Cancer Drug Prices Research Articles

What constitutes meaningful benefit of cancer drugs in the context of LMICs? A mixed-methods study of oncologists’ perceptions on endpoints, benefit, price, and value of cancer drugs

The importance of surrogate endpoints, magnitude of clinical benefit of cancer drugs, and their prices have often been debated in the oncology world. No study, however, has systemically explored oncologists' perception regarding these issues. We conducted a mixed-methods study including in-depth qualitative interviews of medical oncologists prescribing cancer drug therapy in India. Quantitative data were collected using a predetermined proforma. Qualitative in-depth interviews were audio-recorded, transcribed verbatim, anonymized, subsequently coded, and analyzed by generating basic and global themes. We interviewed 25 medical oncologists. Twenty-eight percent of oncologists rarely used cancer drugs that improved response rate (RR) but not overall survival (OS), and an equal percentage mostly/often used such drugs. For cancer drugs that improved progression-free survival (PFS) but not OS, 20% never/rarely used them while 48% mostly/often used them. Oncologists in India considered a 4.5-month (range, 1.5-12 months) advantage in median PFS as meaningful, and considered price of ∼120 United States Dollars (USD) per month (range, 48-720 USD per month) for those PFS gains as justified. For OS, median gains of 4.5 months (range, 2-24 months) and at a monthly price of ∼360 USD (range, 48-900 USD) was considered justified. Oncologists in India were aware and concerned that RR only meant tumour shrinkage not survival benefit, but many assumed that tumour shrinkage meant better quality of life. Many oncologists acknowledged the limitations of PFS but would use a drug with PFS benefit if it was cheaper than the drug with OS benefit. Oncologists in India showed awareness of the limited surrogacy between RR/PFS and OS but assumed that RR/PFS correlated with improved quality of life and acknowledged price as a factor in deciding treatment choices. This is the first study providing a benchmark for minimum clinical benefit (4.5 months in PFS or OS) and maximum monthly price (120 USD for PFS, 360 USD for OS) deemed justifiable by oncologists practicing in low-and-middle-income countries.

Clinical Benefit, Price, and Regulatory Approval of Cancer Drugs Granted Breakthrough Therapy Designation in China, 2020-2024

The China National Drug Administration (NMPA) established the breakthrough therapy designation (BTD) in 2020 to encourage the accelerated development of drugs for the prevention and treatment of diseases that are life-threatening. However, the differences between BTD and non-BTD cancer drugs regarding clinical benefit, regulatory approval, and price are unclear. To compare BTD and non-BTD cancer drugs in clinical benefit (defined as efficacy and safety), novelty, time to approval, and average monthly treatment price. This cross-sectional study analyzes the original indication of BTD and non-BTD novel cancer drugs approved by the NMPA between July 8, 2020, and July 8, 2024. Data on efficacy, safety, regulatory approval, and price of cancer drugs were extracted from pivotal clinical trials based on review reports published by the NMPA, peer-reviewed articles or meeting reports, and winning bid prices for cancer drugs in the Chinese provincial-level centralized procurement process. The main outcome was the efficacy and safety associated with BTD vs non-BTD cancer drugs, including progression-free survival (PFS), response rate (RR), duration of response, serious adverse events, grade 3 or higher adverse events, and treatment-related deaths. In addition, the time to approval, novelty, and initial and latest average monthly treatment prices were evaluated, as well as the average annual reduction rate (AARR; the sum of the reduction rates divided by the number of years for the monthly treatment price) for these cancer drugs. Between July 2020 and July 2024, 18 BTD (36%) and 32 non-BTD (64%) cancer drugs were approved by the NMPA. The median (IQR) clinical development time for BTD drugs was significantly shorter than for non-BTD drugs (5.6 [95% CI, 4.3-7.3] vs 6.6 [95% CI, 6.0-8.5] years; P = .02). No significant differences were observed in PFS (HR, 0.44 [95% CI, 0.38-0.52] vs HR, 0.51 [95% CI, 0.40-0.65]; P = .20), PFS gained (median [IQR], 5.4 [3.9-7.0] vs 2.7 [2.6-5.9] months; P = .77), RR (58% [95% CI, 45%-74%] vs 59% [95% CI, 51%-69%]; P = .85), and duration of response (median [IQR], 18.0 [15.0-21.6] vs 11.1 [7.4-17.4] months; P = .09) between BTD and non-BTD drugs. The rates of serious adverse events (37% [95% CI, 26%-52%] vs 32% [95% CI, 27%-36%]; P = .45), adverse events grade 3 or higher (64% [95% CI, 53%-77%] vs 55% [95% CI, 45%-68%]; P = .31), and treatment-related deaths (2% [95% CI, 1%-4%] vs 1% [95% CI, 1%-2%]; P = .10) were similar between BTD and non-BTD drugs. BTD drugs are more likely to be first-in-class drugs (5 of 18 [28%] vs 1 of 32 [3%]; P = .02). Differences in the median (IQR) initial ($5665 [$3542-$9321] vs $3361 [$2604-$5474]; P = .06) and latest ($5665 [$1553-$9321] vs $2145 [$1318-$4276]; P = .18) average monthly treatment prices for BTD drugs and non-BTD drugs were not significant. The median (IQR) AARRs for BTD drugs and non-BTD drugs were 15.2% (0%-46.9%) and 19.8% (1.0%-42.9%), respectively. The findings of this cross-sectional study suggest that BTD has facilitated faster time to market for cancer drugs and improved novelty, but the price of treatment is relatively higher. There was no significant difference on comparative efficacy and safety.

