The impact of proposed price regulations on new patented medicine launches in Canada: a retrospective cohort study.

BACKGROUND: The Patented Medicine Prices Review Board (PMPRB), the agency that regulates the prices of patented medicines in Canada, published proposed amendments to the regulatory framework in December 2017. Because of a series of changes and delays, the revised policy has not yet been finalized. We sought to evaluate the potential early impact of the uncertainty about the PMPRB policy on patented-medicine launches. METHODS: We developed a retrospective cohort of patented medicines (molecules) sold in Canada and the 13 countries that the PMPRB currently uses or has proposed to use as price comparators, from sales data from the IQVIA MIDAS database for 2012-2021. The outcome was whether a molecule was launched (i.e., sold) in a specific country within 2 years of its global first launch (2-yr launch). We compared the change of 2-year launch before (2012-2017) and after the proposed amendments were published ("uncertain period," 2018-2021) in Canada with the change in the United States and the other 12 countries as a group ("other-countries group"), using interrupted time series and logistic regressions, respectively. We further conducted analyses for each individual country and subgroups by molecule characteristics, such as therapeutic benefit, separately. RESULTS: We included 242 and 107 new molecules launched before publication of the proposed amendments and during the uncertain period, respectively. The corresponding 2-year launch proportions were 45.0% and 30.8% in Canada, 81.4% and 82.2% in the US, and 83.9% and 70.1% in the other-countries group. All analyses showed changes in 2-year launch during the uncertain period in the US and in the other-countries group that were similar to the changes in Canada. Greater decreases were observed in Norway and Sweden than in Canada. The 2-year launch proportion for molecules with major therapeutic benefit decreased from 45.8% to 31.3% in Canada during the uncertain period and from 87.5% to 62.5% in the other-countries group, but increased from 91.7% to 100% in the US. INTERPRETATION: No negative impact of the PMPRB-policy uncertainty on molecule launches was observed when comparing Canada with price-comparator countries, except for molecules with major therapeutic benefit. The reduction in launches of medicines with major therapeutic benefit in Canada requires continuing investigation.

Role of Specialty Drugs in Rising Drug Prices for Medicare Part D.

Importance: Prices for brand-name drugs affect both federal spending and out-of-pocket liability for Medicare Part D enrollees. Objective: To examine how prices for brand-name drugs, net of rebates and discounts, have changed from 2010 to 2019 and to examine the role of specialty drugs in those changes. Design, Setting, and Participants: This study involved a descriptive analysis of prescription drug spending and prices between 2010 and 2019. The universe of prescription drug event data from those years were combined with confidential data from the Centers for Medicare & Medicaid Services on rebates and discounts that manufacturers and pharmacies pay to Medicare Part D plans to calculate rebate percentages, net spending, and net prices at the drug level. Specialty drugs were identified using information from IQVIA, allowing for a stratified analysis by specialty status. Data were analyzed from March 2019 to March 2024. Main Outcomes and Measures: Average prices (net of rebates and discounts in 2019 US dollars) and average annual price growth for brand-name prescription drugs, overall and separately for specialty and nonspecialty drugs. Results: Average net prices for brand-name drugs doubled from 2010 to 2019 (from $167 to $370). Growth in specialty drug prices was an underlying factor in those increases: average annual price growth was 13.2% for specialty drugs compared with 2.6% for nonspecialty drugs. Price growth for specialty drugs over the decade was smaller than what the Congressional Budget Office reported for the 2010 to 2015 period (increase of 22.3% per year vs 4.5% per year for nonspecialty drug prices), suggesting that price growth slowed after 2015. Drugs that treat hepatitis C contributed to that difference because prices for those drugs were initially high and then subsequently fell. Absent those drugs, price growth for specialty drugs averaged 18.1% in the first half of the decade and 6.9% in the second half. Conclusions and Relevance: Results of this study show that prices for specialty drugs have continued to increase over time in the Medicare Part D program, which contributes to high out-of-pocket liability for users of those drugs in addition to US federal budgetary expenditures.

The Inflation Reduction Act and Access to High-Cost Cardiovascular Therapies.

This Viewpoint discusses how the price negotiation for certain drugs under the Inflation Reduction Act will provide a unique opportunity to enhance access to therapies for older patients with cardiovascular conditions and diabetes.

Medicare's Historic Prescription Drug Price Negotiations.

This Viewpoint evaluates the legal claims and policy implications of historic drug price negotiations possible with the Inflation Reduction Act of 2022.

340B-Where Do We Go From Here?

