IMPORTANCE: A common justification for high cancer drug prices is the sizable research and development (R&D) outlay necessary to bring a drug to the US market. A recent estimate of R&D spending is ...$2.7 billion (2017 US dollars). However, this analysis lacks transparency and independent replication. OBJECTIVE: To provide a contemporary estimate of R&D spending to develop cancer drugs. DESIGN, SETTING, AND PARTICIPANTS: Analysis of US Securities and Exchange Commission filings for drug companies with no drugs on the US market that received approval by the US Food and Drug Administration for a cancer drug from January 1, 2006, through December 31, 2015. Cumulative R&D spending was estimated from initiation of drug development activity to date of approval. Earnings were also identified from the time of approval to the present. The study was conducted from December 10, 2016, to March 2, 2017. MAIN OUTCOMES AND MEASURES: Median R&D spending on cancer drug development. RESULTS: Ten companies and drugs were included in this analysis. The 10 companies had a median time to develop a drug of 7.3 years (range, 5.8-15.2 years). Five drugs (50%) received accelerated approval from the US Food and Drug Administration, and 5 (50%) received regular approval. The median cost of drug development was $648.0 million (range, $157.3 million to $1950.8 million). The median cost was $757.4 million (range, $203.6 million to $2601.7 million) for a 7% per annum cost of capital (or opportunity costs) and $793.6 million (range, $219.1 million to $2827.1 million) for a 9% opportunity costs. With a median of 4.0 years (range, 0.8-8.8 years) since approval, the total revenue from sales of these 10 drugs since approval was $67.0 billion compared with total R&D spending of $7.2 billion ($9.1 billion, including 7% opportunity costs). CONCLUSIONS AND RELEVANCE: The cost to develop a cancer drug is $648.0 million, a figure significantly lower than prior estimates. The revenue since approval is substantial (median, $1658.4 million; range, $204.1 million to $22 275.0 million). This analysis provides a transparent estimate of R&D spending on cancer drugs and has implications for the current debate on drug pricing.
ABSTRACTDespite considerable advances in treatment approaches in the past two decades, multiple myeloma remains an incurable disease. Treatments for myeloma continue to evolve with many emerging ...immunotherapies. The first immunotherapy used to treat hematologic cancers, including multiple myeloma, was an allogeneic stem cell transplant. In the mid-2000s, immunomodulatory drugs thalidomide, lenalidomide, and subsequently pomalidomide were proven to be effective in multiple myeloma and substantially improved survival. The next wave of immunotherapies for multiple myeloma included the monoclonal antibodies daratumumab and elotuzumab, which were approved by the Food and Drug Administration in 2015. Subsequently, a variety of immunotherapies have been developed for multiple myeloma, including chimeric antigen receptor T cells, bispecific antibodies, antibody drug conjugates, and checkpoint inhibitors. Many of these emerging treatments target the B cell maturation antigen, which is expressed on plasma cells, although several other novel receptors are also being studied. This review summarizes the evidence of these various immunotherapies, their mechanism of action, and data from clinical trials regarding the treatments’ safety and efficacy.
B-cell maturation antigen (BCMA) was identified as a prognostic marker in multiple myeloma more than two decades ago. Starting in 2014, the first use of BCMA as a treatment target was evaluated in ...patients with multiple myeloma who received chimeric antigen receptor (CAR) T cells.
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Today, a range of myeloma treatments target BCMA, including CAR T cells, along with antibody drug conjugates and bispecific antibodies; some agents are in development, and some have been approved by the Food and Drug Administration (FDA).
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The rapid translation from preclinical studies to several FDA-approved treatments is a new chapter in the remarkable success . . .
Globally, annual spending on anticancer drugs is around US$100 billion, and is predicted to rise to $150 billion by 2020. In the USA, a novel anticancer drug routinely costs more than $100,000 per ...year of treatment. When adjusted for per capita spending power, however, drugs are most unaffordable in economically developing nations, such as India and China. Not only are launch prices high and rising, but individual drug prices are often escalated during exclusivity periods. High drug prices harm patients - often directly through increased out-of-pocket expenses, which reduce levels of patient compliance and lead to unfavourable outcomes - and harms society - by imposing cumulative price burdens that are unsustainable. Moreover, high drug prices are not readily explained by rational factors, including the extent of benefit patients are likely to derive, the novelty of the agents, or spending on research and development. Herein, we summarize the available empirical evidence on the costs of anticancer drugs, probe the origins and implications of these high costs, and discuss proposed solutions.
Resistance mechanisms are being defined for CAR T cells. Tumor cells emerging after GPRC5D CAR T-cell therapy could have decreased or lost expression of the GPRC5D target through biallelic gene loss ...or transcriptional down-regulation.
High prices for cancer drugs preclude independent comparative effectiveness trials that would seek to establish equally effective but cheaper alternatives — thereby protecting the market share of ...expensive drugs. How can we make progress toward conducting such trials?
The high cost of cancer drugs has been criticized by leading academics
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and lamented in the popular press.
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The average price of 1 year of treatment with a new cancer drug now exceeds $100,000,
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and the benefits of many of these therapies — often improvement in median survival on the order of weeks to months — do not appear commensurate with their prices.
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Expensive cancer drugs cost society in two ways. First, high prices are borne by payers each time these drugs are prescribed. And second, high prices preclude independent comparative effectiveness trials that would seek to establish equally effective . . .
GPRC5D-Targeted CAR T Cells for Myeloma Mailankody, Sham; Devlin, Sean M.; Landa, Jonathan ...
The New England journal of medicine,
09/2022, Letnik:
387, Številka:
13
Journal Article
Recenzirano
Odprti dostop
In an early-phase study involving 17 patients with highly refractory multiple myeloma, CAR T-cell therapy with specificity for a G protein–coupled receptor that is expressed on myeloma cells produced ...a response in 71% of the patients.
Summary
Incidence of venous thromboembolism (VTE) varies across different regimens in newly diagnosed multiple myeloma (NDMM) patients. Limited data exist on the use of direct oral anticoagulants as ...thromboprophylaxis in the setting of haematologic malignancies, specifically multiple myeloma. In this retrospective study of 305 NDMM patients, VTE rates in those treated with carfilzomib, lenalidomide, dexamethasone (KRD) + aspirin (ASA), bortezomib, lenalidomide, dexamethasone (RVD) + ASA, and KRD + rivaroxaban were statistically significant, 16·1%, 4·8%, and 4·8%, respectively. The findings confirm a higher incidence of VTE when using KRD induction compared to RVD induction and reveal that the use of low‐dose rivaroxaban thromboprophylaxis can mitigate this risk without an observable increase in bleeding rates.
Summary
Matrix‐assisted laser desorption ionisation time‐of‐flight mass spectrometry (MALDI‐TOF MS) may soon replace routine electrophoretic methods for monitoring monoclonal proteins in patients ...with multiple myeloma. To further evaluate the clinical utility of this assay, we compared the performance of MALDI‐TOF‐MS head‐to‐head with an established bone marrow‐based measurable residual disease assay by flow cytometry (Flow‐BM‐MRD), using Memorial Sloan Kettering Cancer Center’s 10‐color, single‐tube method. Our results suggest that MALDI‐TOF‐MS adds value to bone marrow‐based MRD testing and may be most useful for early detection of relapse in peripheral blood compared to current electrophoretic methods.
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