Breast cancer drugs: FDA approval, development time, efficacy, clinical benefits, innovation, trials, endpoints, quality of life, value, and price

ObjectiveThis study analyzes the development, benefits, trial evidence, and price of new breast cancer drugs with US Food and Drug Administration (FDA) approval.MethodsWe identified 26 drugs with 42 FDA-approved indications for early and metastatic breast cancer (2000–2023). Data were collected from FDA labels, clinicaltrials.gov, and Medicare and Medicaid. Overall survival (OS) and progression-free survival (PFS) hazard ratios (HRs) and tumor response’s relative risk (RR) alongside objective response rate (ORR) were meta-analyzed.ResultsThe median development time for breast cancer drugs was 7.8 years (95% CI 6.2–10.8). 26% of treatments were considered innovative (“first-in-indication”) with 88% acting via a targeted mechanism. 64% were small molecules, 19% antibodies, and 18% antibody-drug conjugates. 38% were approved for HR + and 31% for HER2 + breast cancer. 6 indications were for early and 36 for metastatic breast cancer. Indications utilized FDA’s special programs: orphan (2%), fast track (24%), accelerated approval (19%), priority review (74%), breakthrough therapy (44%). Approval was predominantly supported by phase 3 trials (88%) of randomized controlled design (66%), enrolling a median of 585 patients (IQR 417–752) at 181 centers (IQR 142–223) across 19 countries (IQR 17–20). New drugs’ HR were 0.78 for OS (95% CI 0.74–0.82) and 0.59 for PFS (95% CI 0.54–0.64) with a RR for tumor response of 1.61 (95% CI 1.46–1.76). Median improvements of OS were 2.8 months (IQR 1.8–5.8) and PFS were 4.4 months (IQR 2.2–7.1). In single-arm trials, the average ORR was 31% (95% CI 10–53). In meta-regressions, the correlation between OS/PFS was 0.34 (p = 0.031) and OS/response was 0.01 (p = 0.435). 60% of treatments had a ‘high-value’ ESMO-MCBS score with 14% demonstrating improvements in quality of life. The median price was $16,013 per month (95% CI 13,097–17,617). There was no association between prices and patient benefit. The median value per life year gained was $62,419 (IQR 25,840–86,062).ConclusionsOver the past two decades, the development of innovative and effective drugs transformed the treatment landscape for breast cancer patients. Yet, investigators and regulators must safeguard that highly-priced new drugs demonstrate improvements in patient-centered clinical endpoints: overall survival and quality of life.

