"Orphan drugs", a key issue for the medicine of the future

Subsequently, the clinical trials undertaken to assess the benefits and risks of these treatments were also simplified, she underlines, noting that for the marketing of a classic drug, studies involving thousands of volunteers are necessary. , whereas for rare diseases, on the other hand, smaller cohorts of ten or a hundred patients are sufficient to prove the effectiveness of the product.

With regard to the deadlines for obtaining a marketing authorization (AMM), Dr. Moussayer specifies that they have been shortened by nearly 2 years on average. A set of measures that have lowered costs and encouraged research, she notes.

At the same time, associations of people with rare diseases have mobilized all over the world, says the specialist, like the French Association against Myopathies (AFM), which has played a decisive role in this area.

Indeed, this association has launched a Telethon since 1987 to finance research projects on neuromuscular genetic diseases, says Dr. Moussayer, which has raised a real collective awareness, leading to the formation of a “ Rare Disease Alliance », in 2000, with more than 200 associations representing nearly 2 million patients.

At the European level, the specialist indicates that the European organization of patient associations, Eurordis, born in 1997, is the prime contractor for the international day of rare diseases, which today has taken on a global dimension with the participation of associations from more than 80 countries, noting that Eurordis has contributed to the adoption of European laws on orphan drugs or innovative therapies.

Finally, the European States have completely integrated these diseases into their healthcare system by establishing national action plans which formalize the objectives and the measures to be taken, in particular with the development of national reference centers for expertise and centers of local skills for care. Four European countries had such a system in 2009 compared to 20 today “, informs us the president of the AMRM.

Essential research and development challenge for the pharmaceutical industry

Regarding the aspect of the pharmaceutical industry which has been one of the major sources of innovation with the marketing of new drugs, Dr. Moussayer argues that in 1983, there were only 40 drugs to treat patients. concerned informs the specialist whereas currently, the doctors have the choice between a little less than 500 different treatments. The fact remains that only 5% of rare diseases benefit from an approved treatment, she specifies.

The global pharmaceutical industry has not escaped the global economic crisis in recent years. Restructuring with job losses in this sector corresponds to a very tense situation on the world drug market. With the boom in generics and the expiration of patents, the turnover of the classic activity on – blockbusters -, these drugs relating to major diseases tend to decline. The future is for products with very high added value, biotechnology, treatment of rare diseases and vaccines “, explains the specialist.

Today, all the major laboratories have created their division in this field and are now investing billions of dollars in research on rare diseases, says Dr. Moussayer, especially since the latter suggests transposable solutions in the treatment more common pathologies.

Thus, Rituximab, initially intended for lymphomas, is indicated in the treatment of severe forms of certain autoimmune diseases such as anemia, rheumatoid arthritis or forms of organ transplant rejection. Research into progeria, accelerated aging, has enormous “potential”, because the protein in question is progerin, which is also defective in people with cancer. A discovery of effective molecules in this field would certainly have a side effect on chemotherapies, triple therapies and even normal aging. “, explains the specialist.

Estimated in 2015 at 100 billion dollars, Dr. Moussayer specifies that the market for orphan drugs rose to 175 billion in 2020 (Evaluatepharma: orphan drugs report) and is growing at an annual rate of 11% compared to 4% for conventional drugs.

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