Our Experience

It is estimated that neurological and psychiatric disorders and their sequelae affect as many as a billion people worldwide and impact thereof seems to spread transversally, affecting all age groups and all geographical regions – and even prevalence thereof appears to be growing. Many things can go wrong with the brain and the nervous system, and because of this, a vast number of pathologies have been described. Therefore, therapies to treat these disorders and help patients are also vast and expand from lifestyle changes, to physiotherapy, pain relief, and a broad range of medications. Most of these therapies are oriented to the alleviation of symptoms. Still, there is a clear need for novel therapies that can revert underlying neurological deficits or promote neural regeneration or provide the needed repair. Helping to unravel the causes behind these diseases and to bring new medicines to affected subjects is exciting and challenging but also, and above all, a priceless and rewarding challenge. Over the years, there has been much advance in understanding all these conditions, and in the generation of new treatments.

Advanced Therapy Medicinal Products (ATMPs) represent a fast-growing field of interest and constitute, themselves, a new category of medicines with a vast therapeutic potential for treating different types of diseases that include cancer, neurode­generative or cardiovascular diseases.

However, and despite their enormous therapeutic potential, the time frame between “design” and the conduction of the corresponding trials first – and MA later- is notoriously much longer than expected. And this has to do not only with scientific and technical challenges intrinsic to the nature of ATMPs but also with regulatory ones. Trials with ATMPs have challenging designs, and even the definition itself of a new product as ATMP – and hence the legal frame to stick at development- is a theme of debate, and different regions – US and EU- have adopted diverse legal frameworks that may be contributing to creating confusion among researchers which drive to, eventually, delays in the access to these new remedies.

Infectious diseases are caused by different pathogenic microorganisms, ranging from bacteria to viruses, protozoan’s, parasites or fungi – that can spread from one person to another, or even from one another species to human, causing the so-called zoonotic diseases. And it is assumed that infectious diseases are one of the major causes of death in the world. In fact, and according to the World Health Organization (WHO), globally, 16% of fatalities result from infectious diseases, making these diseases one of the most significant global challenges in medicine in our time. Infectious diseases are present transversally worldwide, and much emphasis needs to be placed in controlling and treating these diseases in areas of significant poverty, specifically.

New infectious agents appear, and known organisms develop increased virulence or resistance mechanisms to current treatments. The world was recently hit by an international health crisis of unprecedented dimensions and costs caused by the uncontrolled spread of the SARS-CoV-2 virus responsible for the COVID19 outbreak.

All elements of our body are in permanent contact and communication, gauging at every moment all functions. Hormones – the core of “endocrinology”- are the principal soluble mediator of this communication within the body. Hormones affect many physiological activities, including growth, metabolism, appetite, puberty and fertility, and any disbalance affecting the endocrine system may, hence, provoke the impairment of many different physiological processes. Diagnosis of endocrine disorders is not a straight-forward process, and signs and symptoms may span the entire clinical spectrum; neither it is the treatment. Treatments may include the use of medication, surgery or radiation-based approaches and are in general aimed to re-establish the normal hormone balance – e.g. administration of a synthetic hormone-. However, a change in one hormone level can throw off another

Understanding the endocrine system, from a basic science perspective – and with a “3D” vision- together with the conduction of robust clinical trials are needed to eventually warrant patients the access to better treatments as well as alleviate their symptoms or even ensure the re-establishment of a normal hormone balance and subsequent return to the normality.

There is not a universally accepted definition of the so-called “medical devices” although, in a very general way, any device intended to be used for medical purposes which exert its function by means that its not direct chemical, pharmacological, immunological or metabolic action, can classify within this category of products. Be as it may, it is generally accepted that medical devices help patients/users by providing new tools for diagnosis and providing solutions for the diagnosis, prevention, monitoring, prediction, prognosis, treatment or alleviation of disease.

Therefore, although different regulations exist across different regions – EU, US, Japan, other areas- it is generally agreed that an evaluation of risks derived from the use thereof shall be considered first. In response to this need, medical devices tend to be classified as a function of the potential risk derived from their use. Thus, for some devices, less or no clinical research at all is needed; for others, setup and conduction of those trials – with its proper particularities- is, on the contrary, required.

The immune system is the body’s defence against infections and other harmful invaders. It is indeed, an elaborate “network” encompassing different types of cells, tissues and organs all working together co-ordinately. And it happens that the immune system does not always function properly, generating a range of pathological conditions. Such malfunctioning of the immune system may be related to genetic errors in any of the genes building up the defences – primary immunodeficiencies- but also to other conditions that weaken our immune system – secondary deficiencies- or that induce a hyperactive response thereof against non-pathogenic agents – allergies- or that drive a non-desired response against the wrong threats – autoimmune diseases-.

All these scenarios lead to pathological conditions – more or less severe- whose potential treatments require a profound understanding of the immune system itself. Depending on the cause behind such malfunctioning, solutions might be quite different – from the prevention and treatment of occurring infections to “chemical” therapies to stem-cell transplantation, the use of gene therapies aimed to re-establish the correct functioning of what is damaged.

It is clear that children are not size-reduced adults, with both groups differing in their physiology and PK/PD profiles. Yet it is estimated that 50-90% of medicines for children are prescribed off-label – meaning that these medicines have not been tested (efficacy/safety) in the pediatric population. In this context, it becomes evident that pediatric clinical research is essential for the development of new and effective treatments to manage diseases affecting children, specifically. But this is not an easy task. The regulatory landscape is especially protective and conservative with this type of trials – which demands a profound knowledge thereof to setup any trial- but also because the design of pediatric clinical trials itself comes with specific associated challenges – that may include the preparation of the ICF itself, or the frequent restrictions in the use of placebo arms intrinsic to these trials, or even the questionable use of healthy volunteers as a control. Besides, when setting up a pediatric trial, enrollment difficulties have to be through and evaluated well in advance.

Non-interventional clinical studies (NIS) are studies in which the medicinal product under investigation is prescribed in the usual manner; that is, in accordance with the terms of the corresponding marketing authorisation. These studies are defined by the methodology used rather than by their scientific objectives. They comprise a number of approaches that include exploring databases, reviewing existing records or the prospective collection of primary data derived from routine clinical practice. Thus, and although medicinal products are used, the study protocol does not dictate the investigators what should be given to their patients. This is left to their clinical judgement and experience.