Join us for a Kelley School Life Sciences event on February 17

Indiana University’s Kelley School Center for the Business of Life Sciences is hosting a conference titled  2016 Election Effects on Life Sciences and Healthcare.

Overview:

Now that the winner of the presidential election and composition of the House and Senate has been determined, what will happen next with healthcare reform, regulatory oversight, new product approvals and the pricing of medical products?

Stakeholders and observers will be sharing their thoughts on the next phase of remaking the nation’s healthcare sector.

Date & location:

This event is part of the Indiana Life Sciences Collaboration Conference Series. The conference will take place on February 17, 2017 at the One America Tower in downtown Indianapolis.

Pearl Pathways is a proud sponsor of the Kelley School Life Sciences series. Please click here to register for the event.

Save the date: IMDMC Medical Device event at the Indiana Statehouse February 15, 2017

On February 15, 2017, the Indiana Medical Device Manufacturers Council (IMDMC) will host the annual Medical Device Exhibit and Breakfast at the Indiana Statehouse. This event provides the opportunity to interact with state and federal officials about the industry. To register for the event, click here.

NCI launches new drug formulary to speed up clinical trials

cancer moonshot clinical trialsLast week, the National Cancer Institute (NCI) launched a new drug formulary. The NCI Formulary will “enable investigators at NCI-designated Cancer Centers to have quicker access to approved and investigational agents to use in preclinical studies and cancer clinical trials ” (cancer.gov).

NCI aims for the new formulary to enable new treatment options to reach patients diagnosed with cancer more quickly. The NCI Formulary will achieve this by reducing the lengthy negotiation process traditionally required for investigators to access certain agents. The new formulary is a public-private partnership between NCI and pharma/biotech companies; it also represents an effort by the NCI to support the Cancer Moonshot. The NCI Formulary launched with 15 agents from six different pharmaceutical companies. NCI expects these numbers to double by the end of 2017 based on ongoing negotiations between NCI and interested organizations.

The current acting director of NCI, Douglas Lowy, states that “the NCI Formulary will help researchers begin testing promising drug combinations more quickly, potentially helping patients much sooner…rather than spending time negotiating agreements, investigators will be able to focus on the important research that can ultimately lead to improved cancer care” (cancer.gov). The formulary puts NCI in a position to facilitate arrangements between investigators at NCI-designated Cancer Centers and participating pharmaceutical companies for access to and the use of pharmaceutical agents. Once approved, investigators can obtain agents from the formulary to test them in new preclinical or clinical studies as well as combination studies of formulary agents from varying companies. Another public-private partnership called  the Partnership to Accelerate Cancer Therapies (PACT) will roll out in 2017 under the NIH. Identification and validation of biomarkers of response and resistance to therapies will be the central focus of PACT.

Pearl Pathways can support your new or ongoing clinical trials with its team of experienced advisors. Please contact us to start a conversation. For more information about new federal and public cancer programs to support the Cancer Moonshot, stay tuned to our blog.

CDER’s novel drug approvals for 2016: a review

Last week, John Jenkins, M.D. issued a review of the FDA’s new drugs program in the Center for Drug Evaluation and Research (CDER). The total number of new drug approvals decreased dramatically in 2016 versus the previous year; however, Jenkins, who is the Director of the Office of New Drugs in FDA’s CDER, approaches the numbers with optimism (fda.gov). Twenty-two novel drugs received approval by CDER, and most of these drugs could add “significant clinical value” to the care of thousands of patients with serious and life-threatening illnesses.

2016 could be viewed as a year of “firsts” for new drug approvals. Spinal muscular atrophy and Duchenne muscular dystrophy both received first treatments. Parkinson’s disease related hallucinations and delusions, primary biliary cirrhosis, and hepatitis C each received new treatments. Several new oncology drugs obtained approval to help patients with ovarian cancer, bladder cancer, soft tissue sarcoma, and chronic lymphotic leukemia. CDER approved two new diagnostic agents used to detect certain forms of cancer as well. Jenkins notes that 73% of the 22 novel products benefited from at least one of the FDA’s programs to expedite new drug development and review. Another promising statistic from the CDER review team is that 95% of the novel drug products they reviewed last year were “first cycle” products. This means that CDER did not need to request additional information from submitters to approve the product. Find an extensive look at all of last year’s new drug approvals in the FDA’s 2016 Novel Drugs summary.

The high usage rate of an FDA fast-track program indicates efficiency within the approval process. The nearly 100% “first cycle” approvals suggests that life science companies are submitting accurate and comprehensive clinical trial data. Pearl Pathways prides itself on helping companies and researchers successfully expedite the product development life cycle. Last year alone, our team provided expert insight into the FDA’s Fast Track, Breakthrough Therapy, and priority review designations to several of our clients. Please contact us today to find out how we can help you with your new treatments in 2017 and beyond.

