The year's new drugs and biologics 2020.

2020 will go down in history as a year marked in every respect by the emergence and astonishingly rapid spread of the first major global viral pandemic in a century. It seems like nearly every event or story of the year was influenced in some way by COVID-19, and in that respect, the year ended on a high note with the authorization for emergency use of the first vaccines to prevent SARS-CoV-2 infection and drugs to treat COVID-19. Despite the pandemic's dominance of the 2020 headlines, productivity was at a record high level across all therapeutic areas, as seen by the number of products in this year's review: approximately 50% more than the previous year. Notable achievements include the launch of the first treatment for hepatitis D; regulatory decisions on a suite of biologics for the prevention and treatment of Ebola virus disease, fruit of the 2016-2018 outbreak in the Democratic Republic of Congo; the approval of the first-ever drug to treat Hutchinson-Gilford progeria syndrome, a rare genetic disorder that leads to premature aging; the first treatment developed specifically for thyroid eye disease, also known as Graves' ophthalmopathy; the first nonhormonal, on-demand, vaginal pH-regulating contraceptive; and the first oral allergen immunotherapy for peanut allergy.

The year's new drugs and biologics 2019.

Highlights of our annual review of new approvals and launches on global drug markets include the approval and launch of Trikafta, the most widely applicable treatment to date for cystic fibrosis; approval of the first Ebola vaccine for general (rather than emergency) use; the pilot rollout in three African countries of the world's first malaria vaccine; approval of a new treatment option for multidrug-resistant bacterial infections; and the approval and launch in China of the first new drug to treat Alzheimer's disease in more than a decade. Several new immune checkpoint inhibitors and antibody-drug conjugates were approved for cancer indications, confirming continued industry enthusiasm for cancer immunotherapy. The most notable trend of 2019 was the granting by the Food and Drug Administration (FDA) of a record number of accelerated approvals, many of which were issued several months ahead of the expected action date.

Effect of patient education on palliative care knowledge and acceptability of outpatient palliative care services among gynecologic oncology patients: A randomized controlled trial.

OBJECTIVES: A randomized control trial (RCT) to estimate the effect of an interventional video on improving palliative care knowledge, acceptability and attendance to outpatient services in gynecologic oncology patients. METHODS: Women receiving treatment for gynecologic malignancy recruited at an academic tertiary care center were randomized to: palliative care educational video or non-directive cancer center video. The primary outcome was referral to palliative care. Function and knowledge were assessed using the Functional Assessment of Cancer Therapy and the Palliative Care Knowledge Scale. Data analyses were performed using t-tests, Wilcoxon rank sum or Fisher's exact tests with significance level of α = 0.05. RESULTS: 111 women were enrolled. Demographic characteristics were equally distributed between groups with respect to age, race, cancer, and stage. There was no statistical difference in knowledge scores or in referral to palliative care between the patients that watched the educational versus control video (29% vs. 27%; p = .79). Secondary analysis showed a statistically significant increase in utilization of palliative care services compared to historic institutional data (8.8% to 31.5%; p ≤.001). Further, those referred had significantly worse baseline functional scores. CONCLUSIONS: Use of a palliative care educational video did not increase knowledge or acceptability of palliative services within this RCT. However, the rate of patients referred to palliative care tripled compared to historic rates. Further studies should investigate whether discussion regarding palliative care services alone may increase desire for referral, and if use of Fact-G scores may identify patients in greatest need of services.

The year's new drugs and biologics 2018: Part II - News that shaped the industry in 2018.

This eagle's-eye overview of the drug industry in 2018 provides insight into some of last year's top stories, including a large and still growing outbreak of Ebola in the Democratic Republic of the Congo, as well as the potential uses (and abuses) of CRISPR technology. As in previous years, we also review orphan drug development, new regulatory agency-supported programs such as Priority Medicines Scheme (PRIME) and Sakigake, pipeline attrition, and pharma/biotech mergers and acquisitions of note. Finally, we take a glimpse into the crystal ball to anticipate the new drugs that will be approved in 2019.

The year's new drugs and biologics 2018: Part I.

Sixty-four new drugs and biologics reached their first global markets in 2018, the greatest number in at least a decade. In addition, 19 important new line extensions (new indications, new combinations or new formulations of previously marketed drugs) were introduced over the course of 2018. Twenty-three other new products and new line extensions were approved but not launched before December 31. As has been the trend in recent years, oncology was the most active therapeutic area in terms of new launches, with cancer drugs accounting for 25% of all novel drugs and biologics introduced worldwide last year. Eight first-in-class agents were launched for the first time in 2018, including the first RNA interference agent as well as the photo-finish approval and rollout of the first three members of a radically different class of non-vasoconstricting antimigraine agents, the anti-calcitonin gene-related peptide (CGRP) monoclonal antibodies. Twenty-nine of the drugs launched last year were designated orphan drugs, reflecting the continued upswing in investment in the area of neglected diseases. This is especially true in the U.S., which welcomed 25 of the newly launched orphan drugs. Overall, the U.S. was the most active market for new drugs and biologics, accounting for two-thirds of all new launches in 2018.

