Film portrait Nicole Schlautmann

How innovative drugs for rare diseases are developed

Innovative drugs can only be developed with high investments.

"Thank you for my life!" The man who says this sentence to Nicole Schlautmann has a disease that drastically shortens his life expectancy. There are few other people in Europe with the same diagnosis - they have all passed away at his age. The fact that he can tell Nicole Schlautmann's former employer, a small pharmaceutical company headquartered in Paris, about his health progress and challenges is largely due to a drug that this company developed shortly before. 

It is moments like this that make Nicole Schlautmann think, "I never want to work in any other field again." The biology graduate then moves to Pfizer in Germany, where she was responsible for the Rare Diseases therapeutic area at the time of filming. Here, as well as in her current position as Managing Director of Pfizer Austria, she can make an even greater contribution to giving hope back to people for whom there were previously no therapies and who also fall through the cracks far too often in the healthcare system: "When children who never learned to walk before are suddenly running around, or people who would never have experienced their daughter's wedding are now there after all, it's gigantic!"

Artikel Teaser Bild Schlautmann
Nicole Schlautmann

However, the effort required to develop drugs for rare diseases is also enormous. Before a drug is approved, it must be tested in clinical trials. This requires a minimum number of test subjects. With more common diseases, these are easier to find. But if a disease is rare, the search involves hundreds of sites around the world. That makes logistics more difficult - and expensive.

At the same time, the market for rare disease therapies is by definition very small. There are often only a few experts, and there is a lack of scientific knowledge compared to larger indications. Each pharmaceutical company carefully considers the area in which it can build up expertise: "We can't develop a drug for which we know from the outset that we won't be successful in bringing it to market," says Schlautmann. "But so far we have always made quite good decisions in this regard, right down to indications for which we know of fewer than 300 patients in Germany. We can manage that," says Nicole Schlautmann.  

But often drugs for rare diseases are relatively expensive compared with drugs developed for a large number of patients. Nicole Schlautmann explains: "Of course, a drug that I treat 100 patients with has a different price than one for 100,000 - the development costs for both are often just as high. As a listed company, we need a certain level of sales, otherwise we can't develop new drugs. I'm only successful if the bottom line is so high that I can invest in the next drug."

Pharmaceutical companies take a big risk when researching new drugs. They invest billions - with an uncertain outcome. This is because only a very small proportion of the substances they research make it through to approval. This makes it all the more important for pharmaceutical companies to be able to achieve commercial success with approved drugs. The next step is for effective therapies to reach the patients who need them. This requires interlinked care structures. After all, only a correctly diagnosed patient can receive adequate treatment. 

The care of people with rare diseases, with or without therapy options, is often costly. Schlautmann says society needs to be made aware of this: "Patients with rare diseases have the same right to diagnosis and treatment as everyone else. And it is absolutely unacceptable to me that this is not yet the case today."


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