Analysis: Large pharma companies do little new drug innovation - STAT
Analysis: Large pharma companies do little new drug innovation - STAT |
- Analysis: Large pharma companies do little new drug innovation - STAT
- Gene Therapy Takes Center Stage at 2019 NORD Summit - Pulmonary Hypertension News
- People Living With Type 2 Diabetes Want Information and Empathy From Their Doctors More Than New Medications - PRNewswire
| Analysis: Large pharma companies do little new drug innovation - STAT Posted: 10 Dec 2019 01:51 AM PST  Large pharmaceutical companies oppose legislation being considered by Congress to lower the prices of prescription drugs. Reducing their revenues, they contend, will reduce their investment in drug development and the discovery of new medicines, and thus lead to a decline in drug innovation. If that argument is credible, there should be evidence to show that the large pharmaceutical companies are responsible for discovering innovative new drugs. To test that claim, we examined the provenance of the highest-selling prescription medicines of Pfizer and Johnson & Johnson, the two largest pharmaceutical and biotechnology companies in 2018. advertisement We found that these large pharmaceutical companies did not actually invent most of the drugs they sell. Indeed, it appears they have already reduced their investment in the discovery of new medicines to the point where the threat of additional reductions rings hollow and is no longer a persuasive reason for opposing legislation to lower drug prices. Pfizer's and J&J's annual reports identify the medications that account for most of each company's sales of prescription drugs. We gathered information on the discovery and early development of these products from peer-reviewed publications, media reports, and company press releases. We scoured the companies' 2017 annual reports. A total of 62 products — 44 from Pfizer and 18 from J&J — were listed in them. The discovery and early development work were conducted in house for just 10 of Pfizer's 44 products (23%), as listed in Table 1. Only two of J&J's 18 leading products (11%) were discovered in house, as shown in Table 2. For example, sildenafil, the phosphodiesterase inhibitor that is the active compound in the erectile dysfunction drug Viagra and the pulmonary hypertension drug Revatio, was synthesized at Pfizer in the 1980s, originally as a cardiovascular medicine. Research leading to the development of risperidone (Risperdal), one of several newer-generation atypical antipsychotic drugs, began at J&J in the 1980s. The majority (81%) of other products were discovered and initially developed by third parties. Some of them came to Pfizer and J&J from the acquisition of other pharmaceutical companies. For example, Pfizer's highest-selling product, Prevnar 13, a vaccine for pneumococcal disease, was developed at Wyeth, which Pfizer acquired in 2009. Pfizer's palbociclib (Ibrance), used to treat breast cancer, had its origins at Warner-Lambert and Onyx Pharmaceuticals. J&J's rivaroxaban (Xarelto), an anticoagulant, originated at Bayer. Research leading to the discovery and development of other Pfizer and J&J drugs originated in universities and academic centers. J&J's highest-selling product, infliximab (Remicade), is a monoclonal antibody that was synthesized by researchers at New York University in 1989 in collaboration with the biotechnology company Centocor. The original work showing its efficacy in rheumatoid arthritis was led by Marc Feldmann and Ravinder Maini at Imperial College London. Etanercept (Enbrel), tofacitinib (Xeljanz), darunavir (Prezista), and daratumumab (Darzalex) are other products for which key discovery or development steps occurred in academic settings. The 34 Pfizer products discovered by third parties accounted for 86% of the $37.6 billion in revenue that its 44 leading products generated. The 16 J&J products invented elsewhere accounted for 89% of the $31.4 billion that its 18 leading products generated. Clearly, the existence of Pfizer and J&J as profitable pharmaceutical manufacturers is dependent on the acquisition of drugs invented by third parties. Our finding that few of the top-selling drugs made by Pfizer and J&J had been discovered in-house complements a recent Government Accounting Office report examining where large pharmaceutical companies spend most of their research dollars. It is also consistent with the latest member survey conducted by PhRMA, which indicated that last year only $13 billion was spent on preclinical studies — the basic and translational science that is the foundation for the discovery of innovative drugs. That is only a fraction of the $39.2 billion taxpayers spent to support the medical research conducted by the National Institutes of Health. More than 80% of the NIH's funding is awarded through almost 50,000 competitive grants to more than 300,000 researchers at 2,500+ universities, medical schools, and other research institutions in every state and around the world. While it is important to give fair consideration to the cost and risk involved in the development of new drugs, Pfizer and J&J were mostly buying drugs that had already been shown to have efficacy. The lack of in-house innovation at Pfizer and J&J is relevant to current efforts in the Senate (S. 2543) to limit annual drug price increases to the rate of inflation, and in the House of Representatives (H.R. 3) to cap drug price increases and limit prices based on what is charged for the same drug in other developed countries. Large pharmaceutical manufacturers have claimed that enactment of this legislation would be an "innovation killer" and trigger a "nuclear winter for the U.S. biopharmaceutical ecosystem." And President Trump tweeted late last month that the Pelosi drug pricing bill "doesn't do the trick. FEWER cures! FEWER treatments!"
