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Orphan Drugs

What Are Orphan Drugs ? Beneficial for Rare Diseases ?!

In general, orphan drugs are those that are intended to treat rare diseases, which put integrity or life at risk.

Orphan drugs are called those medicinal products that serve to prevent, diagnose or treat very rare diseases . Such diseases must be very serious or, in any case, they must constitute a threat to life.

They are called orphan drugs because the pharmaceutical industry has no interest in developing this type of medicine and putting it on the market. This, because they are not profitable, since they are intended for a very small group of patients .

Precisely these medicines are destined to the so-called “orphan diseases”. These are rare pathologies that affect only five people for every 10,000 patients. According to the World Health Organization ( WHO ) there are 7,000 rare diseases of this type .

What are orphan drugs?

A very precise definition for orphan drugs is the following: they are drugs not developed by pharmaceutical companies, due to financial reasons, but that respond to public health needs.

There are three types of orphan drugs. They are the following:

  • Orphan drugs destined for rare diseases . It is the typical case. These are drugs that can only be used by very few people, who have rare or very rare diseases.
  • Medications withdrawn from the market . These are drugs that have been withdrawn from the market because they discovered some danger in them. However, they deprive those affected by certain diseases of treatment.
  • Drugs not developed . They are medicines with the potential to serve some diseases, but they are not commercialized because there are problems with their patent, or because they affect in some way dominant markets.

In all three cases, the common point is that they are products that are requested by patients, but are not made available to them for the reasons stated.

How are orphan drugs designated

How are orphan drugs designated?

Orphan drugs only develop when there are incentives to do so . This usually happens when the State or the private company intervenes and provides favorable conditions for the pharmaceutical companies to do so. The first step in that process is the designation.

For a drug to be designated as an orphan drug, it must meet certain criteria . In developed countries these criteria change:

  • In the United States . Make it useful to treat diseases with less than 200,000 patients per year. This corresponds to a prevalence of approximately 7.5 cases per 10,000 inhabitants.
  • In Japan . That it serves to treat 50,000 patients a year, which equates to a prevalence of 4 cases per 10,000 inhabitants.
  • In the European Union . That it serves to treat 250,000 people a year, which corresponds to a prevalence of 5 cases per 10,000 inhabitants.

The fact that a drug is designated as an orphan drug and that it begins to develop, does not imply that it is approved. First, it has to offer evidence of its safety, quality and effectiveness .

Buying orphan drugs

Access to orphan drugs.

It should be taken into account that the development of a new medication is a complex process. It usually takes about 10 years from the discovery of the molecule, to the commercialization of the product . Therefore, to recover the investment it is necessary that the medicine reaches a high number of people.

As noted above, only orphan drugs are developed when there are incentives. If this happens, the access routes for patients are basically three:

  • Direct acquisition . If the orphan drug is developed and marketed, it can be purchased through laboratories, like any other medicine. Sometimes this varies from country to country.
  • Indirect acquisition . When the drug has been developed, but is not marketed in the country, it is usually necessary to process a petition before the health system to allow its importation and acquisition.
  • Conditional acquisition . Corresponds to cases in which the orphan drug is not fully developed. In that case, patients must meet certain criteria to be included in a clinical trial.

Between 5% and 7% of the world population suffers from a rare disease. Most of these pathologies have a chronic and degenerative evolution. That is why, from many sectors, greater attention is being called to the issue of orphan drugs.

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