Feeds:
Posts
Comments

Archive for the ‘FDA Regulatory Affairs’ Category

Live Conference Coverage @Medcitynews Converge 2018 @Philadelphia: Promising Drugs and Breaking Down Silos

Posted in CANCER BIOLOGY & Innovations in Cancer Therapy, Cancer Prevention: Research & Programs, Clinical & Translational, Disease Biology, Small Molecules in Development of Therapeutic Drugs, FDA Regulatory Affairs, Health Economics and Outcomes Research, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Uncategorized, Value-based Drug Pricing, tagged , , , , , , , , , , , , , , , , , , , , , , , , on July 12, 2018| Leave a Comment »

Live Conference Coverage @Medcitynews Converge 2018 @Philadelphia: Promising Drugs and Breaking Down Silos

Reporter: Stephen J. Williams, PhD

Promising Drugs, Pricing and Access

The drug pricing debate rages on. What are the solutions to continuing to foster research and innovation, while ensuring access and affordability for patients? Can biosimilars and generics be able to expand market access in the U.S.?

Moderator: Bunny Ellerin, Director, Healthcare and Pharmaceutical Management Program, Columbia Business School
Speakers:
Patrick Davish, AVP, Global & US Pricing/Market Access, Merck
Robert Dubois M.D., Chief Science Officer and Executive Vice President, National Pharmaceutical Council
Gary Kurzman, M.D., Senior Vice President and Managing Director, Healthcare, Safeguard Scientifics
Steven Lucio, Associate Vice President, Pharmacy Services, Vizient

What is working and what needs to change in pricing models?

Robert:  He sees so many players in the onStevencology space discovering new drugs and other drugs are going generic (that is what is working).  However are we spending too much on cancer care relative to other diseases (their initiative Going Beyond the Surface)

Steven:  the advent of biosimilars is good for the industry

Patrick:  large effort in oncology, maybe too much (750 trials on Keytruda) and he says pharma is spending on R&D (however clinical trials take large chunk of this money)

Robert: cancer has gotten a free ride but cost per year relative to benefit looks different than other diseases.  Are we overinvesting in cancer or is that a societal decision

Gary:  maybe as we become more specific with precision medicines high prices may be a result of our success in specifically targeting a mutation.  We need to understand the targeted drugs and outcomes.

Patrick: “Cancer is the last big frontier” but he says prices will come down in most cases.  He gives the example of Hep C treatment… the previous only therapeutic option was a very toxic yearlong treatment but the newer drugs may be more cost effective and safer

Steven: Our blockbuster drugs could diffuse the expense but now with precision we can’t diffuse the expense over a large number of patients

President’s Cancer Panel Recommendation

Six recommendations

  1. promoting value based pricing
  2. enabling communications of cost
  3. financial toxicity
  4. stimulate competition biosimilars
  5. value based care
  6. invest in biomedical research

Patrick: the government pricing regime is hurting.  Alot of practical barriers but Merck has over 200 studies on cost basis

Robert:  many concerns/impetus started in Europe on pricing as they are a set price model (EU won’t pay more than x for a drug). US is moving more to outcomes pricing. For every one health outcome study three studies did not show a benefit.  With cancer it is tricky to establish specific health outcomes.  Also Medicare gets best price status so needs to be a safe harbor for payers and biggest constraint is regulatory issues.

Steven: They all want value based pricing but we don’t have that yet and there is a challenge to understand the nuances of new therapies.  Hard to align all the stakeholders together so until some legislation starts to change the reimbursement-clinic-patient-pharma obstacles.  Possibly the big data efforts discussed here may help align each stakeholders goals.

Gary: What is the data necessary to understand what is happening to patients and until we have that information it still will be complicated to determine where investors in health care stand at in this discussion

Robert: on an ICER methods advisory board: 1) great concern of costs how do we determine fair value of drug 2) ICER is only game in town, other orgs only give recommendations 3) ICER evaluates long term value (cost per quality year of life), budget impact (will people go bankrupt)

4) ICER getting traction in the public eye and advocates 5) the problem is ICER not ready for prime time as evidence keeps changing or are they keeping the societal factors in mind and they don’t have total transparancy in their methodology

Steven: We need more transparency into all the costs associated with the drug and therapy and value-based outcome.  Right now price is more of a black box.

Moderator: pointed to a recent study which showed that outpatient costs are going down while hospital based care cost is going rapidly up (cost of site of care) so we need to figure out how to get people into lower cost setting

Breaking Down Silos in Research

“Silo” is healthcare’s four-letter word. How are researchers, life science companies and others sharing information that can benefit patients more quickly? Hear from experts at institutions that are striving to tear down the walls that prevent data from flowing.

