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To control pharmaceutical spending and improve access, the United States could adopt strategies similar to those introduced in Germany by the 2011 German Pharmaceutical Market Reorganization Act. In Germany, manufacturers sell new drugs immediately upon receiving marketing approval. During the first year, the German Federal Joint Committee assesses new drugs to determine their added medical benefit. It assigns them a score indicating their added benefit. New drugs comparable to drugs in a reference price group are assigned to that group and receive the same reimbursement, unless they are therapeutically superior. The National Association of Statutory Health Insurance Funds then negotiates with manufacturers the maximum reimbursement starting the 13th month, consistent with the drug's added benefit assessment and price caps in other European countries. In the absence of agreement, an arbitration board sets the price. Manufacturers accept the price resolution or exit the market. Thereafter, prices generally are not increased, even for inflation. US public and private insurers control prices in diverse ways, but typically obtain discounts by designating certain drugs as preferred and by restricting patient access or charging high copayment for nonpreferred drugs. This article draws 10 lessons for drug pricing reform in US federal programs and private insurance. [ABSTRACT FROM AUTHOR]

Aim: Regulatory and health technology assessment (HTA) agencies have increasingly published frameworks, guidelines, and recommendations for the use of real-world evidence (RWE) in healthcare decision-making. Variations in the scope and content of these documents, with updates running in parallel, may create challenges for their implementation especially during the market authorization and reimbursement phases of a medicine's life cycle. This environmental scan aimed to comprehensively identify and summarize the guidance documents for RWE developed by most well-established regulatory and reimbursement agencies, as well as other organizations focused on healthcare decision-making, and present their similarities and differences.  Methods: RWE guidance documents, including white papers from regulatory and HTA agencies, were reviewed in March 2024. Data on scope and recommendations from each body were extracted by two reviewers and similarities and differences were summarized across four topics: study planning, choosing fit-for-purpose data, study conduct, and reporting. Post-authorization or non-pharmacological guidance was excluded. Results: Forty-six documents were identified across multiple agencies; US FDA produced the most RWE-related guidance. All agencies addressed specific and often similar methodological issues related to study design, data fitness-for-purpose, reliability, and reproducibility, although inconsistency in terminologies on these topics was noted. Two HTA bodies (National Institute for Health and Care Excellence [NICE] and Canada's Drug Agency) each centralized all related RWE guidance under a unified framework. RWE quality tools and checklists were not consistently named and some differences in preferences were noted. European Medicines Agency, NICE, Haute Autorité de Santé, and the Institute for Quality and Efficiency in Health Care included specific recommendations on the use of analytical approaches to address RWE complexities and increase trust in its findings. Conclusion: Similarities in agencies' expectations on RWE studies design, quality elements, and reporting will facilitate evidence generation strategy and activities for manufacturers facing multiple, including global, regulatory and reimbursement submissions and re-submissions. A strong preference by decision-making bodies for local real-world data generation may hinder opportunities for data sharing and outputs from international federated data networks. Closer collaboration between decision-making agencies towards a harmonized RWE roadmap, which can be centrally preserved in a living mode, will provide manufacturers and researchers clarity on minimum acceptance requirements and expectations, especially as novel methodologies for RWE generation are rapidly emerging.

As U.S. policymakers consider strategies to control pharmaceutical spending, they can learn from France, which has stopped drug spending growth without slowing access to innovative medicines. France determines the comparative therapeutic value of new drugs. Insurance pays more for drugs superior to their comparator and the same or less for drugs offering modest or no improvement. Contracts require discounts for high sales volume and prohibit price increases. In addition, payers reduce prices of older drugs. Furthermore, Parliament sets an insurance pharmaceutical spending budget, and manufacturers pay clawbacks when spending exceeds the budget. France offers these lessons: setting prices based on added therapeutic value is a principled means to cap new drug prices and provides incentives for manufacturers to negotiate prices. Restricting formularies can help lower prices. Insurers can link prices and quantity to control spending and improper uses. Insurers can use global budgets to control spending and negotiate prices. Contracts can prevent manufacturers from raising prices after launch. External reference pricing can reduce price discrimination but is difficult to implement. Nations can ensure rapid access to new drugs while controlling prices. Regulation and competition are complementary strategies to control drug spending. [ABSTRACT FROM AUTHOR]

In this latest update we highlight recent publications from the US FDA and a Canadian Health Technology Assessment collaboration which provide insights into the future role of real-world evidence in market access in terms of health technology management.

The article presents information on the history of health technology assessment (HTA). HTA started in the U.S. with the establishment of the U.S. Office of Technology Assessment (OTA) in 1972, which was disbanded in 1995. In 1999, Great Britain established the National Institute for Clinical Excellence (NICE), which reportedly contributed to the globalization of HTA. The article also presents a comparison between HTA and evidence-based medicine (EBM).

The article presents information on health technology assessment (HTA) in the U.S. It is mentioned that the U.S. has a long history regarding the funding for health technology assessment programs, but many of these early U.S. initiatives were later discontinued. The U.S. Office of Technology Assessment (OTA) was established in 1972 to undertake health technology assessments. It is stated that the Medicare and Medicaid programs are the largest purchasers of health care in the U.S.

The article presents information on health technology assessment (HTA) organizations in various countries. It is stated that health technology assessment regarding health policy decisions has received a more favorable response in Europe than in the U.S.; and HTA has become a political priority in Europe. It is stated that compared to Europe, the U.S. has a more privately oriented and decentralized health-care system.