physicians, and the patient community focus on the patients. We have to quickly bring the products to the market, employing innovative alternatives in clinical trial designs. For example, the numbers in rare disease trials are always small, and it's hard to find big samples and control groups. An innovative study design approach for rare diseases is to have an escalated introduction of patients treated with one patient while controlling another. The second patient delays his treatment to control the first, and so on, with the patients progressively getting lifesaving therapy and having a control group. Interestingly, the patients and regulatory agencies have accepted this innovative trial design to bring therapies to the market faster. We have a treatment for a rare disease that causes bone deformities, and after some months of our treatment, the bones return to their original state. We demonstrate our product's value by showing payors how we can stop the progression of the disease through therapy, thus adding value to the disease and patient's life, and it is a powerful message to bring to the table. Funding the patients' budget is very important because we need to allocate resources for these therapies. We sometimes offer compassionate use of our therapies to show how the treatments work for our funder to find the budget for the next year.
EF: What trends do you see in this area for the coming year in Mexico?
NL: I believe the General Health Council (Consejo de Salubridad General) is open to innovation and understands the importance of innovative value. There is interest in including our treatments in the Compendio Nacional de Insumos is a very favourable consideration that brings innovation and value to patients and government institutions. The physicians understand there are alternative treatments and are willing to treat once they know the value these therapies bring to patients. The next step is relevant to the government's centralized purchase process changes and will be a real challenge. The government has made many changes in the acquisition and purchase process, demanding certain flexibility. The funders do not understand how to incorporate the innovation changes into their current budgets, so there is a missing link that remains a challenge. As the challenges are more on the logistic and operational side, we are optimistic. The government has to figure out, for example, engaging with payers to provide innovation. We can take away a lot of positives among the many challenges yet to be resolved. I believe the health system is navigating through the adequate purchasing process learning curve.: The complexities reside mostly in that the government has changed the purchase process three times in as many years; they started by announcing changes in the purchase process led by the Minister of Economy. UNOPS was brought in the next year to handle some part of the equation, and the third year also came with new adjustments. It is very difficult to plan when changes are constantly made. We still don't know if there will be further changes as there have been issues with the distribution of some medicines not getting to hospitals at the end of the chain. It has been a trial-and-error process, but we need to plan for products and the supply chain at least a year in advance. Instability in the purchase environment is never good for long-term planning.
EF: Could you elaborate on the performance of the Mexican operation and its relevance for Ultragenyx?
NL Mexico is one of the most relevant markets in Latin America. The number of patients is significant due to genetic conditions and population. There are many patients to serve so Ultragenyx is committed to bringing our life-changing orphan drugs to help the community. We are just starting to see the first patients both in private and public institutions getting treated with very encouraging clinical outcomes, and while we navigate through the reimbursement process of other products, we will continue providing compassionate use medicine to help Mexican patients and their families.
EF: Education is essential for physicians to adopt and absorb rare disease education. How do you see the adoption of digital in the different generations of physicians?
NL: There are digital trends for physicians, zoom meetings, WhatsApp, symposiums, congresses, etc. Covid accelerated everything digital, and there was initial excitement with high remote attendance, but we went too far in that area, and now we are returning, at least to a hybrid model. It is a good balance, and physicians can decide whether to attend in person or virtually. I believe that if a relationship already exists between a sales rep and a customer or physician, it is easier to transition from face-to-face to digital. Building a digital relationship from scratch is much harder without that previous relationship. Regarding other programs, we are paperless, and a digital platform is a more efficient way of presenting the company story, studies, and products. Pre-pandemic, it was 50-50, and now it is 90-10% of digital over printed information. Our biggest progress has been in rare disease diagnostic programs and requesting genetic tests on a platform. We have digitalized very successfully and made the process more efficient. Hybrid is a fair balance and the way to go.
EF: Considering the hybrid model, how do you maintain the workplace culture with the new model?