Moderator: Vini Jolly, Executive Director, Woodside Capital Partners
Speakers:
Ardy Arianpour, CEO & Co-Founder, Seqster @seqster
Lauren Becnel, Ph.D., Real World Data Lead for Oncology, Pfizer
Rakesh Mathew, Innovation, Research, & Development Lead, HealthShareExchange
David Nace M.D., Chief Medical Officer, Innovaccer

Seqster: Seqster is a secure platform that helps you and your family manage medical records, DNA, fitness, and nutrition data—all in one place. Founder has a genomic sequencing background but realized sequence  information needs to be linked with medical records.

HealthShareExchange.org :

HealthShare Exchange envisions a trusted community of healthcare stakeholders collaborating to deliver better care to consumers in the greater Philadelphia region. HealthShare Exchange will provide secure access to health information to enable preventive and cost-effective care; improve quality of patient care; and facilitate care transitions. They have partnered with multiple players in healthcare field and have data on over 7 million patients.

Innovacer

Data can be overwhelming, but it doesn’t have to be this way. To drive healthcare efficiency, we designed a modular suite of products for a smooth transition into a data-driven world within 4 weeks. Why does it take so much money to move data around and so slowly?

What is interoperatibility?

Ardy: We knew in genomics field how to build algorithms to analyze big data but how do we expand this from a consumer standpoint and see and share your data.

Lauren: how can we use the data between patients, doctors, researchers?  On the research side genomics represent only 2% of data.  Silos are one issue but figuring out the standards for data (collection, curation, analysis) is not set. Still need to improve semantic interoperability. For example Flatiron had good annotated data on male metastatic breast cancer.

David: Technical interopatabliltiy (platform), semantic interopatability (meaning or word usage), format (syntactic) interopatibility (data structure).  There is technical interoperatiblity between health system but some semantic but formats are all different (pharmacies use different systems and write different prescriptions using different suppliers).  In any value based contract this problem is a big issue now (we are going to pay you based on the quality of your performance then there is big need to coordinate across platforms).  We can solve it by bringing data in real time in one place and use mapping to integrate the format (need quality control) then need to make the data democratized among players.

Rakesh:  Patients data should follow the patient. Of Philadelphia’s 12 health systems we had a challenge to make data interoperatable among them so tdhey said to providers don’t use portals and made sure hospitals were sending standardized data. Health care data is complex.

David: 80% of clinical data is noise. For example most eMedical Records are text. Another problem is defining a patient identifier which US does not believe in.

 

 

 

 

Please follow on Twitter using the following #hash tags and @pharma_BI

#MCConverge

#cancertreatment

#healthIT

#innovation

#precisionmedicine

#healthcaremodels

#personalizedmedicine

#healthcaredata

And at the following handles:

@pharma_BI

@medcitynews

Read Full Post »

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

Posted in Cancer Informatics, Cancer Prevention: Research & Programs, Cancer Screening, Diagnostics and Lab Tests, FDA Regulatory Affairs, Genetics & Innovations in Treatment, Genetics & Pharmaceutical, Genome Biology, Genomic Expression, Genomic Testing: Methodology for Diagnosis on March 23, 2018| Leave a Comment »

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

 

Reporter: Aviva Lev-Ari, PhD, RN

genetic testing just became routine care for patients with advanced cancers. And that means precision medicine has finally broken into the mainstream.

Any tests that gain FDA clearance in the future will automatically receive full coverage.

In 3/2018 there are three FDA approved Genetic Tests for Cancer:

UNDER development and not included in the agreement , above, includes:

  • Olivier Elemento, Director of the Caryl and Israel Englander Institute for Precision Medicine at Cornell, the team at Cornell, for example, has developed a whole exome test that compares mutations in tumors against healthy cells across 22,000 genes. To date, it’s been used to help match more than 1,000 patients in New York state with the best available treatment options.

Under the final decision, doctors are still free to order non-FDA approved tests, but coverage isn’t guaranteed; each case will be evaluated by local Medicare administrative contractors. Which means Elemento’s test could still be covered. “To me this is a vote of confidence that next generation sequencing is useful for cancer patients,” says Elemento.

So far, CMS is only covering these tests for stage three and stage four metastatic cancer sufferers. Most of them aren’t going to be cured. They might get a few more good months, maybe a year, tops.

