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This book tells the little-known story of how an upstart biotechnology company created a one-in-a-million cancer drug, and how the core team - denied their share of the profits - went and did it again. In this epic saga of money and science, a veteran financial journalist explains how the invention of two of the biggest cancer drugs in history became (for their backers) two of the greatest Wall Street bets of all time. In the multibillion-dollar business of biotech, where pharmaceutical companies, the government, hedge funds, and venture capitalists have spent billions on funding, experimentation, and treatments, a single molecule can stop cancer in its tracks - and make the people who find that rare molecule astonishingly rich. This book follows a small team at a biotech start-up in California, who have found one of these rare molecules. Their compound, known as a BTK inhibitor, seems to work on a vicious type of leukemia. When patients start rising from their hospice beds, the team knows they're onto something big. What follows is a story of genius, pathos, and drama, in which vivid characters navigate a world of corporate intrigue and ambiguous morality. The author's narrative immerses readers in the explosion of biotech start-ups. He describes the scientists, doctors, and investors who are risking everything to develop new, life-saving treatments, and introduces suffering patients for whom the stakes are life-or-death. A gripping nonfiction read, this book illustrates why it's so hard to bring new drugs to market, explains why they are so expensive, and examines how profit-driven venture capitalists are shaping the future of medicine. -- Adapted from publisher's description.

Only 3.4% of cancer drugs evaluated in phase 1 trials are approved by the US Food and Drug Administration, with most failing in phase 3 trials. To investigate whether an association exists between the sponsorship and conduct of a negative phase 3 randomized clinical trial (RCT) investigating a cancer drug that lacked supporting phase 2 trial evidence for that drug, and to evaluate the association with overall survival among patients randomized to the experimental arm of such phase 3 trials. Articles in the Lancet, Lancet Oncology, JAMA, JAMA Oncology, and Journal of Clinical Oncology published between January 2016 and June 2018 were searched. Phase 3 RCTs of cancer drugs that failed to improve the primary end point were selected and any prior phase 2 trial of the same drug that supported the phase 3 trial was selected without any date or journal restrictions. Percentages of negative phase 3 RCTs of cancer drugs that lacked any phase 2 evidence, had a negative phase 2 trial, or had a positive phase 2 study were extracted. Associations were assessed using the Fisher exact test. Pooled hazard ratios and 95% CIs for the overall survival of patients enrolled in these negative phase 3 RCTs were estimated using a random-effects model. Negative phase 3 RCTs with a lack of a phase 2 trial or the presence of a negative phase 2 trial and overall survival of enrolled patients in the phase 3 RCTs. In this meta-epidemiological study, 67 negative phase 3 RCTs on cancer drugs, which included 64 600 patients, met the criteria of being sponsored by industry or academic groups, of which 42 RCTs (63%) were industry sponsored and the remaining 25 RCTs (37%) were academic. A phase 2 trial was not available for 28 of these trials (42%). Of 29 trials (43%) with a phase 2 trial available, 8 trials (28%) failed to meet their primary end points and 5 of those were industry sponsored. There was no association with overall survival for patients participating in these negative phase 3 RCTs (pooled hazard ratio, 0.99; 95% CI, 0.96-1.02). When the pooled analysis was limited to the 27 RCTs with a hazard ratio above 1.00, the overall pooled hazard ratio for overall survival was 1.11 (95% CI, 1.06-1.16). No association between having a negative or undefined phase 2 trial and trial sponsorship was found using the Fisher exact test. More than 40% of the negative phase 3 RCTs in oncology published in these 5 journals were conducted without a supporting phase 2 trial and were sponsored by both academia and industry. Running such trials not only may risk loss of resources owing to a failed trial but also may be associated with decreased patient survival. Further research and regulations in this area appear warranted.

There is increasing global recognition that national cancer plans are crucial to effectively address the cancer burden and to prioritise and coordinate programmes. We did a global analysis of available national cancer-related health plans using a standardised assessment questionnaire to assess their inclusion of elements that characterise an effective cancer plan and, thereby, improve understanding of the strengths and limitations of existing plans. The results show progress in the development of cancer plans, as well as in the inclusion of stakeholders in plan development, but little evidence of their implementation. Areas of continued unmet need include setting of realistic priorities, specification of programmes for cancer management, allocation of appropriate budgets, monitoring and evaluation of plan implementation, promotion of research, and strengthening of information systems. We found that countries with a non-communicable disease (NCD) plan but no national cancer control plan (NCCP) were less likely than countries with an NCCP and NCP plan or an NCCP only to have comprehensive, coherent, or consistent plans. As countries move towards universal health coverage, greater emphasis is needed on developing NCCPs that are evidence based, financed, and implemented to ensure translation into action.

