The shortage of verteporfin will inevitably lead to irreversible vision loss in a large group of patients in the Netherlands and elsewhere. Researchers from the Ophthalmology department at Amsterdam UMC (led by ophthalmologists Prof. Camiel Boon and Prof. Reinier Schlingemann) have recently published an article on this current problem in the journal Acta Ophthalmologica in collaboration with researchers from Medicine for Society (a platform that is committed to keeping medication for rare diseases available, led by Prof. Carla Hollak). An overview of the causes of this situation and implications for ophthalmological patients in the Netherlands and the rest of the world are provided, and it is explained why usual solutions for a drug shortage are difficult to apply in this case.
Shortage of verteporfin
Verteporfin is a drug that is injected into the bloodstream and then activated in the eye through a specific local laser treatment: photodynamic therapy (PDT). This slows down the growth of abnormal blood vessels and induces changes in the choroid of the eye. This way, permanent damage due to the leakage of fluid under the retina can be prevented, and visual function can often be improved significantly. There are several conditions of the eye that can be treated with PDT laser treatment. In the Netherlands, PDT is performed in approximately 700 patients on a yearly basis. Since the start of the verteporfin shortage, only 20-30% of these treatments could be performed with the remaining stocks that were available. Because of the complexity of this molecule, it was deemed impossible for pharmacists to start producing verteporfin. That is why the Workgroup Medical Retina of the Dutch Ophthalmological Society created a special committee to ensure that these last stocks would be used for the patients that need them the most – based on carefully selected criteria. The Netherlands was the first country to introduce such a system.
In the scientific article that was recently published by the group of Boon and Schlingemann, prominent retinal specialists from around the world were asked to fill in a questionnaire to create an image of the verteporfin situation in their countries. Thus, it was established that the treatment with verteporfin is used in large parts of the world and that the current shortage has had an enormous impact on ophthalmic patient care. In the current shortage, a multitude of alternative treatments are being used, but these treatment options do not provide equal effectiveness to verteporfin. The Netherlands is one of the few countries that have established a system to distribute the remaining ampoules of verteporfin on a national level, alongside France, the United Kingdom, and Canada. The results from the questionnaire indicated that there is a worldwide consensus among the experts that the shortage of verteporfin is a big problem and that endeavors should be made to effectively react to drug shortages such as this one.
Possible measures to prevent scarcity
However, an adequate solution is hard to find. Currently, there are no other companies or factories producing verteporfin. The creation of a new production line is not hindered by a patent in this case but would be so costly and time-consuming – with uncertainty regarding a stable market – that no other manufacturer has attempted this. Unfortunately, no other manufacturers have been able to produce verteporfin due to the complexity of the molecule and the lack of raw materials. Therefore, it is important to ensure that future drug shortages can be covered, like keeping a (national) stock of essential medication or introducing a second manufacturer. The complete assessment of possible measures that can be taken is discussed in the article.
Read the publication in Acta Ophthalmologica: Clinical impact of the worldwide shortage of verteporfin (Visudyne®) on ophthalmic care