Read the Conversation
EF: What are your priorities for 2024, and how are you advancing rare disease treatment in Brazil?
PP: Recordati already has eight rare disease therapies approved by ANVISA for commercialization in Brazil. Our main focus remains on rare disease patients, although we have expanded into rare oncological conditions. This is a result of the acquisition of Eusa Pharma in 2022.
After the acquisition, we could offer two new therapies, one for idiopathic multicentric Castleman disease and the other for high-risk neuroblastoma, offering treatments for highly unmet medical needs in the country. Trying to lead these two therapeutic areas is currently at the top of our agenda.
We also filed for the launch of Isturisa (osilodrostat) to treat Cushing disease and syndrome. This is a breakthrough therapy that can change the course of this severe disease and unlock the full potential of life of hundreds of Brazilian patients.
And we are continuously focused and striving towards improving access, our key strategic imperative for the years to come. Currently, there are a limited number of therapies covered by SUS. Therefore, we are working daily on ways to provide broader access to all Brazilian citizens suffering from rare diseases.
EF: How is Recordati providing treatment access and building the necessary partnerships to achieve this?
PP: We are working alongside important health secretaries and public policy decision-makers to enhance awareness of the diseases we have therapies for. We develop projects tailored to regions, states, and conditions. Our focus is to allow patients to receive proper treatment with a positive and presumable impact on healthcare expenditures. Our efforts include providing diagnostics and medical education, discussing treatment guidelines, and specific reimbursement and commercial conditions.
We are also drawing attention to the complexities of rare diseases and their profound impact on individuals' lives. Emphasizing the economic benefits, we highlight that utilizing appropriate treatments for certain conditions can result in substantial cost savings compared to palliative or placebo options. Unfortunately, some people use lawsuits to challenge this, which we strongly disagree with and actively fight against. We believe in finding solutions through cooperation and positive discussions to improve healthcare access and treatment choices
We have engaged with the MoH and state authorities to discuss Therapeutic Positioning Instructions (TPIs), aiming for mutually beneficial terms to provide specialized therapies, particularly and more recently for neuroblastoma, one of the most prevalent pediatric cancers.
Similarly, in acromegaly treatment, our approved therapy has been available for at least four years. Currently, public patients can access it only through legal injunctions or occasionally through private healthcare reimbursement. Our mission is to ensure that no patient is left behind. In the realm of porphyria, we aim to comprehensively address every case within the country. We are actively identifying patients, offering free diagnostic services to interested physicians, and collaborating with all interested partners to enhance diagnostic support, which is lacking, especially in public hospitals. We are committed to bridging that gap by providing free diagnosis. While therapy availability remains challenging, we acknowledge this discrepancy and are dedicated to finding solutions. Our focus is empowering patients with both diagnosis and treatment, steering them away from any obstacles in their path to recovery.
EF: What distinguishes Brazil's operation from other subsidiaries in the world regarding specialized knowledge promotion in the field of rare diseases?
PP: Latin America faces unique challenges, presenting uncertainties that set it apart from other regions. One notable hindrance is the limited availability of patient transportation to public reference centers. In Brazil, for instance, patients may reside more than 500 kilometers away from healthcare facilities. Compounding the issue, those without access to cell phones may experience prolonged periods without contact with physicians. The lack of basic infrastructure for diagnostic support adds another layer of complexity. Despite these hurdles, Brazilian physicians demonstrate incredible resilience and determination, fighting battles without all the necessary tools and prevailing in the face of adversity.
Seeing a dialogue begin on building a strong healthcare system and driving scientific innovation worldwide is inspiring. Healthcare professionals in Latin America are a testament to the potential for positive change, even in difficult times.
Physicians are taking charge of innovation, leading discussions, and serving as top prescribers for various therapies. Their initiative and interest drive this conversation as they seek to take the lead in addressing existing problems. Our readiness to tackle uncertainties and adapt is crucial. Sometimes we are better, sometimes behind, but we always strive, fight, and never give up.
EF: If you had to build a sustainable healthcare ecosystem in Brazil, what pillars would you base it on?
PP: My first pillar would revolve around access. We do not have a clear, well-stated public access priority related to public patients. The ministry, together with pharma companies, needs to drive an agenda that goes beyond the four-year mandate. We need valid discussions and a timeframe for companies to understand where therapies will be paid.
