Expanding Use of Oral Therapies Puts Challenges of Patient Compliance in Spotlight

Maurie Markman, MD
Published: Friday, May 22, 2015
Maurie Markman, MD, from CTCA

Maurie Markman, MD

Until relatively recently, the use of oral antineoplastic therapy had been limited in routine oncologic care, largely restricted to hormonal treatments for breast cancer and a few cytotoxic agents such as capecitabine and etoposide. In recent years, however, an increasing number of targeted antineoplastic drugs with an oral method of delivery have been introduced into clinical practice.

This development is welcomed both for the overall convenience it offers patients and for the ability to continue treatment for prolonged periods while permitting patients a far greater opportunity to maintain their normal activities, including work. Further, since many advanced and metastatic cancers are increasingly and appropriately considered as very serious but chronic disease processes–with survival and treatment durations measured in years rather than weeks or months–the ability to optimize quality of life for these extended periods of time becomes critical.

In this context, enhancing an individual patient’s quality of life will likely include efforts to minimize the time spent receiving therapy, such as travel or periods away from work at the oncologist’s office for the patient and/or caregiver.

This effort becomes an absolutely essential component of an approach that recognizes the far longer duration of the routine disease management trajectory.

However, as we move into this new therapeutic paradigm, it is relevant that we consider unique aspects of this strategy that may substantially negatively impact outcomes, compared with an approach of systemic antineoplastic drug administration.

Table. Correlation of Outcomes With Compliance

Impact of adherence to imatinib therapy among 87 patients with CML

CCyR indicates complete cytogenetic response; CML, chronic myeloid leukemia; EFS, event-free survival; MMR, major molecular response.

Ibrahim AR et al. Blood. 2011;117(14):3733-3736.

In this regard, perhaps the most serious clinical concern with oral antineoplastic drug delivery is the fundamental issue of patient compliance with the physician-ordered drug regimen. The issue of compliance is not unique to the oncology arena and represents a major challenge for all areas of medicine. Fundamentally, many individuals do not like taking medicine and this can be particularly problematic in the setting of an individual who is (a) required to take many pills every day; (b) likely to continue therapy for prolonged periods of time (for life or until disease progression); (c) forced to cope with any unpleasant side effects that actually (or are perceived to) result from taking the medication; and (d) “feeling well” and not experiencing any direct benefit from taking the medication regularly.

Quantity, Size, and Shape of Oral Therapy

It is reasonable to expect that individuals required to take a number of pills every day or multiple times during a 24-hour period are likely to be far less willing to adhere to a prescribed regimen than if the patient only needs to take a single pill once a day. Further, if the pill is particularly large, has an unpleasant taste, or the patient has difficulty swallowing pills, one can certainly anticipate difficulties with adherence to the drug program.

While one might even speculate that liquid delivery of a medication (assuming reasonable taste) might be more acceptable in settings otherwise requiring multiple pills, accurately measuring the appropriate quantity for delivery will pose a significant challenge, as will the fact that a liquid form is likely to be far less convenient for individuals who do not want to be largely confined to a single location due to the difficulty associated with transport of the medication.

Unique Aspects of Generic Oral Medications

Although not currently a major issue with oral antineoplastic agents (except perhaps in the arena of hormonal therapy), it is increasingly recognized that there are clinically relevant concerns when any patented oral medication becomes generic and is available from several possible manufacturing sources. In a most provocative report, investigators studied the impact of a “change in appearance (shape or color)” of pills when employing a generic oral cardiovascular agent, in this case a beta-blocker, angiotensin-converting enzyme inhibitor, angiotensin II-receptor blocker, or statin.1


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