Venture capital funding in the life sciences industry: a 2016 review

venture capital funding stock photoVenture capital investments in the life sciences industry remain strong despite a decline in 2016 from the record-setting 2015 numbers. A report from PharmaVOICE this month breaks down the investments from the past two years. The life sciences stand in second place behind the software industry in terms of total investment dollars. Fourth quarter data is not yet available, but the life sciences share of total venture funding grew to 23% during Q3, a 5% increase over 2015’s third quarter. Biotechnology received $1.8billion across 87 deals in Q3, but this marks a 15% decrease in deal value and a 30% drop in deal volume compared the previous year’s Q3. Medical device investments also decreased in the same quarter-to-quarter comparison (down 23% for deal value and 19% in volume). A five-quarter streak of declining total deal volume continued in the third quarter of ‘16.

Let’s not forget that 2015 represents a record-setting year for VC money entering the life sciences. The industry is experiencing an impressive investment run: 11 consecutive quarters of more than $10 billion in venture capital investment. Though Q3 of 2016 marked the smallest number of total deals since 2010, “quality deals continue to receive funding…the broader ecosystem remains healthy, bolstered by a lift in biotechnology…strong fundraising, and a continuation of the trend toward investments in nontraditional industries” (Tom Ciccolella, PwC). Focusing on the total number of deals as the key indicator of the strength of the market would understandably bring about concern. Though the number of deals has declined, total dollars have not. Venture capitalists now have the luxury of being picky as high-quality startup companies rise. Series A rounds increased dramatically in ’15 and ’16, from the traditional $10mil or less to $50mil or over $100mil per round. Time to market for certain technologies and drugs continues to decrease, which may be a driving force behind a smaller share of life science companies receiving a larger share of the VC funding pool. Additional improvements in cutting-edge science such as the “human genome sequence…computational power for technology, and better in vitro and in vivo assays” (PharmaVOICE) contribute to investors’ behavior as well.

Our team at Pearl Pathways has helped several VC funded companies as well as VC funds directly over the years in navigating regulatory and clinical pathways to successfully launch products to market. We also frequently communicate with venture capital firms to provide education and insights about the life science industry and ever-changing regulatory landscape.

President Obama provides his perspective on repealing the ACA without a replacement

As President Obama’s time in office wanes, the exiting president offers his perspective in the New England Journal of Medicine on the potential repeal of his administration’s Affordable Care Act. The ACA currently stands as a significant part of the president’s legacy, a landmark bill that transformed the healthcare industry in the United States, for better or worse. President Obama recognizes that “health care policy often shifts when the country’s leadership changes. That was true when I took office, and it will likely be true with President-elect Donald Trump.” The President explains his pride in how the current system offers high-quality care to millions of previously uninsured Americans. Indeed, a larger portion of Americans have health insurance now than ever before. However, Obama recognizes that work remains to be done to improve the Affordable Care Act. He notes a lack of choice exists in some health insurance markets, premiums remain unaffordable for some families, and high prescription-drug costs. His administration’s ideas for overcoming these issues have been documented in articles and budget proposals, but “persistent partisan resistance to the ACA has made small as well as significant improvements extremely difficult.”

The incoming administration’s seems geared on repealing first and replacing later (potentially 2-3 years later), a course of action that President Obama deems irresponsible and “could potentially bleed the health care system that all of us depend on… the health care system will be standing on the edge of a cliff… insurance companies may not want to participate in the Health Insurance Marketplace in 2018 or may significantly increase prices in the next year or two, partly to try to avoid the blame for any change that is unpopular.” Obama lists more potential snowball effects of a full or partial repeal, including the risk of losing coverage for preexisting conditions, an aspect of the ACA that President-elect Trump wishes to maintain. The president exclaims that he will applaud legislation that improves Americans’ care, but improvements need to be identified explained by the new administration before action is taken.

Evidence-based practice is expected within our modern health care system. Health care reform, too, should be evidence-based and driven by what is best for the patient. President Obama concludes by urging policymakers to consider what is best for the patient, in this case the entire population of our country, and abide by the physician’s oath of “first, do no harm.”

What will a full repeal mean for the life science industry which provides so many life changing and lifesaving solutions to patients in clinical care? The impact remains to be seen. Stay tuned!

Live webinar covering Medical Technology Reimbursement in Australia from Pearl Pathways’ partner, Brandwood Biomedical – January 17th

Next Tuesday, January 17th, join our Asia Pacific partner Brandwood Biomedical to learn about the strong yet complex medical technology market in Australia. With changes already announced to some prostheses prices, a Senate Inquiry, and the continuing reform of private health insurance, 2017 is set to be an eventful year for suppliers of medical technology to the Australian market.