Cancerous Genes

SOURCE

WITH MEDICARE SUPPORT, GENETIC CANCER TESTING GOES MAINSTREAM

https://www.wired.com/story/with-medicare-support-genetic-cancer-testing-goes-mainstream/?mbid=social_twitter_onsiteshare

Read Full Post »

FDA: Rejects NDA filing: “clinical and non-clinical pharmacology sections of the application were not sufficient to complete a review”: Celgene’s Relapsing Multiple Sclerosis Drug – Ozanimod

Posted in FDA Regulatory Affairs, Innovations in Neurophysiology & Neuropsychology, Relapsing Multiple Sclerosis, tagged on February 28, 2018| Leave a Comment »

FDA: Rejects NDA filing: “clinical and non-clinical pharmacology sections of the application were not sufficient to complete a review”: Celgene’s Relapsing Multiple Sclerosis Drug – Ozanimod

Reporter: Aviva Lev-Ari, PhD, RN

 

Celgene Provides Regulatory Update on Ozanimod for the Treatment of Relapsing Multiple Sclerosis

Conference call scheduled for today at 5:30 p.m. ET

SUMMIT, N.J.–(BUSINESS WIRE)– Celgene Corporation (NASDAQ:CELG) today announced that it has received a Refusal to File letter from the United States Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for ozanimod in development for the treatment of patients with relapsing forms of multiple sclerosis. Ozanimod is a novel, oral, selective sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator.

Upon its preliminary review, the FDA determined that the nonclinical and clinical pharmacology sections in the NDA were insufficient to permit a complete review. Celgene intends to seek immediate guidance, including requesting a Type A meeting with the FDA, to ascertain what additional information will be required to resubmit the NDA.

“We remain confident in ozanimod’s clinical profile demonstrated in the pivotal program in relapsing forms of multiple sclerosis,” said Jay Backstrom, M.D., Chief Medical Officer and Head of Global Regulatory Affairs for Celgene. “We will work with the FDA to expeditiously address all outstanding items and bring this important medicine to patients.”

Conference Call Information

Celgene will hold a conference call to discuss this update today at 5:30 p.m. ET. The conference call may be accessed by dialing 1-866-428-9517 for U.S.callers and 1-224-357-2194 for international callers. The passcode for the call is 9179457. The call can also be accessed via an audio webcast in the Investor Relations section of the company website at www.celgene.com. An audio replay will be available through March 6, 2018 by calling 1-855-859-2056 or 1-404-537-3406 and entering access code 9179457.

About Ozanimod

Ozanimod is a novel, oral, selective, sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator in development for immune-inflammatory indications, including relapsing multiple sclerosis, ulcerative colitis and Crohn’s disease. Selective binding with S1PR1 is believed to inhibit a specific sub set of activated lymphocytes from migrating to sites of inflammation. The result is a reduction of circulating T and B lymphocytes that leads to anti-inflammatory activity. Importantly, immune surveillance is maintained.

Selective binding with S1PR5 is thought to activate specific cells within the central nervous system (CNS). This has the potential to enhance remyelination and prevent synaptic defects. Ultimately, neurological damage may be prevented.

Ozanimod is an investigational compound that is not approved for any use in any country.

About Celgene

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next‐generation solutions in protein homeostasis, immuno‐oncology, epigenetics, immunology and neuro‐inflammation. For more information, please visit www.celgene.com. Follow Celgene on Social Media: @Celgene, Pinterest, LinkedIn, Facebook and YouTube.

Forward-Looking Statements

This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the U.S. Securities and Exchange Commission.

Hyperlinks are provided as a convenience and for informational purposes only. Celgene bears no responsibility for the security or content of external websites.

Celgene
Investors:
Patrick E. Flanigan III, 908-673-9969
Corporate Vice President, Investor Relations
or
Media:
Catherine Cantone, 908-897-4256
Senior Director, Corporate Communications

Source: Celgene Corporation

SOURCE

http://ir.celgene.com/releasedetail.cfm?ReleaseID=1058943

 

DIVE INTERPRETATION

Celgene’s ozanimod hit with Refusal to File in latest

Dive Brief:

  • Celgene Corp. disappointed investors Tuesday night when the big biotech unexpectedly disclosed a Refusal to File letter from the Food and Drug Administration for its New Drug Application (NDA) for the multiple sclerosis drug ozanimod.
  • The FDA said the clinical and non-clinical pharmacology sections of the application were not sufficient to complete a review. Celgene intends to request a type A meeting with the agency to better understand what is required for filing.
  • While the company did not give exact guidance, analysts expect this could push back launch timelines of ozanimod by as much as a year, shifting the drug launch from 2018 to 2019.

Ozanimod, an oral, selective sphingosine 1-phosphate 1 (S1PR1) and 5 (S1PR5) receptor modulator being studied in multiple sclerosis, ulcerative colitis and Crohn’s disease, was meant to be a key driver for Celgene in the post-2020 time period. Some analysts had previously projected peak sales above $2.75 billion for the drug, but Mizuho analyst Salem Syed speculates this could jeopardize as much as $1 billion in sales.