Clinical benefit, reimbursement outcomes, and prices of FDA-approved cancer drugs reviewed through Project Orbis in the USA, Canada, England, and Scotland: a retrospective, comparative analysis

Project Orbis is a global initiative that aims to streamline regulatory review processes across international regulators in the USA, Canada, Australia, UK, Israel, Brazil, Singapore, and Switzerland to bring promising cancer drugs to patients earlier. We explored the clinical benefit, time to regulatory approval and health technology assessment recommendations, reimbursement outcomes, and monthly treatment prices of cancer drugs reviewed through this initiative. For this retrospective, comparative analysis, we identified cancer drug approvals reviewed through Project Orbis in the USA, Canada, and the UK between May 1, 2019, and Nov 1, 2023. Approvals of cancer drugs reviewed Project Orbis were extracted from the Food and Drug Administration (FDA) Oncology Centre of Excellence and all other FDA approvals from the Drugs@FDA database. The co-primary outcomes were time of regulatory review, time from regulatory approval to health technology assessment recommendation (England, Scotland, and Canada), reimbursement outcomes, clinical benefit (defined as median gains in progression-free survival and overall survival) between cancer drug approvals reviewed by Project Orbis and other FDA approval processes, and monthly treatment prices. The Wilcoxon rank-sum and Fisher's Exact tests were used to examine statistical significance between approvals reviewed through Project Orbis and other FDA approvals during the same period. Between May 1, 2019 and Nov 1, 2023, 81 (33%) of 244 cancer drugs approved by the FDA were reviewed through Project Orbis. The median overall survival gains were 4·1 months (IQR 3·3-5·1) compared with 2·7 months (2·1-3·9) for other FDA approvals. Similarly, progression-free survival gains were 2·6 months (IQR 1·7-4·9) for Project Orbis compared with 2·6 months (0·6-5·1) for other FDA approvals. Neither overall survival (p=0·11) nor progression-free survival (p=0·44) gains were significantly different between the two cohorts of approvals. Of the 14 UK Medicines and Healthcare products Regulatory Agency (MHRA) approvals reviewed by the Scottish Medicines Consortium (SMC), the agency gave positive recommendations for all 14 (100%). Of the 15 MHRA approvals reviewed by the National Institute for Health and Care Excellence (NICE), the agency gave positive recommendations for six (40%). Of the 49 approvals reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH), the agency conditionally recommended 44 (90%). The time between regulatory approval to NICE recommendation increased from a median of 137 days (IQR 102-172) in 2021 to 302 days (184-483) in 2023, SMC recommendation increased from 185 days (in 2021 for one drug only) to 368 days (IQR 313-476) in 2023, and CADTH decision increased from 97 days (in 2020 for one drug only) to 202 days (IQR 153-304) in 2023. The median monthly price of approvals reviewed through Project Orbis was US$20 000 per month (IQR 13 000-37 000). Clinical outcomes of Project Orbis were no different than other FDA approvals during the same time, and access, after a successful health technology assessment, was considerably delayed or absent, raising questions about whether Project Orbis participation translates into faster patient access to medicines with high clinical benefit and sustainable costs. Although future challenges might benefit from regulatory harmonisation, the advantages are currently unclear. None.

Factors influencing the clinical added value (CAV) in France over more than a decade.

e23102 Background: The rise of cancer drug prices has raised alarm over the need to base pricing decisions on the clinical added value (CAV). The French National Authority for Health (HAS) is responsible for health technology assessment (HTA) in France. For each indication retained for reimbursement, the HAS assesses the CAV on a 5-point scale ranging from I (Major) to V (absence of clinical added value). This assessment is primarily based on medical evidence. The CAV score is one of the factors contributing to price negotiations between the French ministry and pharmaceutical companies. Methods: A retrospective and descriptive study analysis of all indications in oncology (hematology and solid tumors), excluding accelerated approvals for innovative treatments, was conducted by the HAS comparing the 2010-2015 and 2016-2022 periods. For each assessment, information relative to the level of evidence and the clinical effect of the drug from the HAS opinion were collected and focused on the study’s conclusive endpoints and design. Results: The global distribution of CAV score between the two studied periods (2010-2015 and 2016-2022) was similar. However, the distribution of CAV score based on study design differed between the two periods. Regarding non comparative studies, the proportion of indications that were attributed a CAV score V increased 1.7 times between 2010-2015 and 2016-2022 and those with a CAV score I-III was nearly divided by 3. Differences were equally observed for the distribution of CAV score between the two periods based on the conclusive endpoints in the pivotal study. For the studies with a conclusive endpoint on overall survival, the rate of CAV I-III was multiplied by 1.68 (an increase from 6 to 36 cases). Concerning the cases with a progression-free survival endpoint, an observed increase of 1.3 times was noted for the CAV I-III. Lastly, studies with a conclusive overall response rate (ORR) saw an increase of 1.4 times for the CAV V as well as a decrease of 0.76 times for the CAV I-III. Over the most recent period (2016-2022), the conclusive endpoints identified in the evaluated studies were predominantly the overall response rate (93%, n = 42/45) for phase II studies. For phase III studies, progression-free survival and overall survival were respectively identified in 45% and 30% of the cases. Conclusions: Thisstudy shows that the number of indications evaluated has increased and that the assessment of the CAV is closely correlated with the choices of the study design (comparative or not) and the primary endpoint (clinically relevant or not). However, other elements underpin and qualify its valuation such as the medical need. These elements are reviewed in the HAS Guidance which emphasises overall survival as a primary endpoint and comparative data.

