Specialty Pharmaceuticals: Policy Initiatives To Improve Assessment, Pricing, Prescription, And Use

Abstract

The value of "specialty pharmaceuticals" for cancer and other complex conditions depends not merely on their molecular structures but also on the manner in which the drugs are assessed, insured, priced, prescribed, and used. This article analyzes the five principal stages through which a specialty drug must pass on its journey from the laboratory to the bedside. These include regulatory approval by the Food and Drug Administration for market access, insurance coverage, pricing and payment, physician prescription, and patient engagement. If structured appropriately, each stage improves performance and supports continued research and development. If structured inappropriately, however, each stage adds to administrative burdens, distorts clinical decision making, and weakens incentives for innovation. Cautious optimism is in order, but neither the continued development of breakthrough products nor their use according to evidence-based guidelines can be taken for granted.

Trends in accessibility of negotiated targeted anti-cancer medicines in Nanjing, China: An interrupted time series analysis

“…In recent years, molecular targeted drugs and other innovative anti-cancer therapies have significantly prolonged the survival of cancer patients (9), alleviated pain and improved quality of life (10), thereby reducing the psychological pressure on family and community (11). However, due to the high costs of pharmaceutical research and development (12), patent protection of new drugs, limited national medical security capacity, skewed regional economic development, and differences in the diagnostic and treatment facilities of medical institutions, the availability of targeted anti-cancer drugs is severely restricted (13,14). A famous Chinese movie named "Dying to Survive" was released in 2018, which described a story of cancer patients who could not afford the high cost of anticancer medicines and had to purchase illegal generic drugs from India (15).…”

Impact of the National Health Insurance Coverage Policy on the Utilisation and Accessibility of Innovative Anti-cancer Medicines in China: An Interrupted Time-Series Study

“…Actively advocating for cancer prevention and promoting early diagnosis and treatment, supplemented by the early use of suitable innovative anticancer medicines, can help patients receive better treatment, thereby improving the overall five-year survival rate (6). However, due to patent protection and technology monopoly of innovative drugs, the cost of innovative anti-cancer medicines is usually higher than the catastrophic health expenditure, which imposes a financial burden on patients and hinders access to these medicines (7,8).…”

Branded vs. Generic drugs: the role of self-perceived seriousness of disease

“…Typically, because of their intake, it is difficult to ignore consulting a specialized professional figure such as the doctor, who prescribes the drug based on the scientific information provided to him by the pharmaceutical manufacturer (Schweitzer and Lu, 2018). Hence, the public authority has the need to regulate the times and methods of placing drugs on the market, to protect the final consumer (Robinson and Howell, 2014). Furthermore, before putting them on the market, pharmaceutical products are subject to an authorization (Marketing Authorization, AIC) for each type of packaging and controls at each production phase (Production Authorization) (Protiere et al , 2017).…”