Expensive drugs for rare diseases have little or no effect



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Very expensive drugs for rare diseases often do not work. Researcher Yvonne Schuller of the UMC of Amsterdam says it in Trouw

She examined drugs for rare cancers and metabolic diseases. These drugs cost a capacity, have been tested and approved, but they show no effect on a large part of the diseases.

Unresolved Probability of Survival

Schuller discovered that four out of ten drugs for rare tumors did not appear to have any chance of survival increase, although they were on the market. For drugs against metabolic diseases, even 62 percent were unclear if there was an effect.

Research on these drugs is difficult and expensive because there are so few patients and the diseases are too complicated to establish in a few years. to take pills. Patients also carry the disease all their lives, so that drug research is often only a snapshot.

For his research, Schuller consulted clinical experts and patients and studied scientific assessments in collaboration with the Medicines Evaluation Board

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