Cancer Drug Price and Novelty in Mechanism of Action

Many economic theories point to regulatory issues and subsidization of research and development costs as the primary factor in the high cancer drug prices in the US. Even so, the association between the median annual cost and novelty of cancer drugs approved in the US remains unclear. To evaluate the association between the median annual cost and novelty of cancer drugs approved in the US over a 6-year period. This cross-sectional study included all cancer drugs approved by the US Food and Drug Administration (FDA) from January 1, 2015, to December 31, 2020. Drug names, indications, manufacturer, dosage, and measures of activity/efficacy were extracted from the FDA announcement. The search was performed in December 2021. Data were analyzed from January 2022 until April 2022. Annual cost of treatment was calculated based on average wholesale price collected from the 2021 Micromedex Red Book database. Mechanism of action was inferred from trial publication or its references. There were 224 cancer drug approvals across 119 individual drugs, with a median annual cost of $196 000 (IQR, $170 000-$277 000). Gene and viral therapies were the most expensive (median, $448 000 [IQR, $448 000-$479 000]), followed by small molecule therapy (median, $244 000 [IQR, $203 000-$321 000), and biologics (median, $185 000 [IQR, $148 000-$195 000]). There was no significant difference in cost between first-in-class, next-in-class, and subsequent approvals of an already approved drug. Findings of this study indicate that the median annual price of anticancer drugs in the US is not associated with the novelty of their mechanism of action.

Why Does the Inflation Reduction Act Exclude Expensive Cancer Treatments in Price Negotiations?

The Inflation Reduction Act (IRA) includes provisions for price negotiations of certain high-spending drugs in Medicare Parts B and D. This provision received considerable attention from those interested in the costs of cancer care since Medicare covers most patients with cancer and many cancer drugs are expensive. We simulate how many cancer drugs may be eligible for IRA price negotiations and examine the reasons that many are likely to be excluded from negotiation. This study uses 2021 Medicare Fee-for-Service Part B and Part D prescription drugs expenditure data. Cancer drugs were identified using the SEER Program list of cancer medications. Our measures included total spending, beneficiary users, and spending-per-beneficiary for all cancer drugs covered under Medicare. Each drug was evaluated for eligibility on the basis of IRA negotiation provisions, including estimated loss of patent exclusivity, current competitors, and orphan drug designation. We found that very few cancer drugs will meet the IRA eligibility thresholds to be included in negotiations. We estimate that only 2.2% of beneficiaries with cancer will see lower costs because of the IRA negotiations. The main reason for this is that although novel cancer drug treatments are priced high, they generally treat relatively few beneficiaries and thus do not meet negotiation eligibility criteria, which are primarily based on a ranking of total spending. The IRA negotiation provisions will have limited impact on cancer drug prices and will likely leave most patients with cancer exposed to high drug costs.

Quantifying Challenges to Development of Generics and Biosimilars in Non-Hodgkin's Lymphoma Therapeutics: The Future of Low-Cost Alternatives from Monoclonal Antibodies to CAR-T