The big biotech has been struggling to diversify its pipeline and has been in desperate need of a win. It already reported an expensive failure in October 2017 for the Crohn’s disease drug that it acquired for $710 million in 2014. And the patent situation around its blockbuster Revlimid (lenalidomide) has been shaky at best, threatening nearly $8.1 billion in revenues. Celgene made two major acquisitions in the first weeks of the year, picking up Impact Biomedicines for $7 billion and then Juno Therapeutics Inc. for $9 billion — but neither acquisition seems enough to stop the bleeding.

Investors were not pleased with the news, with the stock dropping nearly 8% in morning trading on Wednesday to about $89 per share. When Mark Alles took over as CEO in January 2016, the stock was trading above $100 apiece.

“This is yet another blow to investor (and analyst) confidence in this management team and their stewardship of the company’s key operational activities,” wrote Leerink Partners Geoffrey Porges in a note to clients.

Alles said on a Tuesday evening call with analysts said the recent acquisition and near-term catalysts from Juno’s lead drug JCAR17 would give the company the buffer it needs through 2020, and that Celgene is reaffirming its sales guidance for the 2020 time period.

Yet, the company wasn’t able to give much clarity on how this will impact the ozanimod filing in MS and said it will likely create delays with its European filing as well. Analysts worried that the filings of ozanimod for ulcerative colitis and Crohn’s could be delayed — although Alles noted the ulcerative colitis program would not be affected at this time.

“We certainly appreciate the events of the last eight months have caused investors a lot of concern and that is reflected in the value of the company,” said Alles on the call. “The complexity and some of the disappointments make it difficult to be predicting our future,” he added, but emphasized the company would bring in $19–20 billion in topline revenues in 2020.

Refusal to File letters were not a commonly used tool by the FDA, but the new administration under Scott Gottlieb seems to be tapping the tool more often of late.

Several other companies have gotten a no-go on applications recently as well, including Acorda Therapeutics for its Parkinson’s disease drug, Allergan for schizophrenia drug Vraylar (cariprazine), and SteadyMed Ltd. for a blood pressure medication. While the letters are not considered a rejection, they often delay applications and have derailed other drugs in the past.

SOURCE

Read Full Post »

In 2017, FDA approved a record number of 19 personalized medicines — 16 new molecular entities and 3 gene therapies – PMC’s annual analysis, titled Personalized Medicine at FDA: 2017 Progress Report

Posted in FDA Regulatory Affairs, Personalized and Precision Medicine & Genomic Research on January 30, 2018| Leave a Comment »

In 2017, FDA approved a record number of 19 personalized medicines — 16 new molecular entities and 3 gene therapies – PMC’s annual analysis, titled Personalized Medicine at FDA: 2017 Progress Report

Reporter: Aviva Lev-Ari, PhD, RN

 

 

Contact: Christopher J. Wells

Personalized Medicine Coalition

cwells@personalizedmedicinecoalition.org

202-580-9780

FOR IMMEDIATE RELEASE

 

FDA Approves Record Number of Personalized Medicines, Spearheads Six Regulatory Precedents in Field in 2017

Personalized Medicines Now Account for More Than One in Four New Drug Approvals

WASHINGTON (January 30, 2018) — The Personalized Medicine Coalition (PMC) today released a report documenting the record number of new personalized medicines the U.S. Food and Drug Administration (FDA) approved last year, making 2017 the fourth consecutive year that personalized medicines accounted for more than 20 percent of all new drug approvals.

The annual analysis, titled Personalized Medicine at FDA: 2017 Progress Report, shows that FDA approved a record number of 19 personalized medicines — 16 new molecular entities and three gene therapies — in 2017. The report lists a total of six regulatory precedents FDA set last year, as follows:

  1. Record number of 16 personalized medicines approved as new molecular entities
  2. Approval of first three gene therapies
  3. First approval of a tissue agnostic indication for cancer therapy
  4. First authorization for marketing of health-related genetic tests directly to consumers
  5. First approval of a personalized medicine biosimilar
  6. First FDA/CMS joint approval and coverage decision for a next-generation sequencing test

PMC President Edward Abrahams, Ph.D., said the precedents demonstrate how personalized medicine has reshaped drug development in the decade since 2007, when targeted therapies accounted for less than 10 percent of new drug approvals. An influential article published in 2007 in the Harvard Business Review titled “Realizing the Promise of Personalized Medicine,” for example, suggested that FDA was not yet committed to the paradigm. The pharmaceutical industry, the article noted, was at that time hesitant to develop medicines for smaller patient populations, preferring instead to develop “blockbuster” medications that could earn approval for one-size-fits-all applications.

This obviously is no longer true, though there remain many obstacles — notably regarding regulation, reimbursement, access and clinical adoption — that complicate the commercialization of personalized medicine products.

