One of every six clinical trials that is carried out in Spain is already to investigate orphan drugs

Source: farmaindustria.es

The commitment of the pharmaceutical industry to the research and development of treatments for rare diseases is indisputable and, as the data show, growing. About 16% of the clinical trials (15.8%) promoted by pharmaceutical companies in Spain between 2015 and the first half of 2018 (one in six) already correspond to medicines designated as orphans and, therefore, aimed at treating some little prevalent pathology. With more specificity, of the 761 studies that the sample contains, 121 correspond to this type of drugs, and they involve the participation of 2,356 patients. These are figures obtained from the database of the BEST Project to promote biomedical R & D promoted by Farmaindustria, which monitors the research activity of 50 pharmaceutical companies, four cooperative research groups and 54 hospital centers in the country.

This information has recently been updated with a retrospective approach and compared with the Spanish Registry of Clinical Studies (REEC) of the Spanish Agency for Medicines and Health Products (AEMPS), as it was stated on Wednesday Amelia Martín Uranga, responsible for the Platform of Innovative Medicines of Farmaindustria, in the round table Industry and research in rare diseases within the IX International Congress of Orphan Medicines and Rare Diseases, which was held from 13 to 15 February in Seville.

Remarkable improvement

The BEST Project figures presented in Seville reflect a growing activity of the innovative pharmaceutical industry in the field of R & D in rare diseases. In fact, and after the review, the previous ones, published last year and which spoke of 10.8% of orphan drug trials in the 2015-2017 period, improved significantly.

Territorially, this research effort is concentrated especially in Catalonia and Madrid, followed by Andalusia and Valencia. By areas of pathology, a higher percentage of clinical trials were recorded in rare tumors (42.1%) and hematology (36.4%), followed by respiratory (4.9%) and cardiovascular (4.1%).

The analyzed sample reflects that the management indicators of clinical trials in orphan drugs (processing and start-up times or recruitment data) are similar to those of the other studies, although with a couple of nuances. Both the time required to recruit the first patient and the percentage of centers that do not recruit can be improved, although they have the obstacle of finding suitable patients for a clinical trial in these pathologies.

Strong commitment of pharmaceutical companies

Martín Uranga explained that Farmaindustria faithfully reflects the growing commitment of companies associated with research in this field and exposed the potential usefulness for the promoters of clinical trials in this area of ??two projects developed by the Association in recent years: the Guide of Clinical Research Units in Early Phases, whose third edition will be available in the coming months and which already gathers information from 37 centers, and the Clinical Research Guide in Pediatrics, published in September 2017 and with information on 30 units.

He also highlighted the importance of public-private collaboration in this field and pointed out two IMI cooperation projects between the European Commission and the pharmaceutical industry as suitable examples of this cooperative research model that is already a reality. On the one hand, the Connect 4 Children initiative (C4C), in which 33 academic partners and 10 pharmaceutical companies work, and one of whose objectives is to promote new designs for clinical trials that facilitate the development of treatments against rare pediatric diseases. The other proposal is Paradigm, which aims to better articulate the meaningful participation of patients in the development of new drugs, a field in which patient groups and relatives of rare diseases offer extensive experience.

The fruit of this increasing research effort is already a reality in the data of approval of new treatments by the regulatory agencies. Thus, of the 42 new molecules approved in 2018 by the European Medicines Agency (EMA), half of them (21) were orphan drugs. Similarly, 34 of the 59 treatments approved (58%) by the FDA, the US agency, were designed to address some rare pathology.

The representative of Farmaindustria pointed out, in the future, the importance of the advances reached in the field of genomics both for the best diagnosis of low-prevalence diseases (80% are of genetic origin) and for the development of new treatments. Therefore, he stressed the need to develop a National Precision Medicine Plan that includes the field of genomics as a mainstay, as recommended by the Senate Genomics Study Report.

The efforts of the pharmaceutical companies have also been recognized by the organizers of the Seville meeting. Thus, among the conclusions of the Congress highlights the message that “the pharmaceutical industry is now aware of the importance of the development of new products for the diagnosis, prevention and treatment of rare diseases.”

In Distefar we echo this news that reflects the growing activity of the innovative pharmaceutical industry in the field of R & D in rare diseases.