RARE - Juan Vila Alvarez

​​​​Welcome to RARE a blog dedicated to the rare disease industry by Laura Hulley, a Senior Recruitment Consultant at SciPro​.

RARE - Juan Vila Alvarez

In this episode of RARE, I welcomed Juan Vila Alvarez from Recordati Rare Diseases to take us through his experience so far in the pharmaceutical industry and his thoughts on the challenges faced by pharma companies bringing rare disease therapies to market and the importance of Patient Advocacy Groups. Recordati Rare Diseases is a pharmaceutical company that was created in 1990 and provides treatment for patients with rare diseases. Juan is the General Manager for Spain and Portugal.

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Key takeaways:

• Juan is a Physician by training and after some experience working in primary care and the emergency room within the hospital, he decided to make the move into the pharma industry. Juan worked in several companies in different roles, in the likes of Merck Serono, SOBI and Takeda before his current role at Recordati Rare Diseases. His first experience with a rare disease product was at Merck Serono where he realised just how important and how challenging it is just to drive the solutions for rare medical conditions. Juan joined Recordati Rare Diseases two years ago, a company with a real focus on providing treatments for conditions with very few patients. He manages both Spain and Portugal as General Manager, and they provide solutions for 13 rare conditions across oncology, endocrinology, and metabolic diseases.

• There are three main challenges we face when we are talking about rare diseases. Firstly, there are approximately 7000-8000 different diseases, so many conditions that are affecting a very limited number of patients. Due to the limited number of patients, it is not easy to find a diagnosis for a rare condition as it is not easy for a physician to diagnose a condition where they may see the signs and symptoms maybe once every ten years. Another challenge is creating a robust plan for research and development. In most clinical plans, 1000s of patients are managed, but with a rare disease, there are not as many patients that can be enrolled in the clinical research.

• There are two main ways that Patient Advocacy Groups work well including the creation of patient associations. This helps the patients to find a group of individuals who may share the same symptoms, but they also provide several resources for the patients affected by the rare conditions. This helps to find additional clinical support or other therapies and to help them not feel alone in their suffering. They may also find other patients suffering from the same condition as them and be able to share solutions that they have been able to identify.

• The second is to align and to communicate with Healthcare Professionals (HCPs) when they are performing research. When clinical trials are put in place, HCPs or the investigators will propose the patients to enrol and so Patient Advocacy Groups and Patient Associations help to understand from a patient perspective, for present and future patients which research to be involved in that will provide them with solutions in the short and medium-term.

• The challenges faced by pharma companies are similar and related to the limited number of patients within a population suffering from a rare disease. Juan recalls working on a particular drug for a condition that had a total of 70 patients identified across Europe and so just 3-4 patients in Spain, so there is a real limit on the data created to demonstrate the ability to transform a molecule into a future therapy. Juan explains the importance of pharma companies to not only provide the solutions for the conditions but also to raise disease awareness and knowledge. This will not only help raise awareness of the existence of these diseases across the wider community but also will help patients to pursue a correct diagnosis and with that, the right treatment.

• Juan is extremely positive about the future of rare and orphan therapies. He expresses that rare diseases are now known amongst the whole community whether it be Health Authorities, patients or HCPs and they are aware of the challenges they bring and so the limited number of treatments currently available. Therefore, it is important to work together to put in place strong and robust R&D plans that can transform more promising molecules into future drugs for these patients.

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I would like to take the opportunity to thank Juan once again for featuring on RARE.

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For more information about rare disease, check out some of the websites listed below:

• https://www.rarediseaseday.org/

• https://www.eurordis.org/

• https://rarediseases.org/

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Author Details

Laura Hulley - l.hulley@sciproglobal.com