Background: There are numerous monotherapies and combination therapies available to treat Non-Hodgkin lymphoma's (NHL), however relatively few of these varied antineoplastics agents have commercially available, lower-cost generic forms in the United States. High cancer drug prices, in particular for socioeconomically vulnerable and underinsured patients, pose challenges to affordable cancer treatment in the United States. In 2017, FDA initiated the Drug Competition Action Plan (DCAP) that aimed to ensure timely market competition by enabling generic drug development, review, and approval, and streamlining regulatory complexities. This regulatory program was designed to limit so-called “gaming” recognized as a cause of delay to generic drug approvals, a phenomenon that has the further deleterious effect of extending brand monopolies beyond what Congress intended with Hatch-Waxman Amendments (1984) establishing modern generic drug regulatory processes. In 2018, FDA announced analogous Biosimilars Action Plan (BAP). However, to date the efficacy of these measures has not been comprehensively assessed in the NHL development landscape. Methods: A model was created by surveying the FDA Orange Book (drug approval database) and Purple Book (biologic product approval database) for initial approval of drugs/biologics with their initial generic/biosimilar approval. Approvals were classified by drug class and availability of generic/biosimilar for the innovator product with approval from 2002 to 2023 (Fig. 1) and t-test were used to determine significance. Results: Of small molecule drug NHL therapeutics approved in the last two decades, only 37% (19/52; Fig. 2) have available, lower-cost generics, with use in more than one indication and time on market both positively and significantly corresponding with availability of generics for the original innovator drug (P < 0.05). Notably, while generic drug approvals dropped during the 2020-2021 period (possibly due to impact of the COVID-19 pandemic), after 2017 implementation of DCAP more generics for common NHL antineoplastics were approved. Conversely, there are relatively few biologic drugs (total of 41 approvals; Fig. 2) with biosimilars, in part due to retained patents and exclusivity, and perhaps indicating a lack of efficacy in BAP among NHL antineoplastics therapies. For off-patent innovator monoclonal antibodies (mAbs), which make up the majority of these therapies, development of biosimilars is effectively blocked by secondary or “process-related” patents which effectively extend the period of non-competition for complex biologic therapies. It is expected that novel bispecific antibody (biAb) therapeutics and novel therapies, such as CD19 CAR-T will also have a delayed time to low-cost biosimilar availability following a similar trend. Conclusions: With drug price and ever-rising insurance and patient payments a critical area of public health, in particular in the United States, availability of lower cost generics and biosimilars is a critical issue for NHL patients. As we move into the maturation of gene therapies and CAR-T therapeutics, it merits consideration of that additional regulatory programs may be needed to incentivize the development of efficacious and lower cost biosimilars. However, it is critical that controlling competition be carefully balanced with investment in both biologic drug entity and process-related innovations among these relatively complex therapeutic entities.

Launch and Post-Launch Prices of Injectable Cancer Drugs in the US: Clinical Benefit, Innovation, Epidemiology, and Competition.

Rising cancer drug prices adversely affect patients' adherence and survival. We aimed to identify and quantify factors associated with launch prices and post-launch price changes of injectable cancer drugs in the US from 2005 to 2023. All anticancer drugs with US FDA approval between 2000 and 2022 were identified in the Drugs@FDA database. The sample was then restricted to cancer drugs covered under Medicare Part B (injectable drugs). Data characterizing each drug's clinical benefits, disease epidemiology, approved indications, competition, and price were obtained from FDA labels, the Global Burden of Disease study, and the Centers for Medicare and Medicaid Services. The association between launch/post-launch prices and collected variables was assessed in random-effects regressions. Of 170 cancer drugs with FDA approval between 2000 and 2022, we identified 66 (39%) injectable cancer drugs with quarterly price data from 2005 to 2023. In 2023, mean prices amounted to $27,688 per month, with an average price increase of 94% from 2005 to 2023. Launch and post-launch price changes were significantly associated with the treated disease epidemiology. A 1% decline in disease incidence was associated with a 0.2511% (p=0.008) increase in launch prices and a 0.0086% (p=0.032) annual increase in post-launch prices. Accordingly, launch prices were 120% (p=0.051) higher for orphan than non-orphan drugs, with 3% (p=0.008) greater annual post-launch price increases. Post-launch prices declined by up to -2% annually as new supplemental indications were approved for the same drug. We found no consistent association between launch/post-launch prices and the drugs' clinical benefit in terms of overall survival, progression-free survival, and tumor response. The market entry of new competitors was not associated with price reductions. 28 of 33 drug pairs within the same class had positive correlation coefficients. Pearson correlation coefficients were high (>0.80) for PD-1/PD-L1 inhibitors, CD38 antibodies, CD20 antibodies, HER2 antibodies, and mTOR inhibitors. Cancer drug prices regularly increase faster than inflation; however, there is no evidence that launch prices and post-launch price changes are aligned with the clinical benefit a drug offers to patients. In particular, patients with rare diseases experience greater price increases for their orphan drugs. There is no evidence that brand-brand competition results in drug price reductions.

