Orphan drugs and rare diseases

Jose-R-Trigueros-Picture

According to Jose R. Trigueros at Leyva, Montenegro, Trigueros Abogados S.C, better information and incentives are needed to encourage research and development in Mexico.

Mexico is a country with a population of over 112 million, according to the 2010 population census, making it the 11th most populous country in the world and second in Latin America. It is estimated from information issued by the Security and Social Services for Federal Workers Institute (ISSSTE) that nearly 6 million people suffer from conditions or diseases classified as rare or orphan, which as defined in the General Health Law, are classified as such by prevalence, namely, any disease which affects no more than five per 10,000 individuals.

ISSSTE, an Institute which offers health care services for federal workers, has developed a list which indicates that 80% of rare disease patients suffer from genetically derived diseases, while the remaining 20% is affected by immune deficiencies or emerging diseases (defined as endemic or infectious diseases that have been eradicated from developed countries). The prevailing orphan diseases treated at ISSSTE are the Fabry syndrome, Gaucher’s disease, Paroxysmal nocturnal hemoglobinuria, also known as Marchiafava-Micheli syndrome, Mucopolysaccharidose disorders and the Niemann-Pick disease.

However, most of the population rely on the Mexican Institute of Social Security (IMSS) for health care services. In fact, IMSS provides health care services for more than 70 million Mexicans (more than 60% of the country’s population), as recently stated by its General Director. Unfortunately, the Institute does not issue a list of the prevalence of rare diseases among its users, but maintains the Specialized Centre for the Treatment of Lysosomal Diseases. The remaining population receives health care services either from the Government-funded Popular Insurance program or in the private sector, both of which do not keep lists of patients afflicted by rare diseases.

Information regarding rare diseases and their prevalence in Mexico is extremely scarce. Federal government institutes such as the National Center for Prevention Programs and Disease Control do not have a list of rare diseases in Mexico and other related Government institutions do not take them into consideration due to the low percentage of affected population. This results in a very complicated landscape for the provision of information and effective treatment to prospective patients, as well as for analysis of potential new treatments and/or therapeutic options.

Modifications to the law
Nonetheless, Mexico is the 14th largest economy in the world and a very attractive market for pharmaceutical companies. Likewise, the Government’s awareness regarding rare diseases and the urgent need of patients to access treatments and information that is potentially lifesaving has increased. In fact, in 2012 the General Health Law was modified, with two new articles (224 Bis and 224 Bis 1). These articles developed the legal concept of orphan drugs and rare diseases, and directed the Ministry of Health to implement measures to enhance accessibility of orphan drugs and the research and development for new drugs and therapies.

Unfortunately, without budget and/or a step-by-step strategy to effectively boost research and development, or at least a way to provide accessible and affordable drugs and treatments against rare diseases, the new additions to the General Health Law appear mostly cosmetic.

The need for incentives
As a result, both patients and pharmaceutical companies face uncertainty and great disadvantages for the access and introduction of new orphan drugs in the Mexican market. The biggest obstacle is still the absence of incentives, such as market exclusivity periods or tax credits/exemptions for new orphan drugs. Such incentives are currently offered in other jurisdictions and bolster the development of new medicines and treatments against rare diseases. Offering these sort of incentives would help the country become an attractive opportunity for the research and development of emerging diseases which affect the Mexican population and which are not being investigated any further, given that they do not exist in developed countries anymore.

Additionally, the country would also have the opportunity to become a pharmaceutical hub for the research and development of new drugs and treatments against rare diseases in Central and South America, bringing jobs and development opportunities that the country sorely needs.

Incentives may become vital for certain developments and inventions, since more often than not, pharmaceutical companies develop orphan drugs which may not be suitable candidates for patent protection or, if the invention derives from the biotechnological field, patent protection may not be sufficient to guarantee their investment. Therefore, market exclusivity may become the best incentive for attracting capital for R&D in Mexico.

The current Mexican landscape unfortunately does not allow for market exclusivity. The Health Supplies Bylaws only provide a limited form of data exclusivity, which could be available for certain compounds that meet the criteria (new chemical entities and/or innovator biotechnological drugs), and could provide an option for obtaining de facto exclusivity. However, lengthy, costly and highly specialized litigation would be required, and while our firm has successfully handled such cases, we recognize that it diminishes the attractiveness of the Mexican market.

Tax credits or exemptions, along with efficient legislation favouring the development of new drugs, would also help Mexico become a magnet for research and development. Furthermore, the legal and public perception implications of offering credits or exemptions would not be viewed as negatively as other measures, providing a quick and simple option to attract and generate new developments against rare diseases.

Balancing needs
To appease – at least partially – such inefficiencies, a couple of initiatives are being prepared and have received the support of NGO’s and patient associations in Mexico. However, balancing the interests of patients and pharmaceutical companies is always complicated, and one of the reasons why there may be conflicting views on the likelihood of the initiatives addressing the intrinsic complications of the Mexican health and legal systems.

Although both the pharmaceutical companies and patients would like to see more orphan drugs and better treatments for rare diseases in Mexico, patients may not be so amenable to market exclusivity periods and would be more inclined to a more diverse market with cheaper options. Of course, championing the patient’s needs and requirements could be a more attractive political option, however, it should be noted that the interests of both parties share a lot of common ground and minor fine tuning should achieve proper balance. Therefore, efforts should be made to provide the legislative body with information, data and clear examples of the hurdles of introducing new drugs and treatments against rare diseases in Mexico, given the lack of information among the Mexican health authorities. Likewise, building bridges between pharmaceutical companies and patient associations in order to foster a healthy exchange of information between both parties would greatly enhance the goal of curing the deficiencies of information, treatments and drug availability within the Mexican health sector.

Résumé

José R. Trigueros
José is a partner of Leyva, Montenegro, Trigueros, Abogados S.C. (LMT), leading the IP area of the Firm. He is experienced in copyrights, trademarks, trade secrets, industrial designs and patents, specifically pharmaceutical patent litigation, an area in which the Firm represents Pfizer, Inc. in Mexico. His litigation experience covers all IP areas at every stage of a trial, specifically at the Amparo stage. José worked as IP lawyer at Becerril, Coca & Becerril and later as IP Manager of the Mexican Stock Exchange. His experience in academia involves participations as adjunct professor at several Mexican universities and as professor of Economic Law and Commercial Contracts at the Instituto Tecnológico de Estudios Superiores Monterrey (ITESM) Santa Fe Campus.