Small Not Always Beautiful

Complaints from drug firms about the complexities of obtaining clinical trial authorizations in Europe-particularly for multi-center trials in more than one country-are common enough. The other side of that coin was spelled out by a Finnish cancer researcher at the end of 2014. Professor Sirpa Leppä, head of department at the Helsinki University Central Hospital Cancer Center, and Professor of Oncology at the University of Helsinki, pointed to the increasing challenge of inducing trial sponsors to select Finland. Leppä conducts trials with treatments for lymphoma-a condition that is diagnosed each year in 1,400 people in Finland. Recruitment is no problem, she says: Finns generally take a positive attitude towards trials. "It's very unusual for someone to decline participation in a trial."

"The difficulty lies in attracting new drug trials to Finland," she says, expressing concern over Finland's treatment in the selection process. “We could conduct more research into new and significant biological lymphoma drugs, but we have not been offered as many trials in recent years." Clearly, she acknowledges, part of the problem is that Finland is a small country. So "even though we have a good reputation, we are unable to recruit patient volumes on the same scale as bigger countries.”

For her the solution lies in a bottom-up approach that is an intriguing variation on the European Union's attempt at a top-down solution with its new clinical trial regulation. "The problem could be solved through Nordic cooperation," says Leppä. “Finland should not be treated as an individual unit, but rather as part of the Nordic countries"-Denmark, Finland, Iceland, Norway and Sweden. They have a good network in lymphoma research, "and considering the total population in all Nordic countries, combined with the high motivation of scientists and patients, we can easily compete with bigger countries in recruitment volumes.” However, as she notes-and as most readers of this column know only too well, "a separate permit is required for trials in each Nordic country." Since her remarks appear in the web magazine of the Finnish medicines agency, FIMEA, it can only be hoped that the Finnish authorities may be induced to redouble efforts for European countries to go further, much further, than the current attempt to improve EU rules.  

Meanwhile, the issue of size has also been preoccupying another small EU country with similar preoccupations about its capacity to attract trials. The Belgian chapter of the Association of Clinical Research Professionals ran a workshop recently on maintaining and improving the country's clinical trial competitiveness in the era of the new EU regulation. One of its findings is that the leading Belgian bottleneck in the clinical development process is access to patients. This is not uniquely a feature of Belgium's size-after all, its population is more than 11 million. The problem is compartmentalization within the country (a notorious problem at political level). "In Belgium, patients are not easily accessible because they are distributed over different hospital sites," says ACRP. Worse, there are few tools available-such as databases-to assist sponsors with recruitment, it laments.

In confirmation of the worst suspicions of the clinical trials community in Belgium and Finland, a survey published in BMJ Open in 2013 of the factors influencing trial selection put the size of market and number of eligible patients at the top of the list. However, it is not the only factor: as Leppä herself notes, "conducting clinical trials is expensive in Finland," which, she adds, "may be a contributing factor." And on the subject of money, Leppä also adds an interesting and infrequently expressed observation about one of the advantages of clinical trials-at least for the sites selected:

"Clinical trials generate significant savings for hospitals, as pharmaceutical companies provide the research drugs free of charge," she points out. “I don't think people quite understand how much we save in drug costs by conducting research sponsored by the pharmaceutical industry.”  That, at least, offers an agreeable counterpoint to the frequent complaints from trial sponsors about the increasing costs of trials.