Read the Conversation
EF: 2020, the year of diagnostics; 2021 the year of vaccines. What do you think 2022 will be the year in Brazil?
EP: It will be a year of growth in Brazil. Impressively, our people adapted quickly to the vaccination problems. Almost 100% of the population in Brazil got fully vaccinated to push the economy of the country forward. With a strong belief in our country, we remain positive that Brazil will be growing in terms of the economy and the healthcare system.
EF: What do you think have been the lessons learned from managing through the pandemic, in terms of operations, leadership, and business?
EP: One lesson learned is that we don’t need to physically be in the office to manage a business. Before, the pandemic home office was a sore topic, but there has been great proof that it is possible for people to work well together from home. Ultragenyx performed very well with employees doing home office. These times have taught us how to better manage a work-life balance and improve on time management skills. One good example is that no one from my team became burnt out as they had the opportunity to dedicate more time to their personal lives.
EF: How did your role change compared to how it was pre-pandemic and during the pandemic?
EP: In terms of work, it has increased. However, the pandemic has presented the opportunity to learn more. To have to learn how to compress week-long international meetings into a two-hour Zoom meeting has been challenging but it has created the opportunity to get more meetings done. More meetings mean more work and I am pursuing to find a balance between working from home, in the office, and personal life.
EF: What is the footprint of Ultragenyx in Brazil?
EP: In terms of rare diseases, our first step was to hire the best people who are highly experienced with rare diseases and are patient-oriented. The second step was to figure out how to make medications affordable for patients as quickly as possible. We adopted a hybrid model here in Brazil. We filed our medication under a distributor first while I was working on setting up my facilities here. In the second year, we got two medications approved, got the green light to start pricing, and then arranged for reimbursements through CONITEC. Ultragenyx got two approved medications included in the reimbursements listing within two years, which was a record time in comparison to other companies working with rare diseases here in Brazil.
Usually, it is very difficult to get full reimbursements for rare disease medication in Brazil, but we had the right people in place and great collaborations, especially between market access and our medical team. We realized we needed to be progressive and accomplish certain goals like generating local evidence, having feasibly priced medication, and more in order to receive full reimbursements. Our idea was to provide donations to patients who were critically ill, provide early treatment and generate experience for our treating physicians. The difference between Ultragenyx and other companies is that we followed the process as the others did, but also complete the necessary steps in the middle.
EF: How do you attract resources to Brazil from your headquarters?
EP: Since the beginning, Emil Kakkis, our CEO, strongly believes in Brazil and has good connections with treating physicians in Brazil and has been here taking part in the congress, working with some leaders and scientific publications here. His high interest, participation, and drive for the company’s triumph have greatly impacted the group’s success rate in Brazil. Ultragenyx Brazil was the second operation after the US. Two to three months after the company gets the medication approved in the US, it is approved in Brazil which is unusual within the pharma industry for Latin America, but Ultragenyx made the right steps.
EF: What do you look for in other establishments when thinking about collaborations and partnerships?
EP: Externally, we have a lot of collaborations and global partners. We remain close with and support the patient organizations, centres, groups, foundations, and treating physicians across Latin America, especially in Brazil, who provide support for patients with rare and ultra-rare conditions. We are working to open more doors to the broader public in order to increase inclusion and diversity amongst partners for new recruits in the future who prove to be highly competent in the relative fields.
Internally, we work very well cross-functionally due to the competence of our employees and the same level of importance applies to partnerships, especially when working towards successful strategies and ideas regarding treatments for rare diseases. The need for highly competent teams is extremely important.
EF: How did you attract the best and the brightest to work with Ultragenyx?
EP: One thing that attracted top talents to Ultragenyx in the beginning, was me, as I am a highly tenured and experienced person in the field of rare diseases and people saw that I was working with and promoting the company. There was a lot of belief placed in me, my work, and my marketed values in Ultragenyx. This attracted talents from other medical companies that work with rare diseases in Brazil. Some of these experienced people had previously worked with me in other companies geared towards the same purpose. These experienced people, after working with Ultragenyx, also began to attract more talent. As the company grew exponentially and the success of Ultragenyx’s work became more noticeable, more people wanted to know what we are doing and applications to work with us are now coming in rapidly.
EF: How would you rate the level of access to innovation in rare disease therapies in Brazil?
EP: Very low, unfortunately. I would love to see an increase. Now we have things that have changed. We have a new resolution in Brazil that has sped up the market to physicians for medicines that are not available in Brazil- especially for rare diseases- so we can get or match physicians within six months. I’ve had this experience with 3 medicines, and they worked perfectly.
From the price perspective, we are in great shape as well, the price commissioner in Brazil worked diligently to approve prices in six months. It has proven difficult to convince ConeTec to incorporate medicines given the low level of scientific evidence with any medicine for rare diseases, but they have opened the doors to this now. While the possibility to have a medicine incorporated and approved for rare diseases is low today, it was far lower 5 years ago. Today we see a light at the end of the tunnel, thanks to a new team within ConeTec that has opened their eyes to the importance of such aspects to have new medicines approved. However, not every medicine is approved. It depends on the disease if the company brings clinical trials to Brazil or not, and if they are working on generating local evidence- we award the evidence and the data. These things are very relevant to getting access through the public system which, in Brazil, is the only way to gain access to medicine for rare diseases unlike other countries like the US and Columbia which have private insurance.
I think the levels of access will continue to increase in the future.
EF: How does education impact access?
EP: It’s very important. For example, ConiTec was not interested in any kind of meetings with the pharma industries, but nowadays they are open to these kinds of discussions and we now have the opportunity to educate them about the diseases and the therapies that we are bringing to the country. With the opportunity to educate them about the diseases, we have been able to move forward as things have become better. There has also been an opportunity to educate the Ministry of Health here.
It can still be better here in Brazil like it is in other countries like the UK, Canada and Australia, but the opportunities to educate the Ministry of Health and the medical society here have become more accessible.
With these opportunities to educate people on rare diseases, there has been progressively increased support in the field.
EF: The education of physicians and their knowledge of how to diagnose was the number one challenge with regard to rare diseases. How do physicians diagnose?
EP: This is definitely the number challenge. Due to the continental dimension of Brazil, it is almost impossible to reach all the centres to educate the physicians on using the relative tools. The pandemic brought different ways to communicate like Zoom meetings, but not all physicians have enough time to join in the lectures we promote virtually. Some of them are very interested in learning more and do take part in virtual lectures, but it remains a great challenge.
Now, however, we are reaching regions in Brazil we have never reached before like the Amazon Jungle. We had two patients diagnosed there by a treating physician who learned from the virtual lectures we have promoted.
EF: You are going to be celebrating 5 years of Ultragenyx Brazil this year. What would you like to highlight and celebrate in your celebratory speech to your team?
EP: Being able to now tend to a mom with a child who has a rare disease and provide access to medication to help is very gratifying. With our hard work as a company and well-developed team here at Ultragenyx Brazil over the past 5 years, our patients now have the right to have far less complicated access to medicine for their rare disease(s) which they did not have before. This will be our legacy.
