Eli Lilly research focuses on patient needs and partnerships

Participatory Partnering

Emily Kramer-Golinkoff, who was  trained as a bioethicist from the University of Pennsylvania, is “the engine behind a multi-level approach to changing the game for the advancement of treatments for patients with certain protein production mutations of Cystic Fibrosis (CF) called nonsense mutations – all while living with the condition 24/7,” according to an article by Joe Kim in Lilly Trial Guide. Kramer-Golinkoff provides funding for many phases of scientific research, advocates for patients with Cystic Fibrosis, participates in clinical research, influences the design of research, connects disparate scientific fields and scientists and inspires action through her public speaking, Kim said.

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Study merges clinical research investigators data from three government databases

Complete Picture

The three primary U.S. government sources of data about clinical research investigators -- National Library of Medicine: ClinicalTrials.gov, FDA: Bioresearch Monitoring Information System (BMIS)and CMS: Open Payments (Sunshine Act) -- provide incomplete and inconsistent data not linked to data in the other databases. While the FDA is aware of the problems, researchers have used the databases to understand investigator demographics and activities.

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FDA steps up in regulating safety concerns

Data-Driven Progress

The U.S. Food and Drug Administration (FDA) has established a new objective for itself: “to become the international leader in medical device safety through better use of big data,” according to an article by Conor Hale in FiercePharma. The agency has made a commitment to quick device safety responses “using real-world data and active surveillance,” Hale explained.

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Psychedelic mushroom component could be used medicinally

Far Out!

Depending on who is administering it and how it is being used, psilocybin, the active compound in hallucinogenic mushrooms, could be reclassified from a drug with no known medical benefit to a drug with the properties of prescription sleeping pills. In a review to assess the safety and abuse of medically administered psilocybin, researchers at Johns Hopkins University made a case for doing just that, possibly opening up possibilities for the psychedelic drug to be used to treat depression and anxiety and help people stop smoking.

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Analyst says blockchain technology could help clinical trials

Blockchain Bonanza

Caron Dhillon, a health care analyst at Results Healthcare, believes that the use of blockchain technology could solve many of the challenges of the clinical trials process, according to an article in Outsourcing Pharma by Melissa Fassbender.

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Data firm ranks top wearables for clinical research

Litmus Health, a real-world data firm, has created a census of wearable devices for clinical research. The firm is profiling the body and wrist-worn hardware devices from more than 190 brands and manufacturers, according to an article by Conor Hale in FiercePharma.

The company recommends its 15 top devices based on a rubric that scores the transparency of data collection, battery life and aesthetic appeal for study participants. It also looks at factors such as how well it helps investigators to meet federal regulations for maintaining electronic records and audit trails. Litmus is planning to provide quarterly updates to the public report, with emphases on different types of sensors.

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New company targets CNS disorders

Brain Biopharma

US pharma giant Pfizer and private equity firm Bain Capital have joined forces to create  Cerevel Therapeutics, a new privately held biopharmaceutical company. The new company aims to develop drugs to treat central nervous system (CNS) disorders. In addition to securing $350 million from funds affiliated with Bain Capital Private Equity and Bain Capital Life Sciences, there will be further capital available if needed in the future.

New York-based Pfizer, which will hold a 25 percent stake in Cerevel Therapeutics, is providing a portfolio of pre-commercial neuroscience assets, including three clinical-stage compounds and various pre-clinical compounds to target multiple CNS disorders such as Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction. The new company is designed to provide treatment options in a therapeutic area with urgent unmet needs. Two Pfizer executives – Morris Birnbaum, chief scientific officer of internal medicine, and Doug Giordano, senior vice president of worldwide business development – are part of Cerevel’s board of directors.

According to an article by Alaric Dearment in MedCity News, “The current pipeline, consisting of drugs created by Pfizer, includes three clinical-stage candidates – a D1 partial agonist for Parkinson’s, a GABA 2/3 agonist for epilepsy and a muscarinic M4 positive modulator – and several preclinical ones. Modulation of M4 msucarinic receptors has been investigated as a potential mechanism of action for use in schizophrenia. The preclinical candidates include a dopamine D3 antagonist and a kappa opiate antagonist in candidate-stage development, and an M4 agonist, a PDE4b inhibitor and an LRRK2 inhibitor in lead development.”

Pfizer said that putting the set of neuroscience assets in a company with dedicated focus and expertise in CNS was the optimal next step after halting its own research in CNS in January, according to Pharmaceutical Business Review. After stopping that research, Pfizer announced in June that it had bolstered the funding pool of its venture capital arm by $600 million, one-quarter of which was devoted to neuroscience. As Pfizer senior vice president, internal medicine chief scientific officer Morris Birnbaum explained, “We are excited that Cerevel will continue to develop the Pfizer compounds, contributing to the broad scientific understanding of both Alzheimer’s and Parkinson’s and fulfilling responsibility to patients with these devastating diseases and their families.”

As Bain Capital Life Sciences managing director Adam Koppel stated, “We are dedicated to developing this promising portfolio of assets thoughtfully and will provide the right resources to enable Cerevel to build an experienced leadership team that is committed to developing life changing therapies for patients who are struggling with CNS diseases. The broad portfolio we are investing behind consists of several clinical candidates with unique chemistry to enable specific receptor targeting, which has the potential to drive a differentiated clinical profile.”