This free webinar will begin at 4:00 PM US EST and you may complete your registration here.

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21st Century Cures Act: potential impact on the clinical research landscape

clinical trialsThe historic 21st Century Cures Act, written into law last December, is a 362-page bill comprised of several initiatives impacting the life sciences industry. The allocation of $4.8 billion for the “Cancer Moonshot” portion of the bill won over most headlines during news cycles leading up to the bill’s signing, but several other sections will impact the clinical research ecosystem over the next several years. An article published in this month’s volume of the Journal of Clinical Research Best Practices outlines the major sections of the Cures Act that affect clinical trials.

The aforementioned Cancer Moonshot leads the bill, creating a $4.8B “NIH Innovation Account” that will allocate funds to the Precision Medicine Initiative, BRAIN Initiative, Cancer, and Adult Stem Cells research. Another $500M will be set aside for an “FDA Innovation Account.” The Eureka Prize Competition section allocates prize money for significant advancements in biomedical sciences and/or improving health outcomes in serious yet disproportionate research areas. Three sections address confidentiality of personal health information for study participants: Privacy Protection for Human Research Subjects, Protection of Identifiable and Sensitive Information, and Data Sharing.

To support emerging scientists, the NIH will develop and prioritize policies that promote opportunities for new researchers. The NIH also addresses Educational loan repayment addresses by increasing repayments from $35k to $50k in exchange for research work in the areas of basic science, AIDS, and emerging needs. This section also instructs the NIH to prioritize research conducted by professionals from disadvantaged backgrounds.

Reducing administrative burden for researchers also makes up a large section of the bill. Within two years, HHS is to “harmonize and eliminate duplicative Conflict of Interest reporting… [and] examine the varying minimum thresholds, consider allowing for just in time reporting, and consider redefining which investigators and sub-investigators need to report.” Several sections address the patient experience during clinical trial studies. The FDA will create guidance explaining the use and requirement of patient experience data such as data collected by non-clinicians (e.g. patients, family, etc.), the impact of disease and therapy on patient lives, and patient treatment preferences.

The bill would not be complete without detailing the penalties for violation of grants, contracts, and other agreements created under the 21st Century Cures Act. The bill outlines fines from $10k-$50k for each false statement or omission in addition to $10k-$15k fines per day for delays in the transfer of funds to HHS.

Do you need assistance with your NIH funded research? Please contact us at Pearl Pathways.

Must-know updates for the new risk-based ICH GCP Guideline

A new addendum to the ICH Good Clinical Practice (GCP) Guideline carries broad implications for sponsors, CROs, and investigators. The guideline, now known as ICH E6 (R2), was drafted in response to advances in technology within clinical trials (e.g. electronic data recording and reporting, centralized monitoring) since E6 (R1) was prepared. The new addendum aims to “encourage implementation of improved and more efficient approaches to clinical design, conduct, oversight, recording, and reporting while continuing to ensure human subject protection and reliability of trial results.”

Why does this matter for sponsors? The addendum’s changes could transform how clinical monitoring and trial management are conducted by “requiring the adoption of centralized, quality risk management (QRM) throughout the trial lifecycle.” Sponsors must satisfy the principles of a quality management system (QMS) based on the risks and potential errors in critical data and processes. To address identified significant non-compliance, the guidance provides three principles for sponsors to follow: significant non-compliance identification, root cause analysis, and custom made corrective and preventative action. Moving forward, sponsors will need to maintain oversight of tasks delegated to CROs, placing responsibility on the CRO to maintain effective systems to track data and tasks during clinical research.

The guidance also introduces a new framework to encourage sponsors to adopt innovative technologies. An overview of the new addendum describes the three main concerns addressed by this new framework. First, the framework specifies requirements for the validity, longevity, and fidelity of trial data as sponsors transition from physical to digital records and update or change between digital systems. Second, it encourages “standard processes to avoid situations where real-time data aggregation and visualization may inadvertently influence trial outcomes inappropriately early in the trial process.” Third, the framework expands upon the need for control by investigators of their generated data and documents.

Risk-based quality management, risk-based monitoring, and CRO oversight sum up the three main roles and responsibilities outlined in the addendum. Reviewing and understanding the full addendum will be vital in your preparation for the upcoming changes. Please contact us at Pearl Pathways to discuss the needs of your clinical research.

Happy Holidays from the Pearl team

Over the past two weeks, the Pearl team celebrated the holiday season with various “12 Days of Christmas” activities. Events ranged from reindeer racing to gingerbread house building with plenty of company pitch-ins along the way.

 

 

As 2016 winds down and you reflect upon the year’s challenges and successes, we wish you and yours a fabulous holiday season full of peace, hope, and relaxation.