She knows it all too well. Hospital visits, examinations, ignorance, uncertainty and the frustrations. Wendy Olsder’s brother, three years younger, has a genetic metabolic disorder, a rare disorder for which there are no medicines. A situation with a significant impact on the family. But Wendy is the roll-up-sleeves-and-go type.
During her primary and secondary school years, she repeatedly gathers a group of friends to raise money for research into medicines for her brother’s condition.
And when, after completing her Industrial Engineering studies at the RUG, she comes across a PhD project that revolves around improving the availability and accessibility of medicines for rare diseases, she immediately knows that this project is for her, she says enthusiastically.
Behind the wheelchair
“I have been interested in the processes within healthcare from an early age. You can easily come into contact with other people with a rare disease if you walk behind your brother’s wheelchair. One wheelchair says to the other … Well, that’s just how it works. And of course through the meetings of the patient associations. I know from my own experience what patients with a rare disease encounter.”
“It starts with the problems of making the correct diagnosis, to suboptimal care due to a lack of knowledge and expertise. I now have the opportunity to contribute to these issues at a scientific level. A fantastic opportunity.” After a diagnosis, a medication is not always obvious as the next step.
Worldwide, about 350 million people suffer from a rare disease; you speak of rare when it affects less than 1 in 2000 people. In total there are about 7000 known rare diseases. Medication is available for only 500 of these conditions, explains Olsder. She lists the reasons on her hand.
“There is too little knowledge about the disorder itself, the patient group is too small for clinical research, and the market is too small. It is not attractive for a pharmaceutical company to invest in the development of a so-called orphan drug – a drug for the treatment of a rare disease. The costs are high, the market is small.
When a drug does eventually come onto the market, the pharmaceutical company then asks a sky-high price for it. This has a major impact on our healthcare budget. That is why I and my colleagues have looked at whether we can change this entire system in order to break the vicious circle.”
Unique arithmetic model
To make discussions about this complex issue more concrete, Olsder developed a mathematical model. Because, somewhat to her surprise, this did not yet exist. “We have really taken a big step with this. Because with this model we can scientifically demonstrate which aspects can make the entire process more efficient.” One of the first aspects that Olsder investigated was whether the current way of granting subsidies needs to change.
“To encourage pharmaceutical companies to make more orphan drugs available, they receive subsidies from the government. But because they then charge very high prices for the medicines developed, access for patients remains limited. Surprisingly, we see that the subsidies themselves are not so much the problem, but that we need to focus on price regulation.”
“An initiative is now underway within the Benelux in which a consortium of various stakeholders has direct influence on the price of a number of orphan drugs. The first results already show that this is a good option to keep the costs of orphan drugs realistic.”
Another important conclusion of Olsder’s research is that medicines can be made available more quickly in the preliminary phase. “There is of course a whole approval process behind that. But there are several ways to speed up bureaucracy. For example, by issuing a temporary approval, so that patients who would otherwise die have earlier access to a new medicine.
The European Medicines Agency – EMA – also wants medicines to reach patients more quickly and is already running a number of pilot studies with medicines that reach the market via a different route. My thesis contains several suggestions for this adaptive approval program to improve even further. It is good to see that there is growing attention for rare diseases both in the Netherlands and at European level.”
Double cap
The fact that she has ‘a double hat’ makes Olsder extra motivated. She herself was told at the age of fourteen that she has a rheumatic disease after a long process. She immediately went into action. First by organizing events for the patient association, but she soon became a patient representative in various rheumatism studies.
In addition to her papers on her PhD research, her publication list includes many articles about the rheumatism research projects in which she participated. “I can really bridge the gap between scientific research and patient organizations.” Olsder recently started working as an assistant professor at the Erasmus School of Health Policy & Management, where she will continue her PhD research.
Her double hat is not coming off for the time being. Because at the same time she started as a trainer ‘Patient participation scientific research’ at knowledge and advice organization PGO Support. “Yes, I get tired faster because of my rheumatism, and I have a very flexible schedule because of my limited energy. That means that sometimes I leave the office after an hour. But being able to really contribute to health research from both sides in this way also generates a great deal of energy and satisfaction. That is more than worth all that planning.”
