Rare Disease Cases in Mexico Warrant Closer Attention

Q: What has been Ultragenyx’s role since the COVID-19 pandemic?

A: With or without the pandemic, Ultragenyx supports and treats patients. During the pandemic, however, we changed the way we interact with customers and engage with healthcare professionals and institutions to provide the best solutions for neglected or rare diseases. Overall, we try to put healthcare for chronic and rare diseases in the spotlight and keep in continuous communication with patients and physicians.

Q: What is the RARE Formula and how does it help patients with rare and ultra-rare genetic diseases?

A: The RARE formula was created by our CEO, Emil Kakkis, following many years of research into rare diseases. He observed that for new therapies to enter the market they had to be truly innovative and researchers needed to build good relationships with agents from the US Food and Drug Administration (FDA). Another part of the formula is to work with scientists and patient associations.

Many patient associations are interested in developing drugs for their particular disease. The RARE formula teaches them how to get funding and support R&D to bring a therapy from an idea to reality. 

Q: How does the company ensure that the products and services it offers deliver safe medicines to patients in different regions?

A: Safety is a major concern for our company. We follow a strong and rigorous pharmacovigilance process that we offer to different markets. We continuously interact with physicians and patients to get their insights on safety. All the treatments that we have commercialized have a safe profile. Pharmacovigilance is critical and side effects must be reported to regulatory agencies in Mexico. We also monitor this data internally to understand real-world evidence of our own treatments once they are in broad use.

Q: What are the main challenges Ultragenyx faces in the Mexican health sector?

A: Mexico has increased its efforts to treat patients with rare diseases during the past 10 years. IMSS, ISSSTE and INSABI now have programs to treat these patients. The regulatory pathway to get an orphan drug designation from COFEPRIS is different from a regular drug designation. We also keep in constant communication with the authorities so they understand that there are new alternatives that can change the course of a complex or fatal disease.

We are also addressing the lack of genetic testing within Mexico’s healthcare system. Ultragenyx provides free testing for certain genes and diseases. We also provide access to next generation sequencing, a genetic test that provides a very accurate diagnosis. We provide players in the private and public health system a clear view of the benefits of diagnosing and treating patients early.

The first patient treated with one of our therapies has completed a year of treatment and the changes are impressive. By disseminating these success stories among the players in the sector, the health system understands that they can provide a new opportunity for patients and their families.

Q: Regarding local regulations, what are the main differences between the US and the Mexican markets in terms of treatment of rare and ultra-rare genetic diseases?

A: The markets are highly different. The US market relies on the private sector for the provision of treatments for highly complex diseases. In Mexico, the private sector manages only 10 percent of these therapies, so we are really dependent on the public sector. The US operates similarly to a market of goods and services, while Mexico is focusing on socializing healthcare. It is key to understand the government's role in providing healthcare when introducing a new treatment.

Q: How are you bringing attention to the treatment of rare and ultra-rare genetic diseases?

A: Once a treatment gets approved in the US or in the EU, we strive to get it approved in Mexico. We are focusing on Mexico because of its large population and many untreated diseases. We need to approach patients quickly but Mexico’s regulatory pathway usually depends on an initial approval in a reference country.

We are also trying to work with regulatory agencies to bring clinical trials to Mexico, which are key to participating in R&D processes.  In the same way we are trying to become an ally in medical education and diagnostic platforms as critical elements for diagnosis

Medical education and diagnostic platforms are critical for a diagnosis. On average, a patient with a rare disease can go from eight to 10 years without a correct diagnosis, so it is key to put in place diagnostic programs that incorporate genetic testing.

Q: What future do you see for gene therapy in the overall industry and how do you expect this to permeate the Mexican market?

A: Our pipeline includes a couple of gene therapy programs. Gene therapy allows us to understand genetic diseases and match them with potentially curative treatments. It will be a turning point for biotech companies. We still have a great deal to learn about the science and to understand its long-term effects. The biotech and pharma sectors are highly excited about this area. The next generation is not thinking about treating chronic diseases; it is interested in correcting genetic defects. 

In Mexico, we are still working with regulatory agencies to get these therapies to the market. Furthermore, we are taking steps toward improving rare disease clinical development strategies under current regulatory statutes, focused on bring clinical trials to Mexico and ensure that patients receive safe and efficient treatments based on proper data.

Q: What are the company's expectations for the coming years?

A: We successfully brought our first therapy to Mexico to treat a rare disease called X-linked hypophosphatemia (XLH). In the short term, we plan to get our products for mucopolysaccharidosis type 7 (MPS7) and long-chain fatty acid oxidation disorders (LC FAOD) approved in Mexico. We expect to have them incorporated in the reimbursement list soon so they are available to all public institutions.

In the medium and long term, we are looking to grow the Ultragenyx pipeline. Exciting things are coming, including gene therapy programs. My goal is to continue bringing these treatments to patients in Mexico and developing strategies to find those patients and support them. Another priority is to continue bringing clinical trials to Mexico. A Mexican patient participated in a pivotal trial for the approval of the treatment for MPS7 and this is something that we need to replicate.

 Although there has been great progress in Mexico regarding rare diseases, there are still some topics that need to be addressed by the health community. We need to support this community by accelerating rare disease education and work with the government to have a clear view of these patients. This information can support the development of public policy regarding the rare disease community and budget allocation. We estimate that there are between 5-6 million people in Mexico with a rare disease. This is not a small community. All the different actors in the healthcare system need to prioritize these patients.

Ultragenyx is a biopharmaceutical company dedicated to the treatment of rare and ultra-rare genetic diseases.