Penn study gives hospitals bad marks

A recent study led by University of Pennsylvania nursing professor Linda Aiken has found that hospitals have improved somewhat from the findings of a landmark report from the Institute of Medicine 20 years ago that determined that thousands of patients die in hospitals each year from preventable medical errors. Still, according to a new issue of Health Affairs devoted to that topic, various studies show that hospitals have more work to do.

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Inspection prompts FDA shutdown: Ketamine Trial Cut Off

In July a consumer rights group, Public Citizen, with 64 doctors, bioethicists and academics, called on the US Food and Drug Administration (FDA) to investigate the Hennepin County Medical Center in Minnesota because of concerns involving informed consent. What resulted was the suspension of a clinical trial, Ketamine Versus Midazolam for Prehospital Agitation.

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New cloud-based Parexel technology provides better patient interactions

Meaningful Data

Leveraging its relationship with Microsoft to assist with drug development, Parexel has introduced the world to several new patient-centric solutions that utilize its Perceptive Cloud platform. The patient-centric solutions for drug development feature mobile and wearable tech to make access to data for better patient interactions during clinical research faster and better, the company explained.

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Milestone procures $80M to fund at-home phase 3 trial of tachycardia rescue nasal spray

Bringing It Home

Etripamil, a “short-acting calcium channel blocker being developed for paroxysmal supraventricular tachycardia, or PSVT, where the heart can beat over 200 times per minute,” is certainly exciting investors, according to an article by Conor Hale in Fierce Biotech. Milestone Pharmaceuticals of Montreal, Canada, has raised $80 million to fund its lead program, an at-home, phase 3 clinical trial of its nasal spray for rapidly reducing super high heart rates.

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Digital health startups need to analyze stakeholders

Cracking the Code

While some forms of high technology succeed using a “move fast and break things” approach, digital health startups have to go deeper into the requirements of the industry to “truly transform healthcare,” according to an article by Paul Yock, a cardiologist, health technology innovator, professor of medicine and bioengineering and founder of the Byers Center for Biodesign at Stanford University. Dr. Yock, writing in Fast Company (www.fastcompany.com/90251795/why-do-digital-health-startups-keep-failing, said that money and buzz are not enough to be successful in healthcare, “a much more complex and regulated industry.”

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Allogene begins Nasdaq trading at $18 per share

Living Drug

Allogene Therapeutics, a clinical stage biotech company specializing in allogeneic CAR-T (AlloCAR T) treatments for cancer, started trading on the Nasdaq Stock Exchange at $18 per share. The company, which launched in April 2018 with a $300 million Series A financing, is expecting to raise $288 million by selling about 16 million shares, reported Mark Terry in BioSpace.

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Memorial Sloan Kettering’s Stake in Startup Raises Concerns

More Entanglements?

Memorial Sloan Kettering Cancer Center – one of the nation’s most prestigious institutions – is in the news again. In a New York Times article by Charles Ornstein and Katie Thomas that was reported and written in a collaboration with ProPublica, the nonprofit investigative journalism organization, it is explained that a startup in which the hospital has a financial stake is raising red flags among the institution’s pathologists. The dissension comes on the heels of another ethical question.

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DSCS announces “the most practical clinical research conference ever created”

CliniCon19

DSCS Sweat Equity & Investments, LLC, has designed a value-added yearly conference for members of the clinical research community. The company describes it as “what a clinical research conference should be: site-centric, individually focused while catering towards experienced and novice clinical researchers alike, packed with practical and implementable strategies that are proven to produce results, and facilitating networking through arranged "speed dates" at set times.” The conference will be extended online to keep attendees connected with DSCS and each other and to provide “new insights into the industry as they occur in real time.”  

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Veeva announces single app to manage clinical trial data

Cloud Tool

Veeva Systems Inc. -- a leader in cloud-based software for the global life sciences industry -- is offering a new cloud-based application that combines several of its tools. It includes coding, EDC, data cleaning and reporting. Enabling companies to manage clinical trials from build through execution, the new app is designed to achieve a complete and concurrent view of all clinical data within a trial, according to the company.

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FDA suggests heavy fines for not posting clinical trial results online

Transparency 101

The U.S. Food & Drug Administration (FDA) has issued a draft guidance statement proposing heavy fines for pharmaceutical companies and clinical research organizations (CROs) that neglect to post clinical trial results online. According to the statement, the FDA wants to fine organizations that fail to register clinical trials or to report results on to the government database ClinicalTrials.gov as much as $10,000 per day.

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Data Cubed uses gaming technology to facilitate human interaction in clinical trials: Making a Game out of It

Basing its approach on popular computer games, Data Cubed has created a platform that could “increase patient engagement in clinical research,” according to an article by Melissa Fassbender in OutsourcingPharma.com. In 2016 when Data Cubed (D3) was founded, the company took advantage of several trends -- structured big data, mobile technologies, patient-centric care, and social networks, the article explained.

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Forbes contributing writer takes aim at Harvard professor who pans pharma

Disclosure Dilemma

Leading cancer researcher Dr. Jose Baselga, the former chief medical officer of Memorial Sloan Kettering Cancer Center, did not disclose income he received from biopharmaceutical companies in articles that he published in major medical journals and, as a result, resigned from his position. This prompted a scathing response from Dr. Marcia Angell, a senior lecturer at Harvard Medical School and the first woman to serve as editor-in-chief of The New England Journal of Medicine.

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