Abstract
BackgroundPatients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically.MethodsIn this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients’ views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs.ResultsRespondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options.ConclusionWhen the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed.
Highlights
Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs
As we expected, based on the literature, that patients’ experiences with expanded access would be limited, we developed an interview guide in which we successively discussed standard of care and to what extent patients were aware of a standard of care and actively sought information about standard and non-standard treatment options
I don’t feel good about this. [...] You want these things to be accessible for everybody. (I3). This qualitative study was the first to provide insight into what patients with unmet medical needs want with regard to unapproved, investigational treatment options, and what they expect from their treating physicians
Summary
Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. Patients with unmet medical needs may wish to look beyond standard treatment options, and may be eligible to try investigational drugs: new drugs that are not yet approved for marketing and are still under investigation. In many countries, expanded access can be requested for individual patients, through so-called ‘named-patient’ programs, or for groups of patients, in so-called ‘compassionate use’ programs. The latter are often initiated by pharmaceutical companies after the successful completion of phase III clinical trials, to bridge the gap between the trial and the commercial launch of the new drug. In countries where expanded access is not reimbursed, the uptake is generally very low
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