“Despite myriad challenges, the diagnostic and pharmaceutical industries are deeply invested in making health care more effective and efficient by developing products that guide treatments to only those patients who will benefit from them,” Abrahams explained. “As this report shows, FDA is increasingly committed to supporting that effort.”

Laura Koontz, Ph.D., Personalized Medicine Staff Member at FDA, will discuss FDA’s direction in personalized medicine with PMC members during its next Policy Committee Meeting on February 20, 2018.

###

About the Personalized Medicine Coalition:
The Personalized Medicine Coalition, representing innovators, scientists, patients, providers and payers, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. For more information, please visit www.personalizedmedicinecoalition.org.

 

SOURCE

From: Personalized Medicine Coalition <messages@app.production.membersuite.com>

Reply-To: “Christopher Wells (PMC)” <cwells@personalizedmedicinecoalition.org>

Date: Tuesday, January 30, 2018 at 10:03 AM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: PMC Report: FDA Approves Record Number of Personalized Medicines, Spearheads Six Regulatory Precedents in Field in 2017

Read Full Post »

Skin Regeneration Therapy One of First Tissue Engineering Products Evaluated by FDA

Posted in 21st Century Cures Act, FDA, FDA Regulatory Affairs, FDA, CE Mark & Global Regulatory Affairs: process management and strategic planning - GCP, GLP, ISO 14155, Tissue Engineering and Regenerative Medicine, tagged , , , , , on January 8, 2018| Leave a Comment »

Skin Regeneration Therapy One of First Tissue Engineering Products Evaluated by FDA

Reporter: Irina Robu, PhD

Under the provisions of 21st Century Cures Act the U.S. Food and Drug Administration approved StrataGraft regenerative skin tissue as the first product designated as a Regenerative Medicine Advanced Therapy (RMAT) produced by Mallinckrodt Pharmaceuticals. StrataGraft is shaped using unmodified NIKS cells grown under standard operating procedures since the continuous NIKS skin cell line has been thoroughly characterized. StrataGraft products are virus-free, non-tumorigenic, and offer batch-to-batch genetic consistency.

Passed in 2016, the 21st Century act allows FDA to grant accelerated review approval to products which meet an RMAT designation. The RMAT designation includes debates of whether priority review and/or accelerated approval would be suitable based on intermediate endpoints that would be reasonably likely to predict long-term clinical benefit.

The designation includes products

  • defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products;
  • intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
  • preliminary clinical evidence indicates the drug has the potential to address unmet medical needs for such disease or condition.

According to Steven Romano, M.D., Chief Scientific Officer and Executive Vice President, Mallinckrodt “We are very pleased the FDA has determined StrataGraft meets the criteria for RMAT designation, as this offers the possibility of priority review and/or accelerated approval. The company tissue-based therapy is under evaluation in a Phase 3 trial to assess its efficacy and safety in the advancement of autologous skin regeneration of complex skin defects due to thermal burns that contain intact dermal elements.

SOURCE

https://www.rdmag.com/news/2017/07/skin-regeneration-therapy-one-first-be-evaluated-fda

Read Full Post »

The Top 10 Drug launches of 2017 following FDA green light to 22 drugs in 2016

Posted in FDA Regulatory Affairs, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence on June 26, 2017| Leave a Comment »

The Top 10 Drug launches of 2017 following FDA green light to 22 drugs in 2016

Reporter: Aviva Lev-Ari, PhD, RN

 

UPDATED on 7/3/2017

Fears over a medical gold rush in cancer drug race

With almost 800 trials under way observers warn scientific rigour is being compromised
“There is some pushback,” said Jill O’Donnell-Tormey, chief executive of the Cancer Research Institute. “Are there too many trials? Are we just throwing spaghetti at the wall, by taking compound ‘X’ or ‘Y’ and adding it together just to see what happens?”  Most scientists say checkpoints do not need to be replaced with something else, but rather augmented with new drugs that can further cajole the immune system into fighting cancer. This has led to an unprecedented amount of clinical research sponsored by drugmakers, which are combining checkpoints with other medicines to try to find a magic bullet to treat cancer.  The sheer number of studies has sparked fears that some companies are engaging in a medical gold rush, hoping to chance upon the right cocktail without doing the appropriate scientific groundwork.

https://www.ft.com/content/00092dde-578c-11e7-9fed-c19e2700005f?mhq5j=e3

 

After an unusually slow year for new drug approvals—the FDA green lighted just 22 meds in 2016—it remains to be seen whether drugmakers can do much better in 2017. One thing’s for sure, though: No matter what total the industry tallies up this year, the crop will bring some would-be blockbusters and market disrupters.