The year's new drugs & biologics, 2017, part II - News that shaped the industry in 2017.

This eagle's-eye overview of the drug industry in 2017 provides insight into some of last year's top stories, including the growing opioid crisis affecting the U.S. and other developed countries and the 2017-2018 influenza epidemic, with a spotlight on the need for a universal flu vaccine. As in previous years, we also review orphan drug development, new agency-supported programs such as PRIME and RMAT, pipeline attrition and drug pricing, as well as pharma/biotech mergers and acquisitions of note. Finally, we take a glimpse into the crystal ball to anticipate the new drugs that will be approved in 2018.

The year's new drugs and biologics 2017: Part I.

This review presents the 113 new drugs and biologics that were approved or launched for the first time globally in 2017. Fifty-two new drugs and biologics reached their first markets worldwide in 2017. Seven of the new launches were first-in-class agents, meaning the first drug with a novel mechanism of action to be approved and launched anywhere in the world. In addition, 36 notable line extensions (i.e., new indications, new combinations and new formulations of previously marketed products) were introduced last year. The remaining 25 products discussed in this article were approved for the first time during the year just passed, but had not yet been launched as of December 31, 2017. The United States was the most active market for new drugs, and oncology was the most active therapeutic group. Information on these new arrivals is covered in depth in part I of our annual review of the pharma and biotech industry.

The year's new drugs & biologics 2016: Part I.

Nearly 90 new drugs and biologics, including important new line extensions, were approved or launched for the first time globally in 2016, a comparatively lower number with respect to previous years. Forty-four new drugs and biologics reached their first markets worldwide in 2016, nearly 10% fewer than the previous year. Seven of the new launches were first-in-class agents, meaning the first drug with a novel mechanism of action to be approved and launched anywhere in the world. In addition, 23 novel line extensions (i.e., new formulations, new combinations and new indications) were introduced last year. The remaining 21 products discussed in this article were approved for the first time during the year just passed, but had not yet been launched as of December 15, 2016. Information on these new arrivals is covered in depth in part I of our annual review of the pharma and biotech industry.

The year's new drugs & biologics 2016: Part II - Trends and highlights of an unforgettable year.

This eagle's-eye overview of the drug industry in 2016 provides insight into some of last year's top stories, including disease outbreaks that drove R&D, orphan drug development, pipeline attrition, drug pricing, and the ongoing movement in M&A. We also consider recent political events in the U.S. and U.K. and their potential impact on the industry in the years to come, and take a glimpse into the crystal ball to anticipate the new drugs that may be approved in 2017.

Cachexia.

Cachexia is a multiorgan, multifactorial and often irreversible wasting syndrome associated with cancer and other serious, chronic illnesses including AIDS, chronic heart failure, chronic kidney disease and chronic obstructive pulmonary disease. Treatment of the patient with cachexia is currently targeted to correcting the two underlying features of the condition: anorexia and metabolic disturbances. Greater understanding of the mechanisms behind cachexia and muscle wasting have led to new therapeutic possibilities, however. Several classes of drugs are under active development for cachexia including drugs acting on hormone receptors or cytokine receptors, myostatin/activin pathway antagonists, beta-adrenoceptor agonists and cannabinoids. This review will cover the pathophysiology, epidemiology, diagnosis, treatment, drug candidates under active development and targets for therapeutic intervention of cachexia.

Duchenne muscular dystrophy drugs face tough path to approval.

Highly anticipated as new disease-modifying treatments for Duchenne muscular dystrophy (DMD), therapeutics by BioMarin Pharmaceutical (Kyndrisa™; drisapersen) and Sarepta Therapeutics (eteplirsen; AVI-4658) both recently received negative FDA reviews and are now facing battles for approval in the U.S. At present, BioMarin is committed to working with the FDA to forge a pathway to approval following the failure of its NDA, while Sarepta awaits the formal decision on its NDA, which is expected by late May 2016. Despite the critical nature of both reviews, analysts consider that there is still a narrow possibility of approval of both drugs. According to Consensus forecasts from Thomson Reuters Cortellis for Competitive Intelligence, Kyndrisa is forecast to achieve sales of USD 533.71 million in 2021.

The year's new drugs & biologics 2015 - part II:Trends and highlights that marked a complicated year.

This eagle's-eye overview of the drug industry in 2015 provides insight into some of last year's top stories, including drug pricing, orphan drug development, the FDA's priority review voucher system, pipeline attrition, and the ongoing movement in M&A. We also take a look into the crystal ball and anticipate the new drugs that may reach the market in 2016.

The year's new drugs & biologics 2015: Part I.

Nearly 100 new drugs and biologics, including important new line extensions, were approved or launched for the first time globally in 2015. These products are covered in depth in part I of our annual review of the pharma and biotech industry.

The year's new drugs & biologics 2014 - Part II: trends & challenges.

