Drug Selection and Adverse Drug Reactions: Balancing Outcomes and Costs

Regulatory agencies approve drugs for marketing when the ratio of risk to benefit of the drugs is positive, i.e. the potential benefit of using the drug justifies the risk of drug related adverse reactions (ADR). At the point of approval of a drug there is not adequate information about all possible negative outcomes arising from its use in clinical practice. The information is even more limited for patient subgroups, diseases and treatment combinations that were not evaluated in the clinical trials required for approval. Therefore, performing post-marketing pharmacoepidemiological studies is necessary for a better understanding of drug use in usual clinical practice conditions and the effect of the drugs on the general patient population

We Need a Labeling System for Pharmaceutical Prices

As we explained in the Annals of Internal Medicine earlier this year, prescription drug purchases rarely mirror reasonable expectations, partly because the information gaps are extraordinary. With many patients, there is just no way to know up front what will be covered by that patient’s insurance and what the cost will be. In most industries, consumers make informed choices that include cost considerations. With prescription drugs, the doctor doesn’t know the cost to the patient, and the patient may discover the out-of-pocket cost only upon checking out at the pharmacy. We propose, however, a simple solution: a standardized pricing guide, akin to the nutritional information panel on most food products, that generates definite information about the costs for a given drug to a given patient with a given insurance plan

Purchasing Pharmaceuticals (Health, Nutrition and Population (HNP) Discussion Paper)

This paper discusses the purchasing of pharmaceuticals as a key component of costeffective and equitable healthcare delivery. Pharmaceuticals account for a high, sometimes the dominant share of health expenditures in developing countries, but the desired health outcomes can only be achieved if the adequate medicines reach the right people and are used in the correct way. This requires purchasing arrangements that take into account the information asymmetry between patients and providers, ensure selection of effective, safe and affordable medicines and set economic incentives in a way that encourages rational drug use. The organizational and institutional frameworks define the roles of the various public and private stakeholders and establish the rules and regulations for registration, import, prescription and distribution of pharmaceuticals within which active purchasing can take place. While there is a trend towards more active purchasing arrangements for pharmaceuticals, the move from passive to active purchasing, using up-to-date information systems to link inputs and outcomes, and pooled purchasing arrangements to optimize the use of limited resources, has been slow.https://digitalcommons.chapman.edu/pharmacy_books/1019/thumbnail.jp

Effect of Free Trade Agreements on Pharmaceutical Market Competition: The Case of the 2009 US-Peru Free Trade Agreement and Its Implementation as National Drug Policy

Free Trade Agreements (FTA) are controversial for threatening essential aspects of health, especially access to affordable medicines. The US-Peru FTA required changes in the Peruvian pharmaceutical legislation that resulted in the implementation of the National Drug Policy (NDP) of 2009. The NDP included more robust technical requirements for registration, a Peruvian Good Manufacturing Practices certificate, a longer timeline for drug registration, and an increase in registration fees. This study evaluated the impact of the FTA on the number of registrations and competition in the Peruvian pharmaceutical market. Data for the period January 2005 to April 2014 were provided by the Peruvian drug regulatory authority (Dirección General de Medicamentos, Insumos y Drogas, DIGEMID). A total of 31,114 pharmaceutical products with unique registration numbers were evaluated. Brand drug new registrations decreased from 1789 in 2005 to 455 in 2013, and the number of generic registrations decreased from 621 in 2005 to 114 in 2013. Brand re-registrations also decreased from 714 in 2005 to 58 in 2013. There were 228 brand products awaiting registration in 2009 and 1,908 in 2013. The proportion of products awaiting registration was three times greater for brand than for generic products in 2009–2013. Registration of brand and generic medicines significantly declined after the implementation of the US-Peru FTA in 2009. The decline in the number of registrations was associated with more robust technical requirements, a longer DIGEMID registration timeline, and an increase in registration fees. The stronger registration requirements are expected to increase the quality of the drugs marketed in the country, but also less competition and a reduction in domestic registrations

Innovation and Competition in Advanced Therapy Medicinal Products

Advanced therapy medicinal products (ATMPs), including gene therapy, cell therapy, and tissue engineering products, represent a paradigm shift in health care as they have great potential for preventing and treating many diseases (Food and Drug Administration (FDA), 2013). By way of example, only 367 (8.0%) of the 4,603 rare diseases and conditions listed by the NIH Genetic and Rare Diseases Information Center had at least one FDA-approved drug therapy in early 2018. An estimated 3,038 (66.0%) of those rare diseases and conditions are congenital and genetic diseases that could potentially be treated by gene therapy. There are already ATMPs under development to address these and many other unmet medical needs (FDA, 2013; MIT NEWDIGS FoCUS Project, 2017) and for the treatment of prevalent conditions, such as cardiovascular, neurologic, and metabolic diseases

Predictors of Shortages of Opioid Analgesics in the US: Are the Characteristics of the Drug Company the Missing Puzzle Piece?