Clinical benefit, development, innovation, trials, epidemiology, and price for cancer drugs and indications with multiple special FDA designations.

This study analyzes the development, US Food and Drug Administration (FDA) approval, benefits, innovation, trials, epidemiology, and price of cancer drugs with multiple special designations: orphan, fast track, accelerated approval, priority review, and breakthrough therapy. In total, 355 FDA-approved cancer drug indications with 841 special designations were identified (2012-2022). Trial, epidemiology, and price data were collected from FDA labels, the Global Burden of Disease study, and Medicare and Medicaid. The association between efficacy outcomes and indications' number of special designations were compared in meta-analyses. Median development times were 7.3, 7.8, and 5.4 months (P = .027) for drugs with 0 to 1, 2 to 3, and 4 to 5 special designations, respectively. Multiple special designations were associated with higher biotechnological and clinical innovation. Median patient enrollment in trials were 615, 471, 398, 168, 104, and 120 (P < .001) for indications with 0 to 5 special designations. Drugs for rare diseases supported by open-label phase 1/2 trials of single-arm design were granted more special designations. Hazard ratios for overall survival (0.80 vs 0.73 vs 0.73 vs 0.69 vs 0.56 vs 0.52; P = .003) and progression-free survival (0.70 vs 0.61 vs 0.59 vs 0.44 vs 0.37 vs 0.67; P < .001) substantially declined while tumor response increased with more special designations. Mean monthly prices increased for drugs with 0 to 4 but not 5 special designations ($21 596 vs $14 753 vs $32 410 vs $41 240 vs $38 703 vs $19 184). Multiple special designations are associated with faster clinical development and greater benefits for patients with unmet needs but also with nonrobust trial evidence and a tendency toward higher drug prices.

1701P Cancer premium: Explaining differences in prices for cancer vs non-cancer drugs with efficacy and epidemiological endpoints in the US, Germany, and Switzerland

1701P Cancer premium: Explaining differences in prices for cancer vs non-cancer drugs with efficacy and epidemiological endpoints in the US, Germany, and Switzerland

1707P Cancer drug prices in the US: Efficacy, innovation, clinical trial evidence, and epidemiology

1707P Cancer drug prices in the US: Efficacy, innovation, clinical trial evidence, and epidemiology

Designing a Digital Tool to Inform Oncology Practices About Cancer Treatment Cost Burdens.

Paper describes our development of a web-based tool to make Medicare patient prices for cancer drugs known to their care teams.

Cancer Drug Prices in the United States: Efficacy, Innovation, Clinical Trial Evidence, and Epidemiology

ObjectivesRising cancer drug prices challenge patients and healthcare systems. Although prices are routinely assigned to original drug indications receiving US Food and Drug Administration (FDA) approval, the pricing of supplemental indication approvals remains uncertain. This study identifies and quantifies factors associated with cancer drug prices, distinctly analyzing original and supplemental indications. MethodsClinical trial evidence and epidemiologic data supporting new indications’ FDA approval (2003-2022) were collected from the Drugs@FDA database, ClinicalTrials.gov, and Global Burden of Disease study. Indication-specific monthly treatment costs were calculated for Medicare patients. The association between log-prices and collected variables were assessed in regression analyses. ResultsWe identified 145 drugs approved across 373 cancer indications. Drugs were priced at $24 444 per month on average (median = $16 013). For original indications, prices weakly correlated to improvements in overall survival (β = 0.28, P = .037) and progression-free survival (β = 0.16, P = .001). Original indications’ prices were as follows: (1) negatively associated with disease incidence (β = −0.21, P < .001) and prevalence; (2) positively correlated with first-in-class drugs (26%, P = .057), gene and cell therapies (176%, P < .001), hematologic cancers (62%, P < .001), and severe diseases with substantial unmet needs (6% per disability-adjusted life-year, P < .001); and (3) negatively correlated to indications with randomized-controlled phase 3 trials. Prices were poorly associated with supplemental indications’ efficacy, clinical evidence, and epidemiology. ConclusionsCancer drug prices are set based on the original indication’s characteristics, thereby omitting the value of supplemental indications. Indication-specific pricing, coverage, and reimbursement policies considering each indication’s safety, efficacy, innovativeness, and unmet needs are necessary to align a drug’s value and price.

