Incentives For Drug Development Research Articles

The innovation paradox: pharmaceutical marketing exclusivity and incentives for drug development

The innovation paradox: pharmaceutical marketing exclusivity and incentives for drug development

PHP294 - A REVIEW OF RECENT APPRAISALS FROM THE INSTITUTE FOR CLINICAL AND ECONOMIC REVIEW: HOW IMPORTANT IS THE ICER?

The Institute for Clinical and Economic Review (ICER) in the United States utilizes economic analyses to determine the economic value and affordability of interventions, and estimates value-based price benchmarks. Our objective was to review recent appraisals conducted by ICER and to assess what factors are critical to their decision making, with a focus on health economics. Published appraisals from January 2016 to April 2018 (14 appraisals) were reviewed, with key data extracted. We excluded 1 non-pharmacological appraisal. ICER recommendations were compared against the economic analyses and stated critiques. Estimated ICERs ranged from dominant to $1,907,822/QALY; 5/13 appraisals were favorable to the evaluated drugs from a cost-effectiveness analysis (CEA) perspective: treatments were perceived to fulfill an unmet need and/or to demonstrate good value. Average per-patient budget impact estimates over a five-year time horizon were -$178,911 in appraisals that reported potential cost savings and $136,323 in appraisals reporting additional spending. CEA results drove decisions to not recommend an intervention, frequently due to high cost. However, for positive evaluations, CEA results were just one consideration. Whilst ICER acknowledged a need to keep incentives for drug development, most reviews concluded that substantial discounts would be needed to achieve thresholds. On average, discounts from wholesale acquisition cost of 83% and 53% were needed to achieve the $100,000 and $150,000 thresholds, respectively. Criticisms focused on the high cost of health technologies, limitations of trials, and absence of long-term data. A strong evidence base for economic modelling appears to be an important factor in the ICER appraisal process, including long-term data, head-to-head trials, generalizable data, and ideally robust data for subgroup evaluations. It highlights some of the ways in which manufacturers may improve the evidence base for their intervention and in turn reduce the uncertainty with future ICER reviews.

Superbugs: An Arms Race Against Bacteria

Superbugs: An Arms Race Against Bacteria

Implementing Intellectual Property of Pharmaceuticals in Middle-Income Countries: A Case Study of Patent Regulation in Brazil.

The protection of pharmaceutical intellectual property (IP) rights is one of the most controversial debates in contemporary public health as countries have to balance incentives for drug development with the necessity of providing life-saving drugs. Compliance with IP protections is mandatory for members of the World Trade Organization (WTO). However, because of the costs associated with IP implementation we should expect late and/or poor implementation in middle-income countries. Surprisingly, this was not the case in Brazil. The country not only just fully implemented the WTO's requirement but declined the grace period granted for countries to adapt and included extra IP protections, going against a coalition of local industrialists and activists. Notwithstanding, as the consequences of IP regulations unfolds, Brazil also promoted new alliances that tailored and adjusted the regulations toward public health. We demonstrate that arguments of foreign pressure and lobbying are exaggerated and call attention to domestic shifts, long-term processes of regulatory decision, and political dynamics happening at the local level. By analyzing the case of Brazil, we provide a nuanced contribution to the discussion of IP implementation in middle-income countries and call attention to new models of government-society interactions in regulatory policy.

When drugs in the same controlled substance schedule differ in real-world abuse, should they be differentiated in labeling?

The prescription drugs regulated in the most restrictive controlled substance schedule for those with an approved therapeutic use vary widely in their real world risk of abuse and harm. Opioid analgesics have the highest rates of abuse, overdose death, drug abuse treatment needs and societal costs in comparison to other Schedule II drugs. Stimulants for attention-deficit/hyperactivity disorders (ADHD) account for substantially lower rates of abuse, harm, and public health impact. The scheduling of drugs is determined by the World Health Organization, the United States Food and Drug Administration, and other regulatory agencies, through a quasi-public process that relies heavily on pre-marketing studies that are conducted in highly controlled clinical settings. We propose that it is increasingly in the interest of science-based regulation and public health to recognize and communicate differences among drugs based on their real-world abuse and public health harm using surveillance data. Appropriate differentiation through labeling of drugs that will likely remain in the same schedule could provide powerful incentives for drug development and research, would aid prescriber/patient decision making by informing them of real differences across drugs within a schedule, and may also contribute to public health efforts to reduce drug abuse. There are risks of course, that include inadvertent perceptions that drugs labeled to be lower in risk are not taken as seriously as others in the same category. Challenges such as these, however, can be overcome and should not serve as barriers to objective communications regarding a drug's actual risks.

Incentives for Drug Development — The Curious Case of Colchicine

Incentives for Drug Development — The Curious Case of Colchicine

Incentives for Drug Development — The Curious Case of Colchicine

Drs. Aaron Kesselheim and Daniel Solomon write that it came as a surprise to many patients and physicians that the FDA not only approved the new version of colchicine but also granted the manufacturer 3 years of market exclusivity for this ancient drug.

Erosion of the scope of the Orphan Drug Act in the United States

The Orphan Drug Act provides incentives for drug development in patients with rare diseases. To qualify for benefit under the Act, Congress imposed a prevalence limit of 200 000 patients in the United States. Assuming disease prevalence to be constant, the number of rare diseases that qualifies under the Act is gradually declining because the population is growing. The maximum prevalence for a qualifying disease can be estimated by comparing the 200 000 patient limit with population estimates provided by the United States Census Bureau. Among a sample of diseases with investigational drugs that have achieved Orphan Drug Designations in the past, many are too rare for this erosion to have practical effect. However, for several diseases, disqualification under the Act is likely in the medium term, and there is one example (bladder cancer) that became disqualified about 10 years ago. There are invidious implications for both pharmaceutical companies and patients. Some ways to revise the law, e.g. providing Food and Drugs Administration with authority to improve the regulations, or index-linking numbers of affected patients to the national population, are suggested.