The second pillar is infrastructure. Besides the need to bolster the establishment of vaccination centers, we have observed a lack of decentralization. Certain regions in Brazil have exhibited exponential growth in population, surpassing other parts of the country, but are still being neglected. Addressing this issue must transcend the typical four-year leadership tenure. By doing so, we can significantly reduce patient travel time and expenses while speeding up access to medications, particularly those administered via infusion.
Prioritizing patient needs is crucial. Within the oncology system, facilities are well-equipped to receive and treat patients. However, for rare diseases, the deficiency of infusion centers is evident across certain regions, and diagnostic resources are often unavailable. It is not unknown that most public centers lack essential tools for diagnosing porphyria, for example. Consequently, we have taken the initiative to supply these tools ourselves in several cases.
We need to build a positive agenda within the pharma industry. This is a broad pillar and is possibly a priority for everybody. We have been trying to establish partnerships beyond treatment and the specific needs for a broader perspective on supporting diagnostics and providing therapies to the public system. Although Recordati is a small company, we are leaders in the treatment we offer. We can discuss with the public sector, how to reduce the budget burden while supporting the better treatment of Brazilians suffering from rare diseases.
EF: How is Recordati leveraging digitalization to improve awareness, diagnostics, and overall operation efficiency?
PP: Digitalization in the form of AI and advanced technology is an area in which we are investing this year. During the pandemic, we had the opportunity to extensively drive disease awareness virtually for all the therapies we work with. Today, we handle most of our meetings with physicians worldwide online. We are at the forefront of global discussions and advancements in porphyria treatments, spearheaded by Brazilian physicians. In the neuroblastoma sector, Europe boasts numerous advanced centers. Facilitating collaboration, we connect German physicians with Brazilian centers to offer guidance and expertise
Recently, we launched a diagnostic support program benefiting physicians in remote areas by offering logistical and virtual diagnostic exam assistance. Expanding virtual diagnosis to cover more rare diseases is our future aim.
Diagnoses for Castleman disease are particularly more difficult since it is mainly detected by ruling out other conditions as patients present multiple unspecific symptoms. To aid in this assessment, we are developing an algorithm using artificial intelligence and machine learning. This algorithm correlates symptoms to simplify disease identification for physicians. Currently, we are collaborating with a Polish company, Saventic, to pilot this diagnostic tool.
EF: What type of talent does Recordarti look for when building up a company, and what skills must these people have?
PP: Recordati is looking for skilled and creative people willing to work towards a purpose. We are building an affiliate from zero and serving patients without hope until our therapies become available nationwide.
More tangibly, we are looking for people with expertise in specialty care and/or rare diseases within the pharma industry. Our talent plays a crucial role in building a company that focuses on rare diseases and aims to be a leader in this area.
Initially, we established a leadership team spanning various areas: sales, medical, regulatory affairs, back office, pharmacovigilance, quality control, insurance, and supply chain. These six areas remain pivotal for our operations. As we matured, we created space for training new employees in the Recordati approach. Our focus has shifted to recruiting individuals capable of propelling growth and advocating for new therapies among physicians. Consistency in seeking an entrepreneurial mindset persists, with an emphasis on individuals adept at problem-solving and dedicated to patient-centric approaches, navigating the challenges unique to Brazil.
Our growth has been exponential, soaring a remarkable 10-fold since our commercial start-up in 2020. From a mere two-person team without an office, we now boast 25 employees and are growing. Progressing toward significant sales milestones, we have expanded our patient base to over 600 individuals, with continued acceleration due to intensified diagnostic efforts and aiding physicians in identifying underdiagnosed patients
EF: What would you like to celebrate as achieved in five years?
PP: The fight in the rare diseases sector never ends. Our local motto is "No patient left behind.'
All patients must be diagnosed, and all patients must receive treatment. We are working towards promoting broader access to the patients we know are diagnosed and are striving to get treatment. It is frustrating to see rare disease patients dying due to lack of access. We are driving medical education and diagnostics and leading the way wherever there is a space for us to show.
In the areas where we cannot lead, for example, the signing of reimbursements for rare disease therapies, we are trying to influence, discuss, and make an agenda favorable to all the stakeholders.
Recordati recently launched a new global purpose, "unlocking the full potential of life," this is our goal. We can never rest until we are able to provide a better life for rare disease patients.
I envision celebrating universal therapy access for all rare disease patients within SUS.
