UncategorizedRare diseases and orphan drugs, difficulties and statistics

7 January 20210

Rare diseases affect only or as much as 6 – 8% of humanity. They are very rare, but they do exist and people suffer from them.


In Poland disease is defined as a rare when for 10,000 people up to 5 of them become ill. If you take into account the number of people in Poland (almost 38 million), the rare disease is affected from 2.3 to 3 million people. Taking into account the voivodships, there may be up to 150,000 sick people in each.


Difficulty in such diseases is based on the diagnosis and treatment of patients. About 6 thousand rare diseases are diagnosed (there can be up to 8 thousands of them!) and the doctor does not meet with enough patients every day to easily and quickly recognize symptoms indicating a given bankruptcy, as it is the case with more common diseases.

The difficulty of therapy lies in the access to medicines, the price of their preparation and their low sales, which gives a small profit to pharmaceutical companies that will not invest in a product whose sale will not cover the costs of research and will not generate income. All these problems result from the small number of patients.


Orphan drugs – this is the name of medicinal products for rare diseases – are subject to the Regulation of the European Union, the European Parliament and the Council No. 141/2000 of 16 December 1999 on orphan medicinal products. The European Medicines AgencyEMA – consists, among others to make it easier for pharmaceutical companies to develop and authorize medicines for rare diseases.


Sponsors / Pharmaceutical companies that want to conduct research on an orphan drug can take advantage of a number of incentives offered to them by the EMA which supports the development of the drug, the registration procedure and giving orphan products the privilege of exclusive trading on the market for a period of 10 years*.

These incentives are aimed at increasing the amount of research on orphan drugs and lowering their prices.


More about incentives will be available in next article

together with procedures to designate an orphan drug


More and more research is conducted on orphan drugs thanks to EMA incentives but there is still a lot diseases to be diagnosed and to be treated. As you can find below number of application for orphan drug designation has inceread since 2003 and you can see which indications are in the interest of pharmaceutical companies.


Increase in orphan designation


FDA orphan designations increase 12% in 2014, European designations up 62% and Japan designations up 7%.



Non-Hodgkin lymphoma (NHL) has most orphan designations in the EU. It is a is a group of blood cancers that includes all types of lymphoma except Hodgkin’s lymphomas. Symptoms include enlarged lymph nodes, fever, night sweats, weight loss, and tiredness. Other symptoms may include bone pain, chest pain, or itchiness.


The second indication is acute myeloid leukemia (AML), a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal white blood cells that build up in the bone marrow and interfere with the production of normal blood cells.


Third is cystic fibrosis (CF), a genetic disorder that affects mostly the lungs. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs may include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in some males.


Top 10 companies in prescription drug sales

The first four companies are ranked within $240m of each other.


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