At the top of the list, according to EP Vantage’s 2017 preview, which ranks the year’s rollouts by 2022 sales, is Ocrevus (ocrelizumab), the Roche multiple sclerosis drug that’s promising to shake things up in more ways than one. In clinical trials, the candidate bested Merck KGaA’s standard therapy Rebif, and it’s also gone where no other MS drug has gone before, posting positive data in patients with the primary progressive form of the disease. Those data will put the heat on other meds—and invite payers to pile pressure onto the segment, too.

The top 10 drug launches of 2017

The top 10 drug launches of 2017

SOURCE

http://www.fiercepharma.com/special-report/top-10-drug-launches-2017

Read Full Post »

‘Landmark FDA approval bolsters personalized medicine’ by Edward Abrahams, PhD, President, PMC

Posted in Cancer and Current Therapeutics, Drug Discovery Chemistry, FDA Regulatory Affairs, Genome Biology, Personalized and Precision Medicine & Genomic Research, Variation in human protein-coding regions on June 21, 2017| Leave a Comment »

Personalized Medicine been Positively affected by FDA Drug Approval Record

Reporter: Aviva Lev-Ari, PhD, RN

FDA to Clear Path for Drugs Aimed at Cancer-Causing Genes

By Anna Edney and Michelle Cortez

June 20, 2017, 10:41 AM EDT June 20, 2017, 3:02 PM EDT

https://www.bloomberg.com/news/articles/2017-06-20/fda-moves-to-clear-path-for-drugs-aimed-at-cancer-causing-genes

 

 

‘Landmark FDA approval bolsters personalized medicine’

PMC – An Op-Ed in STAT News

by Edward Abrahams

June 21, 2017

Our understanding of cancer has been morphing from a tissue-specific disease — think lung cancer or breast cancer — to a disease characterized more by specific genes or biomarkers than by location. A recent FDA decision underscores that transition and further opens the door to personalized medicine.

Two years ago, the director of the FDA’s Office of Hematology and Oncology Products told the Associated Press that there was no precedent for the agency to approve a drug aimed at treating tumors that generate a specific biomarker no matter where the cancer is in the body. Such a drug had long been seen as the epitome of personalized medicine. But with the rapid pace of progress in the field, director Dr. Richard Pazdur said, such an approval could one day be possible.

That day has arrived.

In a milestone decision for personalized medicine, the FDA approved Merck’s pembrolizumab (Keytruda) late last month for the treatment of tumors that express one of two biomarkers regardless of where in the body the tumors are located. The decision marks the first time FDA has approved a cancer drug for an indication based on the expression of specific biomarkers rather than the tumor’s location in the body.

Keytruda is designed to help the immune system recognize and destroy cancer cells by targeting a specific cellular pathway. The FDA notes that the two biomarkers — microsatellite instability-high (MSI-H) and mismatch repair deficient (dMMR) — affect the proper repair of DNA inside cells.

The approval represents an important first for the field of personalized medicine, which anticipates an era in which physicians use molecular tests to classify different forms of cancer based on the biomarkers they express, then choose the right treatment for it. In contrast to standard cancer treatments, which are given to large populations of patients even though only a fraction of them will benefit, Keytruda was approved only for the 4 percent of cancer patients whose tumors exhibit MSI-H or dMMR mutations. That may help the health system save money by focusing resources only on patients who are likely to benefit from Keytruda.

Such “personalized” strategies now dominate the landscape for cancer drug development. Personalized medicines account for nearly 1 of every 4 FDA approvals from 2014 to 2016, and the Tufts Center for the Study of Drug Development estimates that more than 70 percent of cancer drugs now in development are personalized medicines.

While this is encouraging, the U.S. research, regulatory, and reimbursement systems aren’t aligned to stimulate the development of personalized medicines, and may even deter progress.

The Trump administration’s proposal to cut biomedical research spending at the National Institutes of Health by 18 percent in fiscal year 2018, for example, would undermine its ability to fund more studies like the National Cancer Institute’s Molecular Analysis for Therapy Choice (MATCH) trial, which is designed to test targeted therapies across tumor types.

While the regulatory landscape for these targeted medicines is clear, the path to market for the molecular tests that do the targeting is not. That uncertainty continues to stifle investment in the innovative tests that make personalized medicine possible. The result is a clinical environment in which the patients who could benefit from personalized medicines are often never identified because the necessary tests aren’t available to them.

Finally, increasing pressure on pharmaceutical and diagnostic companies to decrease prices without considering their value to individual patients and the health system could also deter investment in innovative solutions that address unmet medical needs, particularly for smaller patient populations.

Confronted with unprecedented opportunities in personalized medicine, policymakers would do well to ensure that our research, regulatory, and reimbursement systems facilitate the development of and access to these promising new therapies. Only then can we ensure that Keytruda’s groundbreaking approval represents the beginning of a new era that promises better health and a more cost-effective health system.

