Luis Calderón
Alexion Pharmaceuticals and President of the Mexican Association Pharmaceutical Research Industries (AMIIF)
View from the Top

Orphan Drugs- the Right Access Path

Sat, 09/05/2015 - 12:37

Q: How many obstacles remain to the development of a thriving biopharmaceutical sector in Mexico?

A: The challenge that Mexico is facing today is the lack of access to innovative products. COFEPRIS has certainly made a great effort to improve approval times. However, public institutions such as IMSS, ISSSTE, and Seguro Popular still have certain things to do in order to improve their processes and increase access within the public market to medicines. Processes should speed up in order to bring drugs to the population that needs them. Additionally, the number of physicians who specialize in therapeutic areas where biologics are prescribed is concerning; for instance, there are around 600 hematologists in Mexico, which is a very small number when you consider that we have a total population of 120 million. The same happens in other specialties such as endocrinology, where there are only 180 physicians. Endocrinologists should treat patients with diabetes, so in order to take advantage of biologics there is a huge path to follow to increase the number of trained physicians from different hospitals.

Q: Why is specialization such a challenge and what should be done to address the issue?

A: Specialization is necessary to address the modern challenges in the healthcare sector. In the 1980s, the Mexican population increased dramatically and, in order to meet the growing demand for healthcare services for children, the government established the right incentives to create more pediatricians. I do not see the same intensity in the government’s agenda to address the need of specialists in therapeutic areas for chronic and degenerative diseases. In addition to this, the general knowledge that physicians are obtaining in school with regards to chronic and degenerative conditions is still lagging. If they do not enter a high-specialty and get clinical training in specific areas such as diabetes and obesity, their knowledge is very limited. Thus, better education and training programs for physicians are necessary in Mexico. In fact, this reduces the number of biopharmaceuticals that are available in the marketplace.

Q: The government has recently developed a regulatory framework for biopharmaceuticals and biosimilars. Is this any use without conscious corresponding human capital development?

A: I think this is a multi-faceted problem. Healthcare expenditure in Mexico is only 6.2% of GDP, which is very low compared to the average of 9% within OECD countries. Furthermore, considering that 50% of that expenditure comes from out-of-pocket expenses, the government invests only 3.1% in health expenditure. A large part of this budget goes to administrative costs and other processes, rather than being invested in medical training and medicines. Therefore, resources should be better allocated to improve the healthcare system. It is not that the government is not playing its role but it is taking a long time to effect the transformation of the whole sector. On top of that, the national healthcare sector is complicated due to health disparities in the Mexican population. While chronic conditions including diabetes, obesity, and cancer represent a crisis for the sector in terms of mortality rates, there are still 30 million people below the poverty line who require basic primary care services and medicines, such as antibiotics. It is therefore a big challenge for the government to create effective strategies to address such disparities, as well as to define the top-priorities and develop the necessary infrastructure for the future.

Q: Notwithstanding these challenges, have there been encouraging developments?

A: Many new biotech-driven companies are entering the market, and COFEPRIS has successfully shortened the approval times for new molecules. Mexico was the first country to grant market authorization for two biopharmaceutical products, which was the result of several moves such as improving the clinical research pathway in the country, as well as recognizing other high- quality regulatory agencies such as the FDA and EMA. COFEPRIS has significantly improved its review process, which certainly represents a benefit for the industry. Companies are now authorized to start developing but not commercializing biosimilars eight years before patent expirations.

Q: What role is AMIIF hoping to play in developing the market?

A: We are working on a joint effort with COFEPRIS to develop the right norms for biopharmaceuticals, biologics, and biosimilars. We have learned a lot from international experience on biologics and it is a dynamic field that has dramatically changed in the last ten years. The clinical studies that are required for product approvals are completely different from those required for generics. Therefore, this is a matter of continuous learning in order to keep the pace with harmonized regulations and new developments. The same thing will apply for nanotechnology and stem cells therapy. Regulation is always lagging behind innovation because it is impossible to regulate what does not exist yet. The challenge for COFEPRIS is to develop transparent processes for the approval of biosimilars, and we are collaborating with them in this regard.

Q: How is this progress impacting the orphan drugs segment on which Alexion focuses on?

A: Orphan drugs have a specific approval pathway for market authorization to obtain orphan drug designation, which is very different from the one that traditional products follow to get sanitary approval. Also, the approval process for orphan drugs is not as lengthy as for traditional products; it is a faster and less complex process that takes 40-60 days. Last year, COFEPRIS developed a draft norm for orphan drugs along with AMIIF, which will be approved in the upcoming weeks or months. Hopefully, this will bring certainty to the regulatory status of orphan drugs.

Q: How challenging is it to obtain approval for drugs that are targeting such a small handful of patients?

A: Firstly, our products have to comply with regulatory requirements. Once we get approval, we submit a proposal to the General Health Council, and then we request access to one of the different Institutions. In the past, it was very difficult to get approval from the General Health Council, as it was mandatory to present a cost-effect analysis for orphan diseases, which is practically impossible because there is no data to build a consistent model for an accurate assessment. Fortunately, the cost-effect analysis was recently replaced with a budget impact model by decision of the General Health Council, which accelerated the approval process for orphan drugs.

Q: Do you envisage Alexion and other major pharmaceutical companies bringing R&D projects to Mexico?

A: There have been several changes in the Mexican regulatory environment and market to support this. We have a better and quicker regulation for clinical studies in which third-party organizations such as the national institutes of health are now allowed to participate in the approval process for study protocols. AMIIF and COFEPRIS recently signed a collaboration contract with IMSS for the latter to also participate in such processes, and furthermore, IMSS is now authorized to conduct more clinical studies than before. Therefore, more innovation is coming to Mexico not only for Alexion but in general terms. It is expected that many companies expand their research activities in the country, which I am sure will benefit the whole Mexican industry.

Q: Do you believe Mexico should pick an area to focus on for economic development based on investment returns?

A: This is a matter of the government deciding where it wants to win and how. A couple of years ago, the South Korean government set the bold goal of becoming a leader in the innovative pharmaceutical market. They signed agreement contracts with the US, redefined their intellectual property model, and heavily invested in the pharmaceutical industry. Similarly, the Mexican government has to decide if it really wants to win in the biotech and healthcare sector, and if it is willing to invest more in this field. Progress will not only happen due to entrepreneurship but it requires a huge amount of money and resources. This sector is very risky and high a return of investment is needed to sustain a biotech business. The government needs to realize that a risky sector has higher failure rates, and in addition high interest rates make this sector even a more difficult one for business development. Therefore, a clear play-to-win strategy is needed in Mexico to invest the right resources in the right sector, whether it is automotive, energy, or biotech.

Q: Should Mexico be looking to other countries, such as South Korea and Brazil, as role models?

A: In terms of innovation, Mexico could replicate what South Korea has done regarding intellectual property law and creating the right environment for innovation. South Korea is a unique example of how products have been developed and how they have emerged from the pipeline. Brazil has a complete different approach due to the fact that it decided to take the biosimilars path rather than the innovation one. In order to bring biopharmaceuticals to the country, it has to be defined that of would happen through the innovation path or biosimilars one. Being an emerging market, we need to take a look at which path is more successful than the other. The government is realizing that the opportunity to go abroad is important and that will be transforming the industry. I do not know whether the focus will be on innovation or on generics and biosimilars, as this is not yet clearly defined.