Should patients with no further treatment options be given access to unregistered drugs through a commercial company? And if so, under what conditions? Researchers in the NWO MVI Responsible Innovation programme are examining this question.

myTomorrows is sometimes called the Uber of the pharmaceutical industry. The start-up brings patients who have run out of treatment options in contact with drug manufacturers and physicians through an online platform. The patients are therefore given one last chance: to purchase drugs that will not be available for some time to come through the normal channels because they have not yet been fully tested. This is an innovative plan, and may save lives, but it is also highly controversial. It is therefore time to assess the possible impact of this initiative.

Team
Medical ethicist Eline Bunnik is conducting the research together with Suzanne van de Vathorst, physician and ethics lecturer at Erasmus MC and professor of quality of the terminal phase of life at the AMC, and Loes Visser, hospital pharmacist in The Hague and associate professor in pharmacoepidemiology at Erasmus MC.

Search for a final cure
“Governments, drug manufacturers and physicians used to have all the time they needed to decide when a new drug was considered safe and effective enough,” says Bunnik. “The authorisation procedures date from that time and they are very thorough; on average, it takes almost 15 years for a new drug to be authorised. However, many patients who have run out of treatment options no longer have the patience for this, and they or their families now trawl the internet in search of a final cure. If they see that a new, but as yet unregistered, drug could save them or their child, they will do anything they can to get it.”

Protect patients
A company like myTomorrows meets this demand. Why, therefore, is it so controversial? “Physicians and patient associations believe that terminally-ill patients must be protected from themselves,” explains Bunnik. “They are scared of giving people false hope and believe that it is better that people are allowed to die in peace than continue to fight to the bitter end. It is even feasible that, in their desperation, people will sell their home to pay for an expensive drug that only offers a small chance of extending their life.”

Ask patients
In this project, the researchers will find out whether this concern is justified. They will start by asking the patients themselves. Do they think that they need protection? “We are going to hold in-depth interviews with people who have made use of the possibilities for early market authorisation and with people who have chosen not to. We want to identify their wishes and experiences. The field of ethics provides us with the instruments we need to classify all the different arguments.”

Analyse physicians’ arguments
The researchers will also interview physicians. “We want to get an idea of the process that they go through when deciding whether or not to recommend that someone tries an unregistered drug. At what point do they consider a new drug to be sufficiently promising to start the experiment? Do they consider the patient’s financial situation and perhaps decide not to point a patient in the direction of myTomorrows if they think he or she cannot afford an expensive drug? How large are the differences between physicians?”

Overview and recommendations
By combining systematic data collection and ethical analysis, the researchers hope to produce a good overview of the factors involved in the early use of experimental drugs. Based on their findings, they will also make recommendations for future policy.