This Viewpoint summarizes inefficiencies in the 340B program and provides suggestions for equitable reform that will potentially benefit patients.

Added Therapeutic Benefit of Top-Selling Brand-name Drugs in Medicare.

Importance: The Inflation Reduction Act of 2022 authorizes Medicare to negotiate prices of top-selling drugs based on several factors, including therapeutic benefit compared with existing treatment options. Objective: To determine the added therapeutic benefit of the 50 top-selling brand-name drugs in Medicare in 2020, as assessed by health technology assessment (HTA) organizations in Canada, France, and Germany. Design, Setting, and Participants: In this cross-sectional study, publicly available therapeutic benefit ratings, US Food and Drug Administration documents, and the Medicare Part B and Part D prescription drug spending dashboards were used to determine the 50 top-selling single-source drugs used in Medicare in 2020 and to assess their added therapeutic benefit ratings through 2021. Main Outcomes and Measures: Ratings from HTA bodies in Canada, France, and Germany were categorized as high (moderate or greater) or low (minor or no) added benefit. Each drug was rated based on its most favorable rating across countries, indications, subpopulations, and dosage forms. We compared the use and prerebate and postrebate (ie, net) Medicare spending between drugs with high vs low added benefit. Results: Forty-nine drugs (98%) received an HTA rating by at least 1 country; 22 of 36 drugs (61%) received a low added benefit rating in Canada, 34 of 47 in France (72%), and 17 of 29 in Germany (59%). Across countries, 27 drugs (55%) had a low added therapeutic rating, accounting for $19.3 billion in annual estimated net spending, or 35% of Medicare net spending on the 50 top-selling single-source drugs and 11% of total Medicare net prescription drug spending in 2020. Compared with those with high added benefit, drugs with a low added therapeutic rating were used by more Medicare beneficiaries (median 387 149 vs 44 869) and had lower net spending per beneficiary (median $992 vs $32 287). Conclusions and Relevance: Many top-selling Medicare drugs received low added benefit ratings by the national HTA organizations of Canada, France, and Germany. When negotiating prices for these drugs, Medicare should ensure they are not priced higher than reasonable therapeutic alternatives.

Pursuing Value-Based Prices for Drugs: A Comprehensive Comparison of State Prescription Drug-Pricing Boards.

Policy Points In the absence of federal action on rising prescription drug costs, we reviewed the details of five states that have enacted prescription drug-pricing boards seeking to lower drug prices based on products' value. Within these states, six such boards are currently authorized; they have similarities but vary in terms of structure, authority, scope, and leverage. As of June 2021, only one of the boards in our sample has conducted pricing reviews; legislators in other states can learn from the successes and challenges of existing boards. Prescription drug-pricing boards represent a novel and promising way to curb state spending and pay for value in prescription drugs but face legal and political barriers in implementation. CONTEXT: Rising prescription drug costs are consuming a growing proportion of state and private budgets. In response, lawmakers have experimented with a variety of policies to contain spending and achieve value in prescription drugs. As part of this series of reforms, some state legislatures have recently authorized prescription drug-pricing boards to address the high prices of brand-name prescription drugs and assess the value of those drugs. METHODS: We identified state prescription drug-pricing boards in the United States, defined as any agency authorized by a state legislature to review specific drugs and pursue value-based drug prices. To describe the characteristics of the boards, we obtained public records of authorizing legislation, guidance documents, and board meeting minutes. We compared the boards' powers and responsibilities and analyzed completed pricing reviews. FINDINGS: Six state drug-pricing boards in five states met our definition; their design varied substantially. Two of the boards (New York Medicaid and Massachusetts) have authority over drug rebates paid by state Medicaid programs, one (New York Drug Accountability Board) has jurisdiction over state-regulated commercial insurance, and another three (Maine, Maryland, and New Hampshire) oversee non-Medicaid, state-funded insurance. Three boards are authorized to require manufacturers to confidentially submit information related to the pricing and clinical effectiveness of reviewed drugs to inform value determinations. Only one board (New York Medicaid) had completed pricing reviews as of June 3, 2021. CONCLUSIONS: Boards' structure, scope, and statutory leverages to compel manufacturers to negotiate lower net costs are key factors that influence whether and to what extent boards can achieve cost savings for states. Though legal constraints may limit the effective reach of prescription drug-pricing boards, these agencies can enable states to address rising prescription drug costs, in part by virtue of their very existence. To overcome practical limitations, states seeking to implement similar policies can build on the experiences and designs of current boards.