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Rare diseases and orphan drugs: starting point on the MUG pharma-biotech lecture series

The orphan drug segment is at an exciting and dynamic moment, with scientific advances that will allow us to treat rare diseases for which no treatment is currently available.

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24/11/20 13:21 Miguel Angel Echavarri

Álvaro Herreros, Head of Global Rare Hematology & Neuroscience Franchises at Takeda, has been in charge of opening the lecture series organized within the Master's Degree in management of Pharma-Biotech Companies. This is a series of sessions that complement the academic program and are given by industry professionals from different business areas. The goal is to bring students closer to the reality of the pharmaceutical and biotechnology sector from a practical and professional point of view.

En esta primera conferencia se abordó el tema de las enfermedades raras y los medicamentos huérfanos. Se trata de enfermedades que afectan a un número limitado de personas (<5/10.000 habitantes). Existen actualmente unas 7.000 enfermedades raras reconocidas, de las que sólo el 5% tienen un tratamiento disponible.

The speaker highlighted that one of the main problems faced by these patients is the diagnosis. Given the lack of knowledge, it is common for them to rotate between different specialists until they find an adequate diagnosis. He also emphasized that "these diseases have a very special ecosystem, where the relationship with the patient becomes very intimate. The partnership with patient associations is also fundamental, and the patientadvocacy Departments is especially important in companies like ours".

"Although I knew about orphan drugs, I was unaware of their impact on society and the repercussions and progress currently being made in this field," says Juan María Carralero, student of Master's Degree. He also values this complementary training : "Because of the subject of degrees we have, we are more used to seeing everything from a more theoretical point of view. These conferences help us to fill in our vision with real projects, people and companies, which gives us a much more real perspective and which financial aid complement our programs of study".

To young professionals
  • Firstly, and related to the topic of the lecture, explore the area of rare diseases and orphan drugs because it is a area that, in addition to being very interesting and of great satisfaction at the staff level, is a segment of the biopharmaceutical market in clear growth and of great dynamism. Moreover, scientific and technological advances are beginning to enable us to treat (and even cure) for the first time diseases that were previously untreatable, for which patients have a great need.

  • Secondly, and from my own experience staff , I think it is beneficial in the early stages of the degree program professional to expose yourself to different roles and functions in order to develop a better overview of the business, and how the different Departments and parts of the organization relate and integrate with each other. Therefore, I think it is not good to become obsessed with a particular position or department , but to be open to different options that fit with your individual profile and allow you a broad and diverse learning experience with the possibility of evolving into other related positions and functions. Therefore, it is also important to select the company, team and manager of your first work positions, rather than the position itself. This will determine in a very important way your professional growth and degree program .

  • Finally, be curious and open to exploring an international degree program . The biopharmaceutical industry is global in nature and scale. In my experience, the international degree program is very enriching both professionally and professionally staff. There are jobs and roles in the sector that can only be developed in international positions. A good approach is to gain experience at the country level first, to develop a solid experience close to the needs of the 'doctors and patients receiving our drugs' and then evolve to international positions where you can continue to grow, but always keeping in mind the country perspective and the day-to-day needs of the business.

Álvaro Herreros

Alvaro HerrerosÁlvaro is a former student from the University of Navarra with a double degree: graduate in Economics and an International MBA from IESE. He has developed extensive experience in global strategy and commercial positions leading cross-functional teams of products and therapeutic areas, as well as leading teams and commercial operations at country and regional level in the launch of new products.

He is currently Senior Vice President and Global Head of Neuroscience and Hematology Franchises at Takeda Pharmaceuticals based in Boston. He joined Takeda in early 2019 following the integration/acquisition of Shire Pharmaceuticals, a pioneering biopharmaceutical company specializing in rare diseases, where he had spent 10 years in positions of varying responsibility at country, regional and global levels.

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