C & en: market point of American pharmaceutical industry in 2017

Recently, C & en, a well-known Journal of the American Chemical Society, published a long article called year in pharma, which, as a tradition at the end of each year, made a general survey of the American pharmaceutical industry in 2017 The pharmaceutical industry is facing a lot of innovations, such as cell therapy, gene therapy, and improved CRISPR technology At the same time, Donald J trump, the new president, fired on pharmaceutical companies at his first press conference, blaming the high drug prices and profiteering pharmaceutical market In a word, under the positive or negative uncertainty, it promotes the development and reflection of the pharmaceutical industry for another year 1 Uncertainty in the new government on January 11, Donald J trump began to condemn pharmaceutical companies at his first press conference as president-elect He vowed to shake the government and continue to pay high prices for drugs That 29 second comment sent the biotechnology index down 3% It's just one of a series of informal remarks about the pharmaceutical industry during Trump's first term in office So far, the threat of price negotiations has not been sharp But the general uncertainty on some key issues in the trump agenda (the fate of health care reform, corporate tax reform, even the ability to attract foreign scientists to the United States) has marginalised businesses and investors The meeting between trump and senior executives of the pharmaceutical industry (source: C & EN) Congress has put forward a lot of agenda for the new government because of the failure of previous implementation of medical reform However, there is little progress on a problem that the observers of the pharmaceutical industry pay attention to, which will have a significant impact on pharmaceutical companies in 2018: tax reform High corporate tax rates in the United States have led many companies to leave large amounts of cash overseas, estimated at $150 billion High taxes have even prompted some companies to acquire overseas companies in order to move their headquarters to tax-efficient countries Trump has introduced a law to encourage companies to repatriate or recover overseas funds, and many in the industry think it could spur big pharmaceutical companies to do more deals 2 The U.S Food and Drug Administration (FDA) has achieved great success in cancer immunotherapy this year, and approved two kinds of treatment methods using patients' own genetically modified cells to treat specific types of blood cancer Both new drugs are car-t cell immunotherapy, in which individual T cells are injected with DNA encoding chimeric antigen receptor (car) The first approved car-t drug is kymriah from Novartis, and about 83% of the patients treated achieve complete remission within three months The second drug is kit Pharma's yescarta, and more than 50% of the people who are treated have been completely relieved Despite the exciting results, there are still many challenges in car-t therapy First, both drugs are approved for only a small number of cancers The first challenge of car-t is to make it more widely used in more cancers, otherwise it will not have a great impact on the existing medical treatment Secondly, the toxicity of many car-t therapies is also a long-term problem Several car-t drugs in the R & D stage have been urgently stopped, that is, some patients died due to serious systemic immune response during the treatment process It's no exaggeration to say that it almost killed you before it helped you Personalized customization of car-t also makes manufacturing more complex and expensive Pricing has always been a public concern Kymriah's one-time cost is $475000, and yescarta's price is $373000, raising the issue of car-t and gene therapy costs But there is no doubt that car-t is an advanced technology Now it's just the beginning How about the future? We'll see 3 When President Donald J trump appointed Scott Gottlieb as FDA director, the pharmaceutical industry was relieved Gottlieb, a physician and former Deputy Commissioner of the agency, is considered the best choice After seven months in office, most people still think he is the most suitable person for the position He is a real push for policies based on medical needs, and he is also concerned about the growing medical science Scott Gottlieb (source: C & EN) describes Gottlieb in terms of balance While controlling the price of drugs is outside the FDA's remit, Gottlieb acknowledges more directly than his predecessors the agency's role in reducing medical costs This means policies will be introduced to accelerate the approval of generic drugs and measures will be taken to prevent artificial monopoly of complex non patented products On the other hand, he regrets the high and unsustainable cost of developing new drugs To make the process more efficient, he is calling for the modernization of clinical trial design and data collection Scott Gottlieb understands the FDA's role as a catalyst for innovation and is doing what it can to strengthen that role 4 As gene therapy matures, spinal muscular atrophy type 1 (SMA1) is a diagnostic result that no parents want to hear Due to mutations in the SMN1 gene, this rare genetic disease can lead to muscle atrophy and motor neuron necrosis It's hard for babies to even sit up Most people don't see their second birthday However, a clinical trial from avexis in Bannockburn, Illinois, showed that at least 15 infants born with SMA1 received some treatment The results, published last month, suggest that injecting gene therapy, which delivers a healthy copy of the SMN1 gene through the adeno-associated virus (AAV), gives infants 20 months or more to stay alive and kick Now most people can sit up, some are nearly three years old, and even walk and dance A hollow AAV model for delivering a piece of DNA (source: C & EN) in gene therapy Although after decades of research, only two gene therapies have been approved for use in Europe, the US FDA has not given any green light to gene therapy The success stories of avexis are inspiring this area Another gene therapy is luxturna from spark therapeutics, which is a one-off treatment that can make people with hereditary blindness see better The FDA Advisory Board unanimously recommended approval of the drug, which is expected to be approved early next year 5 The death toll from overdose in the United States has been high, and opioids are the main culprit According to the latest data from the Centers for Disease Control and prevention, in 2016, more than 64000 people died of drug overdose, mainly from opioid analgesics, heroin and synthetic analogues As far as the FDA is concerned, it has taken tougher measures against companies that make painkillers Earlier this year, the U.S FDA advisory committee voted by 18-8 votes that endo pharmaceutical company opana Er painkillers were at risk of abuse The FDA rarely asked it to remove the drug from the market The government is also tracking manufacturers for allegedly concealing the risk of addiction to opiates In September, prosecutors from 41 states jointly issued subpoenas to endo and other opioid manufacturers to investigate whether the companies had deceived doctors and patients about the risk of addiction to opioid analgesics Opioids (source: C & EN) in October, President Donald J trump declared that the opioid problem in the United States was a national public health emergency, but critics derided Trump's statement as mere formality The FDA's director, Gottlieb, has said solutions to emergencies could include withdrawing more products from the market, reducing the number of opioid prescriptions and requiring opioid education for doctors Gottlieb told the house energy and Commerce Committee in October: "we are facing such a huge crisis that the actions that need to be taken will be difficult." Original link: https://cen.acs.org/articles/95/i48/year-in-pharma-2017.html