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Medical, technological and legal progresses boost R&D of new medicines to fight rare diseases

17:54 Farmaindustria
World Rare Diseases Day is commemorated tomorrow

Madrid, February 27th 2017.- the progress experienced in the fields of research and biomedical technology, as well as the implementation of specific legal framework in Europe and the United States have allowed the pharmaceutical industry to intensify their efforts in recent years with the development of new medicines to fight rare diseases, the so-called ‘orphan medicines’, notably increasing the number of products available.

Only in Europe, has the approval of orphan medicines multiplied tenfold in 15 years since the entry into force of the Regulation for these specific medicines, passing from eight authorized products with this indication before the year 200, in which the Regulation started to apply, to 90 medicines in 2017, according to the data provided by the European Federation of Pharmaceutical Manufacturers and Associations (EFPIA).

The increasing efforts undertaken by the European pharmaceutical industry in this field is reflected, likewise, in the number of professionals whose companies engage in research and develop these medicines, which went from 2,000 to 5,000 in less than a decade after the adoption of the Regulation.

As far as the United States is concerned, efforts have also multiplied to find new effective treatments to treat rare diseases, affecting one in each 15 citizens on a global scale (more than 400 million people). There, where 30 million people suffer from some less common diseases, 230 orphan medicines have been approved during the last decade, and in 2015, 47% of total new medicine approvals corresponded to treatments for rare diseases, a figure which reflects the change of paradigm that the pharmaceutical sector is experiencing with regards to the tackling of less frequent pathologies, especially taking into account the situation experienced in the last decades.

New treatment options

“Many of these products provide treatment options for patients for whom there where only a few (if any) therapies available,” stresses Phrma, the US association for R&D based pharmaceutical industry.

In 2016, the pharmaceutical industry had other 566 potential medicines to treat rare diseases in different research and development stages, according to Phrma, among which were, 151 for less frequent tumors, 148 for genetic pathologies, 38 for neurological disorders, 31 for rare bacterial infections and 25 for self-immune diseases.

The higher bet for orphan medicines comes within a general context of increase of the costs to develop innovative medicines. Thus, according to the latest estimation, the marketing and development of a prescription medicine accounts for 10 to 12 years and an investment of around $ 2.600 million.

Still a long way ahead

However, together with the great number of these pathologies, (there are around 7,000 already described); the difficulties in their clinical research derived from the reduced number and geographical dispersion of patients, the trouble when reaching a previous diagnosis, the scarce number of centers with sufficient clinical experience, and the absence of valid biomarkers, make these diseases one of the greatest challenges in healthcare.

“The biology underlying these diseases can be very complex and the result incomprehensible; thus, the necessary research to fill in the existing gaps in the knowledge of these pathologies may be difficult and may take up a great amount of time, given the reduced number of people who suffer from these diseases,” remarked Phrma representatives. Notwithstanding, it is obvious that it we need to continue researching and improving the knowledge of these illnesses to succeed in developing effective treatments. “In spite of all these difficulties, it is the time of progress and hope. Thanks to the support of all these technologies and the increasing knowledge about these diseases, biopharmaceutical researchers have been capable to developed disruptive therapies during the last ten years.”

There is still a long road ahead of us. In this regard, the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) acknowledges that, “there is still a lot to do in order for rare diseases to be considered an international public health priority,” highlighting initiatives such as the Efpia-EuropaBio Task Force on Rare Diseases and Orphan Medicines, fostered 15 years ago by Industry in Europe, counting on the participation of 15 pharmaceutical companies.

Following this line of action, the pharmaceutical industry deems it necessary that the regulatory support comes backed up with a swift access to these medicines once they are approved; this would be the way to continue boosting the bet of pharmaceutical companies on this complex therapeutic field, so that they can provide, in the future, more and better results for patients.

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