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What are orphan drugs?


Orphan drugs are pharmaceutical therapies authorised to diagnose, prevent or treat a condition that may threaten the patient's life or is very serious, rare, and affects less than 5/10,000 people within the European Union territory.

They are called ‘orphan’ because due to the low number of cases, the market cannot guarantee the profitability of the investments needed to develop these drugs, so the pharmaceutical industry has little interest in them. The professionals who research these drugs are the same ones that research all other drugs, but the conditions to do so are much more complicated.

Not all rare diseases are treated using orphan drugs. An orphan drug can be needed for the treatment of a rare disease, but not all drugs used to treat rare diseases are orphan drugs. There are rare diseases that are treated with drugs that are not qualified as 'orphan' and that have been commercialized following general procedures.

The process from the discovery of a new molecule to its commercialization is long (an average of 10 years), expensive (tens of millions of Euros) and very unsafe (out of ten tested molecules, only one tends to have a therapeutic effect). Developing a drug to treat a rare disease does not allow, generally, to reap any profits.

The companies that manufacture them are usually the same ones that manufacture other drugs, but they need special funding that normally only governments can give. These can be direct aid or tax benefits and other kinds of exemptions.


There are different ways to get hold of orphan drugs:

For those orphan drugs commercialized in Spain, hospitals can acquire them via the supplier laboratories (the usual established procedure to acquire any drug).

For those orphan drugs not commercialized in Spain, but commercialized elsewhere, they can be obtained through the procedures established to acquire foreign drugs, via the Ministry of Healthcare, Social Policy and Equality, which authorises such imports.

Access to orphan drugs under investigation is more restricted. Nevertheless, there are two ways to obtain them:

Drug access through the patient’s incorporation to a clinical trial, if any are using the required drug (the patient must meet the selection criteria written on the trial’s protocol).

Request the Ministry of Healthcare, Social Policy and Equality the necessary authorization to access the drugs through the procedure ‘Compassionate use of drugs under investigation, which regulates the availability of drugs in special cases (Reial decret 1015/2009).

Unfortunately, orphan drug allocations, aid protocols and commercialization authorizations are centralized procedures, but the evaluation of the therapeutic value, the cost and the reimbursement of these innovative drugs are under the responsibility of each member state.Orphan drugs search engine.