NL: The same way we did with the physicians, many interactions can be digital, operational meetings can be easily done digitally, and it makes sense to continue the trend in sales and KPI follow-ups. But face-to-face is much more productive for discussing strategy and bouncing ideas for the future. In the first scenario, we follow an agenda, but personal interaction is more effective for dynamic meetings where we think, plan, and strategize. Personal interaction is important for both employees and customers. We are very culture-dependent in Mexico, a lot of business is done around food, and we are very interactive with our customers. Recently we travelled to Mexicali to meet a customer for a 30 minutes breakfast, which became a three-and-a-half-hour meeting. We first went over the agenda topics and then started bouncing ideas off each other. Virtually this customer-building relationship interaction would not have been possible.
EF: In the changing scenario, what are the new skills necessary for the future employee?
NL: In a pharma environment, self-management matters; we need independent and reliable people to do their share of the work responsibly and without a manager's constant vigilance. I read a book called "No Rules, Rules", about the Netflix culture, and the message is that if we treat people as adult professionals, they will respond as such. Companies must empower their employees to self-manage and be responsible for their work. Pharma has been paternalistic with managers following up on employees, but in the future, in a digital and virtual world, this will change to personal interaction every two or three months. The future employee must also be tech-savvy and understand the different working tools. Those are the two maybe most important factors; however, we must also see the big picture in Mexico. Pharma has very specialized departments with specialized sub-departments within companies, and everybody is in their lane. In the complex Mexican health system, we must avoid getting stuck in one lane and with one stakeholder. We must see the full picture, the whole journey -diagnosis to access- to move forward. We have to cover more areas as we are only eleven people working in Ultragenyx Mexico. Most of our people have experience as reps; they are very skilled in interacting with physicians but not necessarily in administrative and financial areas or with other stakeholders. The learning process demands that they have to work on the whole journey, and it has been quite a learning curve. I don't believe in overspecializing. There is, of course, well-defined compliance, and this is key. I want my people to work towards having the patient always at the centre. That is their collective job, even if it includes working with stakeholders. We are a hands-on operation, and we all work towards a single objective: the patient, not revenues or finances; they follow from the patients getting what they need.
EF: Has rare disease incidence increased in Mexico over the last two years?
NL: That is a hard question to answer as in Mexico, we have very little data; Brazil, for example, has a lot more data on epidemiology and rare diseases. But now, more rare disease players are coming to Mexico, bringing additional therapies for new diseases. Pharma companies are putting effort into creating diagnostic programs, disease awareness, and identifying more patients. The virtuous cycle will build a new ecosystem; more people will be diagnosed and be part of the different data basis. There is a good initiative, originally owned by the Consejo de Salubridad General, but has since migrated to the Ministry of Health (Secretaria De Salud) and has now been put on hold. It's a census to create a platform for healthcare providers, government institutions, and associations to register and describe the number of patients who have a Rare Disease, for the collective knowledge to be contained in a specific database. I hope the initiative is retaken and launched shortly. It will give us clear data on what is happening in Mexico with rare diseases.
EF: There have been many changes and developments in rare diseases; what specific achievement would you like to celebrate this year?
NL: A breakthrough this year would be to get our first rare disease treatment delivered for purchase in the Mexican public sector. Last year the Consejo de Salubridad approved the treatment, and this year actual patients can receive our therapy -a big milestone. Mexico is probably the country in Latam where it is hardest and takes the longest to gain product access, so it is very nice to finally offer patients this benefit. We have interesting therapies in our pipeline, some complex, and we are looking to the future. In AMIIF, we have seen the landscape and the regulatory framework is not yet in place. We are prepared and waiting for a government initiative to help set the ground rules for regulatory evaluation of these therapies for market access. When gene therapy comes to the Mexican market, we want a regulatory path for approval, so we are working on the building blocks for the new generational therapies. The first gene therapy has been approved in the US, and we need to be ready when they come to Mexico.