Approximately 1 in 2 Japanese people are estimated to be diagnosed with cancer during their lifetime. Cancer still remains the leading cause of death in Japan, therefore the government of Japan has decided to develop a better cancer control policy and launched the Cancer Genomic Medicine (CGM) program. The Ministry of Health, Labour, and Welfare (MHLW) held a consortium at their headquarters with leading academic authorities and the representatives of related organizations to discuss ways to advance CGM in Japan. Based on the report of the consortium, the CGM system under the national health insurance system has gradually been realized. Eleven hospitals were designated in February 2018 as core hospitals for CGM; subsequently, the MHLW built the Center for Cancer Genomics and Advanced Therapeutics (C‐CAT) as an institution to aggregate and manage genomic and clinical information on cancer patients, and support appropriate secondary use of the aggregated information to develop research aimed at medical innovation. As the first step in Japan's CGM in routine practice, in June 2019 the MHLW started reimbursement of 2 types of tumor profiling tests for advanced solid cancer patients using the national insurance system. Japan's CGM has swiftly been spreading nationwide with the collaboration of 167 hospitals and patients. The health and research authorities are expected to embody personalized cancer medicine and promote CGM utilizing state‐of‐the‐art technologies. As the first step in Cancer Genomic Medicine (CGM) in routine practice in Japan, since June 2019 the Ministry has started reimbursement of 2 gene panel tumor profiling tests for advanced solid cancer patients using the national insurance system. CGM has swiftly been spreading nationwide in Japan with the collaboration of 167 hospitals and patients. The Ministry are expected to realize further personalized cancer medicine and promote CGM utilizing state‐of‐the‐art technologies.

In 2008, 2·45 million people were diagnosed with cancer and 1·23 million died because of cancer in the 27 countries of the European Union (EU). We aimed to estimate the economic burden of cancer in the EU. In a population-based cost analysis, we evaluated the cost of all cancers and also those associated with breast, colorectal, lung, and prostate cancers. We obtained country-specific aggregate data for morbidity, mortality, and health-care resource use from international and national sources. We estimated health-care costs from expenditure on care in the primary, outpatient, emergency, and inpatient settings, and also drugs. Additionally, we estimated the costs of unpaid care provided by relatives or friends of patients (ie, informal care), lost earnings after premature death, and costs associated with individuals who temporarily or permanently left employment because of illness. Cancer cost the EU €126 billion in 2009, with health care accounting for €51·0 billion (40%). Across the EU, the health-care costs of cancer were equivalent to €102 per citizen, but varied substantially from €16 per person in Bulgaria to €184 per person in Luxembourg. Productivity losses because of early death cost €42·6 billion and lost working days €9·43 billion. Informal care cost €23·2 billion. Lung cancer had the highest economic cost (€18·8 billion, 15% of overall cancer costs), followed by breast cancer (€15·0 billion, 12%), colorectal cancer (€13·1 billion, 10%), and prostate cancer (€8·43 billion, 7%). Our results show wide differences between countries, the reasons for which need further investigation. These data contribute to public health and policy intelligence, which is required to deliver affordable cancer care systems and inform effective public research funds allocation. Pfizer.

Precision oncology has not been shown to work, and perhaps it never will, says Vinay Prasad.

Important breakthroughs in medical treatments have improved outcomes for patients suffering from several types of cancer. However, many oncological treatments approved by regulatory agencies are of low value and do not contribute significantly to cancer mortality reduction, but lead to unrealistic patient expectations and push even affluent societies to unsustainable health care costs. Several factors that contribute to approvals of low-value oncology treatments are addressed, including issues with clinical trials, bias in reporting, regulatory agency shortcomings and drug pricing. With the COVID-19 pandemic enforcing the elimination of low-value interventions in all fields of medicine, efforts should urgently be made by all involved in cancer care to select only high-value and sustainable interventions. Transformation of medical education, improvement in clinical trial design, quality, conduct and reporting, strict adherence to scientific norms by regulatory agencies and use of value-based scales can all contribute to raising the bar for oncology drug approvals and influence drug pricing and availability.

July, 1, 2026, will mark 10 years since the launch of the OCM, perhaps the most ambitious of the alternative payment models from the Center for Medicare and Medicaid Innovation. Although official reports from Abt Global have centered on financial loses to Medicare, due to generous payments to oncology practices for required services, oncology practice leaders have insisted that the true legacy of the OCM has been overlooked; in their view, the model transformed everyday practice and physician thinking about their responsibility for what happened beyond the 4 walls of the cancer clinic. This qualitative look at the OCM features interviews with physicians and other key leaders involved in the OCM’s early days. The author integrates recent scholarship that takes note of flaws in the OCM model and other considerations when evaluating long-term Medicare savings.