If our findings are representative of the level of innovation at other large pharmaceutical manufacturers, a reduction in pharmaceutical revenues would not have the supposed devastating impact on the level of biopharmaceutical innovation. Rather, a reduction in revenues as a result of lower drug prices may reduce the astronomical acquisition prices now being paid by the large manufacturers to acquire innovations made by others. But the biopharmaceutical ecosystem will continue to thrive as long as those who actually innovate are provided with the resources to do so while those who play other roles in bringing new drugs to market are fairly compensated for their contributions to those aspects of the development process. As a recent report from the National Academies of Medicine concluded, "drugs that are not affordable are of little value and drugs that do not exist are of no value." The problem of affordability will not be solved if Congress continues to succumb to questionable assertions by lobbyists claiming that excessively high drug prices are essential to maintaining biopharmaceutical innovation. Passage of legislation to curb ridiculously high medication prices and price increases will not only make medicines more accessible to patients but will also reduce government expenditures on drugs by more than $345 billion dollars over 10 years, according to the Congressional Budget Office. That will enable the government to make greater investments in NIH and produce an even more robust biomedical innovation ecosystem than now exists. Emily H. Jung is a first-year medical student at Emory School of Medicine in Atlanta and a former research assistant at the Program On Regulation, Therapeutics, And Law (PORTAL) in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women's Hospital. Alfred Engelberg, J.D., is a retired pharmaceutical intellectual property attorney and philanthropist. Aaron S. Kesselheim, M.D., is a professor of medicine at Harvard Medical School and director of PORTAL. Funding for this work was provided by the Engelberg Foundation, a charitable foundation that focuses on health policy research. Kesselheim's work is also supported by the Harvard-MIT Center for Regulatory Science and Arnold Ventures.
* Origins listed for each drug based on methods described in article and do not exclude the possibility of contributions from other scientists or organizations.
* Origins listed for each drug based on methods described in article and do not exclude the possibility of contributions from other scientists or organizations.  | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Gene Therapy Takes Center Stage at 2019 NORD Summit - Pulmonary Hypertension News Posted: 10 Dec 2019 08:00 AM PST With so much recent publicity surrounding gene therapy, it's no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high costs and limitless potential for curing diseases — generated much discussion at the Washington, D.C., event, which was organized by the National Organization for Rare Disorders (NORD) and attended by more than 900 delegates Oct. 21–22.  "On one hand, this is a splendid time to be doing what we do," said Scott Gottlieb, MD, former commissioner of the U.S. Food and Drug Administration (FDA), and now a partner at Washington-based New Enterprise Associates. "On the other hand, it's imperative that all patients have access [to these new therapies], regardless of someone's ability to afford them. Many of the treatments are so dramatic, they can quite literally alter the destiny of someone's life. He regarded his leadership of the agency, from May 2017 to April 2019, as "a wonderful time at the FDA because we had the benefit of cures and new money as a tailwind, but also in large measure because of the opportunities before us — such as gene therapy and CRISPR — to fundamentally alter diseases." Gottlieb's comments come toward the end of a year that saw the FDA approve the Novartis treatment Zolgensma, the first gene therapy for spinal muscular atrophy (SMA). With a price tag of $2.1 million, it's the most expensive medication in history. "When it comes to curative therapies, it's not that we can't sustain the cost of a million-dollar therapy if it means curing a pediatric disorder. We can afford that," he said. "But what we can't afford is to let an insurance company determine your child's destiny." While there's a "real sense of excitement" about gene therapy for illnesses such as hemophilia, SMA, sickle cell disease, and Duchenne muscular dystrophy, these expensive new treatments may actually accentuate rather than reduce disparities among patients, said Steven Pearson, MD, founder and president of the Boston-based Institute for Clinical and Economic Review. "There's a real anxiety that this wave of innovation will lead to unexpected high costs," he said. "Insurance companies are writing policies today that will exclude gene therapies. That's ridiculous. It doesn't help anybody." Fair pricing strategiesFair pricing strategies are badly needed to ensure that all who need future gene therapies will have access to them, regardless of the cost, said Pearson, whose independent, nonpartisan agency objectively evaluates the clinical and economic value of prescription medications. "We could just give this to the federal government and create risk pools, but I think the private market can sort this out," he said. "A fair price should never mean that a patient should have to repeat their genetic tests every six months to show they still have the same bloody condition."  