Background Shortages of opioid analgesics are increasingly common, interfere with patient care and increase healthcare cost. This study characterized the incidence of shortages of opioid analgesics in the period 2015–2019 and evaluated potential predictors to forecast the risk of shortages. Methods This was an observational retrospective study using the US Food and Drug Administration (FDA) drug shortages data. All FDA approved opioids were included in the study. Opioid analgesics were identified using the FDA National Drug Codes (NDC) and classified according to the Drug Enforcement Administration (DEA) schedule. We conducted Least Absolute Shrinkage and Selection Operator logistic regression analysis to assess direction of the association between risk of shortage and potential predictors. We used multivariable penalized logistic regression analysis to model predictors of shortages. We split the dataset into training and validation sets to evaluate the performance of the model. Findings The FDA approved 8,207 unique NDCs for opioid analgesics; 3,017 (36.8%) were in the market as of April 30, 2019 and 91(3.0%) of them were listed as in shortage by the FDA. All NDCs in shortage were schedule II opioids; 86 (94.5%) were injectable and 84 (92.3%) generics. There were 418 companies with at least one opioid NDC listed by the FDA. Three companies accounted for more than 4 in 5 of the schedule II active injectable opioids. For each unit increase in the number of prior instances of shortages of a company, the likelihood of an NDC shortage for that company increased by 3.4%. For each unit increase in number of NDCs marketed by a company, the odds of an NDC shortage for that company decreased by 1%. Conclusions In the period 2015–2019, shortages of opioid analgesics disproportionally impacted schedule II and injectable opioids. The risk of shortage of opioid analgesics significantly increased with the incidence of previous instances of shortages of a manufacturing company and decreased with the number of NDCs marketed by a company. The characteristics of the manufacturing company, rather than the number of companies, might be the missing piece to the complex puzzle of drug shortages in the US

Ethical Imperatives of Timely Access to Orphan Drugs: Is Possible to Reconcile Economic Incentives and Patients’ Health Needs?

Background More than 6,800 rare diseases and conditions have been identified in the US, which affect 25–30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of drugs to treat rare diseases and conditions. This study analyzed all orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to orphan drugs. Methods Study data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of Orphan Drugs Development. A search was conducted to assess literature on the ethical principles and economic incentives for the development of orphan drugs. Results In the period 1983–2015, the FDA granted 3,647 orphan drug designations and 554 orphan drug approvals. The orphan drug approvals corresponded to 438 different brand names. Cancer was the therapeutic area with the highest number of approvals. The increased number of patients with rare diseases and the growth in the cost of orphan drugs pose a significant economic burden for patients, public programs and private third party payers. Regulatory differences to qualify for orphan designation and various population thresholds employed by the FDA and the European Medicines Agency lead to further unmet health needs for patients with rare diseases and aggravate health inequities. There is no societal consensus on the population and economic thresholds, the drug effectiveness indicator(s), or the societal value to be placed for the approval and reimbursement of orphan drugs. Conclusion Orphan drug development and marketing in the US concentrate in few therapeutic areas. Despite the increase in the number of FDA approved orphan drugs, the unmet needs of patients with rare diseases evidence that the current incentives are not efficiently stimulating orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing patients’ access to treatment. Thus, aligning pharmaceutical companies’ incentives with societal budgetary constraints is necessary and the ethical imperatives of timely access to orphan drugs need to be agreed upon

Provision of Health Care Services in Canada: Challenges and Opportunities

The Canadian health care system provides comprehensive coverage of hospital and outpatient care, including therapeutic, diagnostic and preventive services. The level of coverage of services varies across the country. This study examines the key characteristics of the Canadian health and long-term care systems; presents a structured analysis of the insurance, financing and provision of health and long-term care services in Canada; describes the main challenges of the Canadian health and long-term care systems; and concludes with feasible opportunities for the Canadian health policy. Main challenges to the Canadian system are related to population ageing; prevalence of avoidable diseases caused by poor health habits; coverage and financing of long-term care services; financing of expensive new technologies and pharmaceuticals; and the shortage and unbalanced geographic distribution of health care professionals. Opportunities for the Canadian health policy are: strengthening public health policy, continuing shifting care to the ambulatory level; improving the coordination between primary care and specialist services; implementing a system-wide national human resources planning; and integrating home-based care as part of overall primary health care