The cancer premium - explaining differences in prices for cancer vs non-cancer drugs with efficacy and epidemiological endpoints in the US, Germany, and Switzerland: a cross sectional study.

High treatment prices of new cancer drugs are a global public health challenge to patients and healthcare systems. Policymakers in the US and Europe are debating reforms to drug pricing. The objective of this study was to assess whether drug efficacy or epidemiological characteristics (prevalence, incidence, mortality) explain the gap in treatment prices between cancer and non-cancer drugs in the US, Germany, and Switzerland. This cross-sectional study identified all new drugs approved in the US, Germany, and Switzerland between 2011 and 2020. Drug efficacy was extracted from pivotal trials, drug prices from public and commercial databases, and epidemiological characteristics from the Global Burden of Disease (GBD) 2019 study. We used regression models to explain drug prices with drug efficacy and epidemiological characteristics (prevalence, incidence, mortality). The cohort included 181 drugs, including 68 (37.5%) drugs approved for treatment of cancer. A significant negative correlation was found between incidence/prevalence and treatment prices, and a significant positive correlation was observed between mortality and treatment prices for both, cancer and non-cancer drugs. A significant association between relative drug efficacy and treatment prices of drugs was observed, however, less pronounced for cancer drugs. Our regression estimates indicated that after adjusting for efficacy and epidemiological characteristics, cancer drugs were on average approximately three times more expensive compared to non-cancer drugs in all three countries, indicating a cancer premium; i.e., treatment prices of cancer drugs were on average USD 74,412 (95% CI [62,810; 86,015]) more expensive in the US compared to non-cancer drugs, USD 37,770 (95% CI [26,175; 49,367]) more expensive in Germany, and USD 32,801 (95% CI [27,048; 38,555]) more expensive in Switzerland. Our model explained 72% of the variance in observed prices (R2). Drug pricing reforms should target the cancer premium to improve access of patients to cancer drugs as well as to achieve equity across the different therapeutic areas and sustainability in the health care systems. This study was funded by the Swiss National Science Foundation (SNSF, grant number PCEGP1_194607) and the Swiss Cancer Research Foundation (Krebsforschung Schweiz).

Marketing authorisation and pricing of FDA-approved cancer drugs in Brazil: a retrospective analysis

Most cancer drugs enter the US market first. US Food and Drug Administration (FDA) approvals of new cancer drugs may influence regulatory decisions in other settings. The study examined whether characteristics of available evidence at FDA approval influenced time-to-marketing authorisation (MA) in Brazil, and price differences between the two countries. All new FDA-approved cancer drugs from 2010 to 2019 were matched to drugs with MA and prices approved in Brazil by December 2020. Characteristics of main studies, availability of randomised controlled trials (RCTs), overall survival (OS) benefit, added therapeutic benefit, and prices were compared. Fifty-six FDA-approved cancer drugs with matching indications received a MA at the Brazilian Health Regulatory Agency (Anvisa) after a median of 522 days following US approval (IQR: 351-932). Earlier authorisation in Brazil was associated with availability of RCT (median: 506 vs 760 days, p=0.031) and evidence of OS benefit (390 vs 543 days, p=0.019) at FDA approval. At Brazilian marketing authorisation, a greater proportion of cancer drugs had main RCTs (75% vs 60.7%) and OS benefit (42.9% vs 21.4%) than that in the US. Twenty-eight (50%) drugs did not demonstrate added therapeutic benefit over drugs for the same indication in Brazil. Median approved prices of new cancer drugs were 12.9% lower in Brazil compared to the US (adjusted by Purchasing Power Parity). However, for drugs with added therapeutic benefit median prices were 5.9% higher in Brazil compared to the US, while 17.9% lower for those without added benefit. High-quality clinical evidence accelerated the availability of cancer medicines in Brazil. The combination of marketing and pricing authorisation in Brazil may favour the approval of cancer drugs with better supporting evidence, and more meaningful clinical benefit albeit with variable degree of success in achieving lower prices compared to the US. None.

Determinants of cancer drug pricing and how to overcome the cancer premium

The high and increasing prices of cancer drugs are a public health challenge. To disrupt the cancer premium and improve patient access to cancer drugs, different action steps are indicated: more transparency on the price determination process and actual prices, value-based pricing, and "price with evidence development."