2014 was a year of continued high activity in the pharma and biotech industry, as evidenced in part I of this annual two-part review article published last month in this journal (1). As of December 23, 2014, a total of 55 new chemical and biological entities had reached their first markets worldwide, together with another 29 important new line extensions. Another 19 products were approved for the first time during the year but not yet launched by December 23. Furthermore, during the now-traditional year-end sprint, several regulatory agencies issued last-minute approvals for other compounds that missed the deadline for inclusion in that article, bringing the total of new approvals for the year to a somewhat higher number. In addition to the successful development, registration and launch of new drugs and biologics, there are various other trends and tendencies that serve as indicators of the overall health and status of the industry. These include the pursuit of novel programs designed by regulators to stimulate the development of drugs for diseases that are currently under-treated; the regular and pragmatic culling by companies of their R&D pipelines; and the decision to unify pipelines, portfolios and sales forces through mergers and acquisitions.

The year's new drugs & biologics, 2014: Part I.

A year-end wrap-up of new drug approvals and launches reveals that activity in the pharmaceutical industry continues at a high level, with 55 new drugs and biologics introduced on their first markets in 2014 (as of December 23, 2014). Additionally, 29 important new line extensions (new formulations, new combinations or new indications for previously marketed products) also reached their first markets during the year. The most active therapeutic group in terms of new launches was anti-infective therapies, with 11 new drugs and biologics launched, most for the treatment of multidrug-resistant bacterial infections or hepatitis C. The most active market for new launches was again the U.S., site of more than half of all new launches in 2014. However new launch activity increased considerably last year in Japan, which actually pulled ahead of the E.U. for the first time in many years. In another important new development, 15 of the new drugs and biologics launched last year had orphan drug status, 5 had breakthrough therapy designation and 3 had Qualified Infectious Disease Product (QIDP) status. Another 19 products were approved for the first time during the year but not yet launched by close of this article; most are slated for launch in the first months of the new year.

Parkinson's disease in the limelight.

The 2014 Lasker-DeBakey Clinical Medical Research Award -one of three prestigious awards granted by the Lasker Foundation in recognition of scientists, clinicians and public servants who have made major advances in the understanding, diagnosis, treatment, cure or prevention of human disease- has been granted to two pioneers in the field of Parkinson's disease therapy. In spite of the availability of more than two dozen drugs and fixed-dose combination products to treat the symptoms of Parkinson's disease -most notably the gold standard levodopa, a dopamine precursor- as well as nonpharmacological treatments like deep brain stimulation, many patients do not respond to available drugs or experience breakthrough symptoms, and the disease is ultimately uncurable. This article reviews currently available therapies as well as biomarkers and novel diagnostics.

The year's new drugs & biologics, 2013: Part II.

The demise of the pharmaceutical industry, so pessimistically predicted by many in recent years, has not come to pass and in fact the patient is alive and well. New programs enacted by drug regulators have been enthusiastically taken up by the industry, including the FDA's breakthrough therapy and qualified infectious disease product (QIDP) designations, as well as the now-consolidated orphan drug programs in many countries. Pharma companies pragmatically wean nonperformers from the pipeline in an efficient manner, resulting in somewhat leaner but higher-quality pipelines. Mergers and acquisitions also continue to drive consolidation and efficiency in the industry, a trend that continued during 2013. This article provides an updated review of these and other trends in the pharmaceutical industry in the year just passed.

The year's new drugs & biologics, 2013: Part I.

This article provides a comprehensive overview of the 56 new drugs and biologics introduced for the first time in 2013, the largest number in at least a decade. This includes 20 new orphan drugs and 10 first-in-class agents, as well as the first three products bearing the FDA's new Breakthrough Therapy Designation. The review also covers 30 important new line extensions, encompassing new indications, new formulations and new combinations of previously marketed agents. In addition to this bumper crop of new launches, another 19 products were approved for the first time during the year but not yet launched by the close of this article; these new products are also discussed.

2012 in review - part II: overcoming the obstacles in the pharma/biotech industry.

As highlighted in the first part of this review published last month, the year 2012 saw the approval of a remarkable number of new drugs, and among the new drugs reaching the market, a significant proportion were orphan drugs developed for treating less prevalent diseases. These drugs are certainly not expected to become blockbusters, but are of high interest because of their efficacy in a narrow spectrum of patients. This trend aligns with the general tendency of staying away from fit-for-all blockbusters into personalized medicine as one of the strategies for overcoming the patent cliff that resulted in a long list of drugs going off patent and being approved as generics also during last year. The emerging scenario resulting from new developments in the form of new drugs and biosimilars and newly available generic medications paralleled by strategic movements within the pharmaceutical industry to reinforce their position in the market, as reflected by merger and acquisition deals accompanied by significant efforts into prioritization resulting in spin-off and split transactions, is reviewed in this second part. This paper includes a significant amount of data in tables for quick review and to profile the new strategic movements in drug pipelines. Further information, including details on mechanisms of action, current status, itemized pharmacology, pharmacokinetic and clinical trial research findings and updated information can be found in the proprietary databases Thomson Reuters Integrity(SM) and Thomson Reuters Cortellis™.