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Orphan Drugs - Medicines against rare diseases

Just recently, in May this year, the European Medicines Agency (EMA) recommended approval for three new orphan drugs. These are special drugs that are used to treat rare conditions or diseases. Many diseases are still incurable, and the development of therapeutic options is intended to give those affected by rare diseases the chance for a better life. To receive the title Orphan Drug, a product must be recognized and approved as such by the EMA. This approval process has some special features, as the research and development of such drugs is very callenging.

What is special about this procedure and with which difficulties the research is confronted here, you will read in this article.


Regulatory requirements for Orphan Drugs

Until the year 2000, it was very difficult for life science companies to bring drugs against so-called orphan diseases (= rare diseases) to market. In addition to the major obstacles in development, economic success was mostly absent, so that the expenditure for research was disproportionate to the economic benefit. The investment in research exceeds the income, because the number of people affected is usually too small compared to the development effort. To counteract this problem, the EMA decided in December 1999 to introduce a new approval procedure to make the development of such drugs more attractive for the pharmaceutical industry. Translated with (free version)

This is a voluntary procedure that is free of charge. If the drug is approved, it receives ten years of market exclusivity in the EU, independent of patent protection, which keeps even similar competitor drugs off the market unless they can demonstrate significantly greater efficacy or are intended to overcome a supply bottleneck.

Early in the drug development process, and in some cases years before marketing approval, orphan drug designation can be applied for from the Committee for Orphan Medicinal Products (COMP) based on disease models or early clinical data. Orphan drug designation gives access to pre- or post-approval benefits and privileges, but does not affect any of the basic requirements for drug approval.

The requirement is that the condition or disease to be treated must not affect more than one person in 2,000. Alternatively, a drug can also be approved if the company can verify that the profit from the sale of the drug would not cover the investment. In addition, the drug must provide a significant benefit, i.e. no satisfactory therapy may exist at the time of approval.


Facts about Orphan Drugs

Orphan drug designation by COMP does not affect the actual drug approval. For approval, the disease to be treated must be an orphan one. The EMA alone decides whether this is the case. Known diseases are, for example: arcomegaly (giant growth), Lennox-Gastaut syndrome (severe form of epilepsy in children) or narcolepsy (microsleep).

Approximately 8000 rare diseases are currently known worldwide. However, there are only about 200 approved orphan drugs. 68 drugs have already lost this status due to the expired maximum duration of ten years, or the status has been partially returned by the companies themselves. However, the future offers some hope. There are currently around 2400 orphan drugs in the pipeline awaiting approval.

The largest producers include Celgene with Revlimid against blood cancer, Roche with Rituxan against limph gland cancer, Novartis with Afinitor against pancreas cancer and Johnson & Johnson with Velcade against the multiple myeloma (=bone marrow cancer).

(The cancers listed here are each a particularly rare manifestation of the disease).


Challenges in Orphan Drug development

Development is similar to regular drugs in terms of quality, safety and efficacy criteria. However, there are many reasons why the development of orphan drugs remains a challenge.

The basic condition for the development of a drug is to know the exact molecularly understood disease process in the body. In the case of rare diseases, this is often the biggest obstacle. There is a lack of experts who are familiar with these diseases, and as a result, there is a huge knowledge gap.

Another hurdle is the small number of study participants. The limited number of people affected means that research and development can only draw on a very limited number of participants. In addition, the symptoms of rare diseases are often heterogeneous, which means that one and the same disease can manifest itself very differently in patients. This makes it difficult for experts to find the right approach for developing a pharmaceutical or biotechnological drug. Translated with (free version)

Due to a lack of knowledge and the low number of people involved, the development process involves a great deal of effort and enormous costs.

On average, the cost of development is 23 times higher than for normal drugs.

On the ethnic side, the limited knowledge and testing collides with the patients' desire for the therapy to be available quickly. However, this can hardly be guaranteed due to the challenges of development. Also, many patients are just children. Parents have to make a difficult decision here and weigh up whether participation in a drug trial makes sense at all because of the high risk for their children. Translated with (free version)


Criticism regarding the approval process for Orphan Drugs

The special approval procedure repeatedly earns criticism. The main reason for this is that simpler drug studies are accepted for approval than for ordinary drugs. Due to the small number of participants in these studies, they cannot be performed in the usual quantity. In these cases, new drugs are often used without a solid data basis.

In contrast to ordinary drugs, an exemption rule applies to orphan drugs when they are launched on the market: an additional benefit compared to other therapy options does not have to be proven by corresponding data, but is automatically assumed. This facilitation is intended to provide additional motivation for life science companies to conduct research in the field of orphan diseases.

Critics, however, demand that orphan drugs also have to prove their worth in the course of the early benefit assessment, because in many cases the assumed superiority cannot be proven and the promised therapeutic success fails to appear.

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