Edward Abrahams, Ph.D., is president of the Personalized Medicine Coalition.

 

 

 

SOURCE

From: <cwells@personalizedmedicinecoalition.org>

Date: Wednesday, June 21, 2017 at 1:38 PM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: PMC in STAT: “Landmark FDA Approval Bolsters Personalized Medicine”

Read Full Post »

Scott Gottlieb to Newly Confirmed Head of the FDA & HSPH Interview with Margaret Hamburg, 21st Commissioner of the U.S. Food and Drug, 5/9/2017

Posted in FDA Regulatory Affairs on May 10, 2017| Leave a Comment »

Scott Gottlieb to Newly Confirmed Head of the FDA & HSPH Interview with Margaret Hamburg, 21st Commissioner of the U.S. Food and Drug, 5/9/2017

Reporter: Aviva Lev-Ari, PhD, RN

 

WATCH VIDEO 

Margaret Hamburg, 21st Commissioner of the U.S. Food and Drug Administration

 

 

 

 

Public Health Leadership in Challenging Times: Learning from the Past and Preparing for the Future

Tuesday, May 9, 2017

Margaret Hamburg is an internationally recognized leader in public health and medicine, where she is known for advancing regulatory science and modernizing regulatory pathways. From 2009-2015 she served as the 21st Commissioner of the U.S. Food and Drug Administration. She was also the founding vice president and senior scientist at the Nuclear Threat Initiative, a foundation dedicated to reducing nuclear, chemical and biological threats. Other positions have included Assistant Secretary for Planning and Evaluation (HHS), Health Commissioner for New York City, and Assistant Director of the National Institute of Allergy and Infectious Disease.

SOURCE

https://www.hsph.harvard.edu/voices/events/margaret-hamburg-21st-commissioner-of-the-u-s-food-and-drug-administration/

 

Senate Confirms Scott Gottlieb to Head F.D.A.

By KATIE THOMAS MAY 9, 2017

Excerts from NYT article

At his confirmation hearing in April, Dr. Gottlieb told senators that he believed in upholding the F.D.A.’s reputation as the world’s “gold standard” for drug approval. If confirmed, he said then, he would be “an absolutely objective regulatory watchdog” and would not do anything that could “besmirch the agency” or undermine public confidence in its work.

One of Dr. Gottlieb’s biggest jobs will be putting in place the 21st Century Cures Act, a law signed by President Barack Obama late last year that directs the F.D.A. to speed up drug approvals. He also said he would make addressing the nation’s opioid epidemic a top priority.

SOURCE

Read Full Post »

VC Investment in BioTech MegaHubs and Top R&D Spenders among Big Pharma

Posted in BioTechnology - Venture Creation, BioTechnology - Venture Creation, Venture Capital, Drug Development Process, FDA Regulatory Affairs, Venture Capital on April 24, 2017| Leave a Comment »

VC Investment in BioTech MegaHubs and Top R&D Spenders among Big Pharma

Reporter: Aviva Lev-Ari, PhD, RN

UPDATED on 4/26/2017

The top 10 pharma R&D budgets in 2016

The Top 10 Pharma R&D Budgets in 2016

http://www.fiercebiotech.com/special-report/top-10-pharma-r-d-budgets-2016?utm_medium=nl&utm_source=internal&mrkid=993697&mkt_tok=eyJpIjoiT1RSbE9ESTRNR1pqWTJFNCIsInQiOiJFcUx4MFhxSFVGbVZhUkRGdUdRMTJMUGxFSEkrR0VTMEdXbjRvZkxmdXM4em4wRkg5QXZIOWJJWTgwNHR1a1dVbTRIUFwvNWRIXC9ZTkF5dHlpUUZ4bG1lS2c2NkszQk9oeGtRczhLcnYyalRSZEFjOEl6U3dUY2VaakxUbDdkNGNwIn0%3D

 

 

 

Table SOURCE: Thomson Reuters abd ENDPOINTS

According to both sources:

  • $3.5 billion for Silicon Valley plus the Bay Area and
  • $2.8 billion for New England.