Gastric cancer is the fourth most common cancer globally, and is the second most common cause of death from cancer worldwide. About three-quarters of newly diagnosed cases in 2008 were from Asian countries. With a high mortality-to-incidence ratio, management of gastric cancer is challenging. We discuss evidence for optimum management of gastric cancer in aspects of screening and early detection, diagnosis, and staging; endoscopic and surgical intervention; and the concepts of perioperative, postoperative, and palliative chemotherapy and use of molecularly targeted therapy. Recommendations are formulated on the basis of the framework provided by the Breast Health Global Initiative, using the categories of basic, limited, enhanced, and maximum level. We aim to provide a stepwise strategy for management of gastric cancer applicable to different levels of health-care resources in Asian countries.

Recommendations by countries’ health technology assessment (HTA) agencies are used to decide which new therapies warrant the allocation of limited health-care resources to make them available through publicly funded health systems. This process is of public health importance for balancing the dual aims of optimising patient outcomes while ensuring financial sustainability. We evaluated which factors affect HTA outcomes and the time to positive HTA outcome, focussing on the role of clinical benefit evaluated with the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS). In this retrospective analysis, data were extracted from publicly available HTA reports and related sources from six country settings and their respective HTA agencies (Australia, Canada, England, France, the Canadian province of Quebec, and Scotland). We evaluated new cancer medicines for treating solid tumours in a non-curative setting with published ESMO-MCBS scores and that had been assessed by at least three HTA agencies between Jan 1, 2011, and Dec 31, 2020. Using ESMO-MCBS score as an independent variable, we did descriptive and multivariable regression analyses to evaluate: (1) factors associated with the time between marketing authorisation and positive (unrestricted [List] and restricted [List with Constraints]) HTA outcome; and (2) factors associated with HTA outcomes. 67 medicine–indication pairs used in non-curative settings were identified, totalling 360 HTA submissions (medicine–indication–country triplets) reviewed by the six HTA agencies. Factors significantly associated with a reduced interval between marketing authorisation and a positive (unrestricted or restricted) HTA outcome included a high ESMO-MCBS score (ie, 4 or 5, vs a low or average score of 1–3; hazard ratio [HR] per 1 month increment 1·42 [95% CI 1·11–1·81], p=0·0055), parallel review (vs standard marketing authorisation process; HR 1·69 [1·13–2·54], p=0·011), having a risk-sharing agreement or special funding arrangements (vs no funding agreement, HR 4·62 [95% CI 2·51–8·51], p<0·0001, and HR 4·16 [2·03–8·50], p=0·0001, respectively), and assessment by particular HTA agencies (pan-Canadian Oncology Drug Review vs National Institute for Health and Care Excellence [NICE], HR 2·82 [1·68–4·75], p=0·0001; and Haute Autorité de Santé vs NICE, HR 5·70 [2·87–11·33], p<0·0001). Accelerated marketing authorisation was significantly associated with a longer time to positive HTA outcome (vs standard authorisation process; HR 0·70 [95% CI 0·51–0·95], p=0·024). Positive HTA outcomes (both unrestricted and restricted) were significantly associated with a high ESMO-MCBS score (vs low or average ESMO-MCBS score; relative risk ratio [RRR] 14·10 [95% CI 3·54–56·20], p=0·0002, and RRR 4·52 [1·90–10·75], p=0·0006, respectively) and acknowledgment of unmet medical need (vs unmet need not recorded, RRR 22·73 [5·51–93·73], p<0·0001, and RRR 1·87 [1·18–2·97], p=0·0075, respectively). By contrast, positive HTA outcomes (unrestricted and restricted) were inversely associated with uncertainties regarding inputs to economic models informing HTA submissions (vs uncertainties not recorded, RRR 0·28 [0·10–0·78], p=0·014, and RRR 0·45 [0·25–0·82], p=0·010, respectively). Regarding country-relevant effects, inverse associations with positive HTA outcomes (both unrestricted and restricted) were observed for assessment in Quebec (vs England; RRR 1·15×10−6 [1·44×10−7–9·09×10−6], p<0·0001, and RRR 0·33 (0·24–0·46), p<0·0001, respectively) and for assessment in Australia (vs England; RRR 1·78×10−6 [1·04×10−8–3·00×10−4], p<0·0001, and RRR 0·30 [0·15–0·61], p=0·0008, respectively). Several factors informed HTA outcomes for new cancer medicines. A high ESMO-MCBS score, defined as indicating substantial clinical benefit, increased the likelihood of a positive HTA outcome and shortened the interval between marketing authorisation and HTA outcome, and this association was not affected by other variables. Additional factors informing HTA outcomes include evidence uncertainties and unmet medical need. Country-relevant differences exist in the time-to-HTA outcome and the propensity of some countries to achieve positive (restricted or unrestricted) outcomes compared with others. None.