80P Efficacy, FDA approval, innovativeness, clinical evidence, and price of breast, ovarian, endometrial, and cervical cancer drugs

This study analyzes the innovativeness, clinical evidence, and efficacy of new drug indications with US Food and Drug Administration (FDA) approval for breast, ovarian, endometrial, and cervical cancer. We identified 26 drugs with FDA approval in 55 gynecological cancer indications from 2000 to 2021. For each indication, we collected drug, indication, and clinical trial characteristics from FDA labels. Hazard ratios (HRs) for overall survival (OS), progression-free survival (PFS), and relative risk (RR) for tumor response were meta-analyzed. Monthly treatment costs were calculated for US patients covered under Medicare & Medicaid. Of 55 indications, 36 received approval for breast, 12 for ovarian, 3 for endometrial, and 4 for ovarian cancer. Drugs acted via a targeted (84%), immune-regulating (13%), and cytotoxic (4%) mechanism of action. Approval was granted to 34 (62%) small-molecules, 16 (29%) antibodies, and 5 (9.1%) antibody-drug conjugates. Indications were predominantly first-or second-line (82%) combination therapies (62%) with a biomarker (84%). Indications frequently received the orphan (24%), fast track (20%), accelerated approval (27%), priority review (82%), and breakthrough therapy (40%) designation. Approval was mostly supported by double-blind (51%) phase 3 (75%) randomized-controlled (82%) trials enrolling a median of 521 patients (IQR: 302-724) for 3.8 years (IQR: 2.5-5.2). On average, new drugs HRs were 0.79 for OS (95%CI: 0.75-0.82) and 0.55 for PFS (95%CI: 0.50-0.60), whereas tumor response was 1.48 (95%CI: 1.37-1.60). Novel drugs increased patient survival by a median of 3.20 months for OS (IQR, 2.0-4.90 months) and 4.75 months for PFS (IQR, 2.40-7.10 months). Greater OS benefits were observed for drugs with a biomarker (HR: 0.76 vs. 0.84, p=.034). In 2022, Gynecological cancer drugs cost an average of $13,053.30 per month. Over the past 22 years, the approval of innovative and effective drugs transformed the landscape of gynecological cancer treatments. Especially biomarker-guided targeted therapies resulted in substantial improvements in OS and PFS. However, rising drug prices pose a challenge to healthcare systems and patient access.

Assessment of Price and Clinical Benefit of Cancer Drugs in Canada, 2011-2020

This cohort study examines the association between approval characteristics, clinical benefit, and prices of cancer drugs recommended for reimbursement by the Canadian Agency for Drug and Technology in Health.

Medication Payments by Insurers and Patients for the Treatment of Metastatic Castrate-Resistant Prostate Cancer.

The implications of high prices for cancer drugs on health care costs and patients' financial burdens are a growing concern. Patients with metastatic castrate-resistant prostate cancer (mCRPC) are often candidates for multiple first-line systemic therapies with similar impacts on life expectancy. However, little is known about the gross and out-of-pocket (OOP) payments associated with each of these drugs for patients with employer-sponsored health insurance. We therefore aimed to determine the gross and OOP payments of first-line drugs for mCRPC and how the payments vary across drugs. This retrospective cohort study included 4,298 patients with prostate cancer who initiated therapy with one of six drugs approved for first-line treatment of mCRPC between July 1, 2013, and June 30, 2019. We compared gross and OOP payments during the 6 months after initiation of treatment for mCRPC using private payer claims data across patients using different first-line drugs. Gross payments varied across drugs. Over the 6 months after the index prescription, mean unadjusted gross drug payments were highest for patients receiving sipuleucel-T ($115,525 USD) and lowest for patients using docetaxel ($12,804 USD). OOP payments were lower than gross drug payments; mean 6-month OOP payments were highest for cabazitaxel ($1,044 USD) and lowest for docetaxel ($296 USD). There was a wide distribution of OOP payments within drug types. Drugs for mCRPC are expensive with large differences in payments by drug type. OOP payments among patients with employer-sponsored health insurance are much lower than gross drug payments, and they vary both across and within first-line drug types, with some patients making very high OOP payments. Although lowering drug prices would reduce pharmaceutical spending for patients with mCRPC, decreasing patient financial burden requires understanding an individual patient's benefit design.