During a panel, titled "Gene Therapy: Overcoming Challenges for Both Patients and Manufacturers," pharmaceutical executives and patient advocates discussed what the coming scientific revolution means for them. Gene therapy offers the possibility for kids with devastating conditions and severely limited life spans to live long, productive lives and fulfill their potential, said Mark Rothera, president and CEO of London-based Orchard Therapeutics, who moderated the panel. Rothera said he's worked in rare diseases since the late 1980s, and has launched seven orphan drugs in his career. "But I joined Orchard a few years ago because I've never seen data like this before. It's taken 20 or 30 years of work to get to this point," he said. "The whole system is geared up for chronic long-term therapy. Now we're talking about one single intervention that will deliver lifelong benefits, so the system has to welcome that and adapt to it." The panel included Olivier Danos, PhD, senior vice president and chief scientific officer at Regenxbio; Neil Horikoshi, CEO and executive director of the Aplastic Anemia & MDS International Foundation; Stephanie Uder, vice-president of product management at Accredo; and Amy Price, outreach coordinator at the Leukodystrophy Family Forum. "You can imagine the enormous financial and emotional toll that 24/7 care takes on moms and dads," Rothera said. "Maybe one of the parents has to give up a job to be there. Imagine if that's no longer required, because the parents have a healthy child, and can use that time and energy to look after healthy children." Gene therapy for ADATo that end, Orchard is focused solely on treating children with primary immune deficiencies, neurometabolic disorders, and hemoglobinopathies — most of which lack treatments today. The company's pipeline includes Strimvelis, the first ex vivo autologous gene therapy to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA) for whom no suitable stem cell donor is available. Approved by the European Medicines Agency in 2016, Strimvelis was acquired by Orchard from GlaxoSmithKline in March 2018.  ADA affects about 15 people per year in Europe and 12 per year in the United States. Strimvelis retails for $780,000, compared to $4.25 million for 10 years of weekly enzyme replacement therapy. "We're building a company on a global scale to bring these medicines to patients around the world as quickly as we can, but we face plenty of challenges with patient identification," Rothera said. "When you're bringing gene therapy for the first time in the world to a community that hasn't had treatment, there's been less of an effort at diagnosis." "But time also matters," he added. "Some patients, you want to treat as early as possible before symptoms develop — so newborn screening is very important." Rothera also noted the complicated logistics of bringing patients from all over the world to a specific hospital to receive gene therapy. "Imagine the case of a child born with a rare disease that has been cured through one single intervention," he said. "Effectively, that child is no longer a patient — yet will need a further 15 years of follow-up. A procedure like this requires exquisite coordination. The patient's own cells become the product." Flying abroad for gene therapyAmy Price knows about the complicated logistics of traveling across the ocean to cure a child. She and her husband, Brad, have three children with metachromatic leukodystrophy (MLD). The disease affects white matter in the brain, causing progressive loss of mobility and sensation as well as intellectual decline.  Their daughter, Liviana, was born in January 2008 and diagnosed with MLD in December 2010. Giovanni was born in January 2010. After genetic testing revealed that he also had the disease, the boy was accepted into a clinical trial at Italy's San Raffaele Telethon Institute for Gene Therapy in Milan. He was treated in 2011, though it was too late for Liviana, who died in 2013. The following year, Price gave birth to triplets. One of them, Cecilia, inherited MLD and got the gene therapy in Italy at 9 months old. According to a post in the PLOS Blogs Network, the gene therapy that Giovanni and Cecilia received "adds corrected genes for the enzyme arylsulfatase A to bone marrow stem cells taken from the children. The cells are infused and migrate to the brain, where they give rise to corrected glial cells that make the necessary enzyme. Done early enough, symptoms never begin." A psychologist, Price recently interviewed 16 families whose children — 12 with MLD — underwent gene therapy treatments, mostly between 2011 and 2016. Half of the children was American and the other half was European. Their ages ranged from 8 months to 11 years, with an average age of 3 years. She found that the most formidable challenges for families were fear of unknown or long-term treatment outcomes; family separation; having to care for other children at the same time; social isolation; unfamiliar language or customs; and financial well-being. "It seems like such a small thing, but if you don't have WiFi and nobody speaks your language, it's a cumulative effect," said Price, noting the emotional and physical stress of travel logistics, flying, and general exhaustion on the patients and their families. Among her suggestions:
"There is so much unknown with a new treatment, and the patients make an immeasurable and priceless sacrifice of hope," said Price. But even so, she added, "it's a life-or-death choice, and 100% [of respondents] said they would do it again."  ×  | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Posted: 10 Dec 2019 12:11 PM PST The issue is not a lack of understanding about the importance of lifestyle changes. The gap is in getting practical tips for successfully adopting lifestyle changes -- especially in the context of dealing with the wide-ranging impact diabetes has on daily life. MyHealthTeams identified two key areas in which people living with diabetes know they want to improve - but aren't sure what to do or how to start:
Quality of Life Impact Beyond high blood sugar, people with Type 2 Diabetes report experiencing a wide range of symptoms of the disease in the past year, including:
The impact of diabetes on daily life is significant, with survey respondents reporting top challenges including:
"What's clear is that managing blood sugar is just one piece of the diabetes puzzle, and people living with this condition are juggling a lot," said Eric Peacock, cofounder and CEO of MyHealthTeams. "The call to action across the healthcare ecosystem is to empower consumers with information and support to act as their own health advocates within this context. People need practical advice and emotional empathy. It's about much more than medicine." This research was conducted among the more than 100,000 registered members of DiabetesTeam. 478 individuals responded to the online survey. Full survey findings are available at https://www.diabetesteam.com/resources/the-results-are-in-what-people-with-type-2-diabetes-want-most-from-their-doctors-is-information-not-new-treatments. About MyHealthTeams SOURCE MyHealthTeams  Related Links |
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