Chain Drug Review, July 8, 2019

64 Chain Drug Review July 8 2019 RX News R D Changes Spur Record Pace for Cancer Drug Approvals DURHAM N C Fifty nine new drugs were approved by U S regulators in 2018 a record pace driven by ongoing changes in the clinical development process including wider use of biomarkers prescreened patient pools regulatory shifts and application of artificial intelligence and predictive analytics Thats according to a recently released report from IQVIA that examines historical and future clinical trial productivity trends across therapy areas The report titled The Changing Landscape of Research and Development Innovation Drivers of Change and Evolution of Clinical Trial Productivity asserts that trial productivity in the coming years will be heavily influenced by the trends the authors identified as key drivers These drivers are Digital health technologies that enable the capture of drug efficacy and safety data remotely which can improve patient safety enable virtual trial formats and ease site work burden Patient reported outcomes that will shed new light on patient experience and drug efficacy and safety outside the clinical setting and lead to accelerated trial times as endpoints shift Real world data to optimize trial design speed investigator and site selection and enable new trial designs by acting as virtual control arms and supporting pragmatic adaptive and RWE registry trials Predictive analytics and artificial intelligence deployed to identify new clinical hypotheses reduce trial design risks and speed enrollment by identifying protocol ready patients Shifts in types of drugs tested for instance to targeted therapies and next generation biotherapeutics that improve efficacy and success rates and have accelerated development time lines but require longer term patient follow up Biomarker testing availability to help narrow patient populations to those more likely to see effect resulting in improvements in efficacy safety and success Regulatory landscape changes that will encourage the adoption of precision medicine approaches novel trial designs and endpoints while providing means for accelerated drug approvals and regulatory success Pools of prescreened patients and direct to patient recruitment which facilitate enhanced trial enrollment shortened trial duration and faster market availability The report concluded that these eight trends could increase R D productivity by double digits over the next five years As advances in science technology and data gradually find application within clinical development the length of time that trials take to complete the resources required due to trial complexity and likelihood of trial success are all shifting with impacts varying by therapy area said Murray Aitken IQVIA senior vice president and executive director of the IQVIA Institute for Human Data Science Our study assesses the current activity within research and development the productivity levels of the clinical development process and how key trial trends will transform clinical development over the next five years The report found the likelihood that a drug entering clinical development would be a success was 11 in 2018 down three percentage points from the year earlier The report found that emerging biopharma companies account for 72 of all late stage pipeline activity up from 61 a decade earlier Large pharma companies with more than 10 billion in annual pharmaceutical sales have seen their R D share drop from 31 to 20 during that period This pipeline mix reflects smaller companies being most active in the fastest growing areas of oncology and orphan drugs and their diminishing need for partnering or acquisition to develop and commercialize their innovative medicines according to the report Investment in medical innovation grew in 2018 reflecting confidence in scientific development to propel new treatments to tackle unmet health needs across a broad range of diseases IQVIA reported Citing the National Venture Capital Association the authors note that more than 1300 life science venture capital deals were closed in 2018 with an aggregate value of more than 23 billion up from roughly 10 billion in deal value in 2013 Developing innovative medicines continues to be a slow process with the 2018 cohort taking a median of 137 years from the time of first patent filing to product launch said IQVIA which noted that this was nearly six months faster than the median of the past five years Of the 2018 new drug launches four new molecular entities launched in under eight years while another 12 drugs launched more than 20 years after their first patent filing reflecting in some cases older mechanisms of action and the approval of drugs that had launched in countries outside the United States the authors said The report noted dramatic R D successes and failures in 2018 Within chronic liver disease research nine new drugs were added to the late stage pipeline in 2018 as drugs with several mechanisms continue to show promise within that therapeutic area IQVIA said However within the Alz heimers category four drugs failed in development bringing the total number of failures to 85 during the past decade even as unmet needs remain significant and potential health care cost impacts loom over future generations Treatment With Newer CDK 4 6 Targeted Breast Cancer Therapies Has Increased Dramatically in Both the United States and Europe Exhibit 8 Metastatic Breast Cancer Patients Treated with Regimens Containing CDK 4 6 Agents in Thousands 2015 2016 2017 2018 2015 2016 2017 2018 Top 5 Europe Palbociclib Ribociclib Abemaciclib US Source IQVIA Real World Evidence Medical Claims Dec 2018 IQVIA Oncology Analyzer Dec 2016 Oncology Dynamics Dec 2018 711 Companies Are Active in Late Stage Oncology R D With a Significant Contribution From Exhibit 13 Company Late Stage Pipelines Number of Oncology Indications and Oncology Percent of Pipeline 463 22 7 2 Academic Mid Size 60 40 20 Source IQVIA R D Intelligence Dec 2018 IQVIA Institute Jan 2019 Companies n 494 Top 10 Oncology Other Large Pharma EBP 0 0 20 40 60 80 100 Oncology Indications in Late Stage Pipeline Oncology Percentage of Late Stage Pipeline Bubble size Number of Molecules in Late Stage Pipeline 25 20 15 10 5 1 Emerging Biopharma Companies 50 40 30 20 10 0 Source IQVIA Real World Evidence Medical Claims Dec 2018 IQVIA Oncology Analyzer Dec 2016 Oncology Dynamics Dec 2018 Murray Aitken INNOVATION IN PATIENT THERAPIES RESEARCH AND DEVELOPMENT ACTIVITIES Source IQVIA R D Intelligence Dec 2018 IQVIA Institute Jan 2019 Report Mortality Rates From Cancer Keep Falling ROCKVILLE Md The latest Annual Report to the Nation on the Status of Cancer finds that for all cancer sites combined cancer death rates continued to decline in men women and children in the United States from 1999 to 2016 Overall cancer incidence rates or rates of new cancers decreased in men from 2008 to 2015 after increasing from 1999 to 2008 and were stable in women from 1999 to 2015 The report is a collaborative effort among the National Cancer Institute NCI part of the National Institutes of Health the Centers for Disease Control and Prevention CDC the American Cancer Society ACS and the North American Association of Central Cancer Registries NAACCR The report appeared in the Journal of the National Cancer Institute We are encouraged by the fact that this years report continues to show declining cancer mortality for men women and children as well as other indicators of progress said Betsy Kohler executive director of NAACCR There are also several findings that highlight the importance of continued research and cancer prevention efforts A special section shows a different picture for cancer incidence and mortality among men and women ages 20 to 49 than among people of all ages In the main report from 2011 to 2015 the average annual incidence rate for all cancer sites combined was about 12 times higher among men than among women and from 2012 to 2016 the average annual death rate among men all ages was 14 times the rate among women However when the researchers looked only at men and women ages 20 to 49 they found that both incidence and death rates were higher among women The authors reported that in the 20 to 49 age group from 2011 to 2015 the average annual incidence rate for all invasive cancers was 1153 per 100000 people among men compared with 2033 among women with cancer incidence rates decreasing an average of 07 per year among men and increasing an average of 13 per year among women Length of trials and chances of success are seeing shifts Death rates dropped for all sites combined from 1999 to 2016