Bottlenecks in pharmacotherapy
Ensuring that a newly registered drug reaches as many as possible benefiting patients is primarily about reimbursement and subsequent drug utilization. While the former can largely be controlled by license holders setting the required reimbursement price and the proposed indication criteria, the use of medicines may be entirely beyond their control. The seemingly easy course of many chronic diseases associated with the late onset of symptoms is often responsible for treatment delays. However, any delay in starting treatment has a particularly adverse effect, and early treatment helps to prevent early progression in many diseases, such as neoplasms, autoimmune diseases and neurological disorders. It has been repeatedly shown that preventive and diagnostic screening offers major health and economic benefits by facilitating the timely treatment of curable or manageable early manifestations and simultaneously preventing progression to often incurable stages of disease.
Similarly, bottlenecks in the availability of specialised diagnostics may hinder the appropriate treatment of many patients with known or unrecognised chronic conditions, such as cardiovascular diseases, osteoporosis or autoimmune diseases. This mainly refers to the use of expensive drugs, which are conditioned by the diagnosis of specific diagnostic markers. Often, patients are kept in early lines of therapy for too long by a specialist or even a general practitioner who made the primary diagnosis before—if ever—they are referred to a specialised centre to receive advanced treatment (e.g. biological). There are several reasons why patients may not receive medication appropriate to their condition:
An asymptomatic course of a condition prior to exacerbation of the disease, such as osteoporosis;
Poor awareness of the underlying condition and its severity by the patient, for example, in COPD;
Poor awareness of the underlying condition and its severity by a doctor, such as in rheumatoid arthritis, which requires treatment that is only available at designated centres;
Generally insufficient or unavailable diagnostic equipment, for example, in the event of large-scale pandemics;
Misdiagnosis, for example, with rare diseases diagnosed by exclusion;
False negatives, such as in infectious diseases diagnosed by low-sensitivity tests, such as sputum culture tests;
Legislative and administrative burdens, including the subsequent replacement or unavailability of diagnostics at an early age.
Wherever diagnosis bottlenecks result in inadequate treatment for any of the above reasons, it is in the interest of society, the patient and the manufacturer to raise awareness of this fact. However, data that clearly show that a fraction of patients fail to receive treatment are often lacking. Existing reimbursement claim records, registries or other data are severely skewed because they only track the passing cohort.
In order to obtain a representative picture of the actual situation, it is often necessary to perform a cross-sectional study on the use of a treatment in real conditions. Due to the specifics of disease states, the design of such studies must account for the patient’s path of treatment through various specialisations, diagnostics and other medical facilities. Appropriate tools for randomisation and correct reading of data are needed to obtain an unbiased picture of the actual situation suitable for publication. Despite all the difficulties in conducting and publishing a treatment utilisation study, it is often worth the effort. Strong evidence of inadequate treatment of diseases accompanied by an assessment of economic impacts provides a strong and convincing argument for promoting a new prevention and treatment policy for health professionals, payers and governments.