Broken down by city, $6.1 billion went to

  • Boston ($2.7 billion),
  • San Jose ($2.5 billion) and
  • San Francisco/Berkeley ($1 billion).
  • San Diego ($725 million),
  • New York ($454 million) and
  • the Great Lakes area ($412 million)

SOURCE

Where the money is: Biotech’s megahubs command VC’s billions by john carroll, June 30, 2016 10:41 AM EDT, Updated: November 17, 2016 07:32 PM

https://endpts.com/where-the-money-is-biotechs-megahubs-command-vcs-billions/

The top 15 spenders in the global drug R&D business: 2017

by john carroll, 

April 24, 2017 05:22 AM EDT, Updated: 05:27 AM

The top five in the business saw their collective spending jump by more than $5 billion, from 2015 to 2016, based on the annual numbers filed largely — though not entirely — with the SEC and gathered by Endpoints News. Two of those companies,

  • Roche and the new number 2, a hard charging
  • Merck, accounted for the lion’s share of the increase. (To be sure, some onetime non-R&D spending, such as Merck’s patent settlement with Bristol-Myers on Keytruda, figured in. But so did bread and butter spending.)
  • Gilead also saw a significant increase in research costs, with
  • Eli Lilly — now off course following two bad setbacks for solanezumab and baricitinib — and the ever aggressive
  • Celgene joining the action as they pressed the accelerator on new drug programs.

Curiously, the added spending coincided with a bad drop in new drug approvals in 2016. But they don’t correlate, and we’ve already seen that turnaround under way as regulators get busy with a brand new year — and soon a brand new FDA commissioner.

SOURCE

https://endpts.com/special/top-research-budgets-in-pharma-and-biotech/

Read Full Post »

Personalized Medicine Meeting – Discussion on FDA Regulation with Outgoing FDA Commissioner, Pharma and Investors

Posted in FDA Regulatory Affairs, Personalized and Precision Medicine & Genomic Research on January 25, 2017| Leave a Comment »

Personalized Medicine Meeting – Discussion on FDA Regulation with Outgoing FDA Commissioner, Pharma and Investors

Reporter: Aviva Lev-Ari, PhD, RN

The agency has long felt that the current oversight provided by the Centers for Medicare & Medicaid Services under CLIA has critical gaps and is putting patients at risk. However, for more than two decades, the lab industry has aggressively fought against FDA regulation by threatening legal action and lobbying Congress. Despite industry objections, the FDA two years ago pushed ahead a draft guidance outlining a risk-based oversight plan for LDTs. However, following the November elections, the agency said it would hold off on finalizing those plans in order to consider input from other groups and await further input from the new administration.

Then, a week before Donald Trump was sworn into office, the agency released a discussion paper outlining a revised regulatory framework for LDTs based on more than 300 comments to its draft guidance, a public workshop, and meetings with stakeholders. The paper allowed the agency, without issuing enforceable regulations, to publicly respond to the lab industry’s concerns about burdensome requirements, demonstrate that it had listened to critics of the draft plan, and lay out the rationale, once again, for why FDA needed to step in to look at aspects of test development that CMS doesn’t.

“I think the community better take this really seriously,” Califf said discussing LDT regulation at the PMWC. While on the one hand regulation shouldn’t stifle innovation, he noted that doctors can’t be left to figure out which test they should order. “We’ve got to come up with some middle ground, so regardless of where you are in the US you can get a reproducible laboratory result,” he said, especially when patients’ treatment decisions depend on those results. “I think that’s upcoming work for this year,” Califf added.

Some industry observers have noted that in a crowded immunotherapy market, the availability of multiple PD-L1 tests, the availabilty of FDA approved kits and unapproved LDTs that seemingly gauge the same analyte, and the companion versus complementary diagnostic categories, are actually confusing physicians. This has led to some to suggest that drugmakers work with regulators to advance one test.

Additional Sources

  • New Complementary Dx Category Provides Regulatory Flexibility, but Poses Real WorldChallenges

GenomeWeb , 2016

  • At CDx Harmonization Meeting, Drugmakers Take First Step Toward Exploring Test Differences

GenomeWeb , 2015

  • In Approving Opdivo With Dakos Complementary Test, FDA Advances Rx Personalization Option

GenomeWeb , 2015

  • In 2016, Personalized Medicine Saw More CDx Deals, Flexible FDA, No LDT Guidance

GenomeWeb , 2017

  • Agilents Dako Lands FDA Approval for Lung Cancer Complementary Dx

GenomeWeb , 2015

  • In 2015, Precision Medicine Options Grew; FDA, Labs Still at Odds; Payment Remained Mostly Elusive

GenomeWeb , 2015

Outgoing FDA Commissioner, Pharma, Investors Discuss Regulation at Personalized Medicine Meeting

Jan 24, 2017 | Turna Ray

SOURCE

https://www.genomeweb.com/molecular-diagnostics/outgoing-fda-commissioner-pharma-investors-discuss-regulation-personalized?utm_source=SilverpopMailing&utm_medium=email&utm_campaign=Daily%20News:%20NSF,%20USDA%20Award%20Microbiome%20Research%20Grants%20-%2001/24/2017%2004:15:00%20PM

Read Full Post »

« Newer Posts - Older Posts »