Diana Caldwell speaking at 3rd annual KLLC Women’s Empowerment Retreat on March 3rd

kelley-school-of-businessOn March 3rd, the Indiana University Kelley Living Learning Center (KLLC) will be hosting their third annual Women’s Empowerment Retreat. This impactful event gives 50 first-year KLLC women an opportunity to network with successful business women and learn how to be a successful female leader. The goal is to build confidence and abilities, and discuss planning, navigating, and recalculating the future. We are proud that our own Pearl Pathways President and CEO, Diana Caldwell will speak and share her own lessons learned in business.  Diana will speak to the successes and challenges she experienced starting her own company with the freshman business students attending. This event is by invite only.

Indy Life Science event this Thursday

life science eventThursday, February 23, Hoosiers Work for Health will host an event, Indiana Life Sciences Industry Update: Key federal issues including PDUFA. The federal briefing will feature Alicia Subasinghe of Pharmaceutical Research and Manufacturers of America, Washington D.C. Participants can look forward to learning about federal policy affecting the life sciences and biopharmaceutical industry in Indiana including the Prescription Drug User Fee Act.

The presentation will be held at Bose Public Affairs Group (111 Monument Circle Suite 2700 Indianapolis, IN 46204), and run from 8:30am – 10:00am EST. Please visit the Hoosiers Work for Health event page to learn more.

Obstacles facing biosimilar developers

biosimilar developer stock photo optionThe biosimilars industry is still in its infancy in the U.S. marketplace, resulting in some growing pains for developers of these products. Biosimilars were created under the Biologicals Price Competition and Innovation Act (BCPIA) of 2009 and signed into law through the Patient Protection and Affordable Care Act (Affordable Care Act) on March 23, 20101. Under the BCPIA, a biological product may be demonstrated to be biosimilar if data show the product to be highly similar to a previously-approved biological product. The biosimilar must show no clinically meaningful differences to the reference product and follow the same mechanism of action.

Last month, BioPharmaDive published a series on biosimilars, one of which addressed “5 hurdles facing biosimilar developers.” With only four biosimilars currently approved in the U.S. market, there is plenty of room for growth. That is, of course, if developers can navigate the hurdles facing them as they jump into this young, bourgeoning industry.

BioPharmaDive listed the following challenges for developers:

  1. Pricing Pressures
  2. Legal battles
  3. Reimbursement
  4. Education
  5. Manufacturing

Regarding pricing pressures, all too often we can see various news organizations covering the backlash from consumers, government, and key stakeholders towards the biopharmaceutical industry regarding pricing. Biosimilars are not immune to this scrutiny. The expectation exists that biosimilars will play a similar role on the market as generic drugs. Generics “revolutionized the way drugs were sold in the U.S., offering an 80% to 90% discount to branded drugs…yet, Pfizer priced its Remicade (infliximab) biosimilar Inflectra (Infliximab-dyyb) at only a 15% discount – raising the issue of whether biosimilars will have their desired impact on the market.”2

A few key legal battles have already broken out since the BPCIA paved the way for biosimilars, potentially creating an unsettling precedent for this new industry. As for reimbursement challenges, locations where nurses or physicians infuse drugs typically use a “complicated reimbursement formula based on average sales price.”2 The BioPharmaDive article goes on to explain the pushback from reference product developers and biosimilar developers about Centers for Medicare & Medicaid Services (CMS) using codes that do not specifically identify the maker of the product. The product payment and required modifiers for biosimilars outlined by CMS can be found here.

A lot of questions still exist surrounding biosimilars, which the FDA began addressing last year through advisory committee meetings. Education for developers, health care professionals, and patients will continue to contribute to the success of the industry moving forward. The drug industry is far from immune to manufacturing setbacks, and biosimilar developers must account for these issues as well. Biosimilars, like the reference biologics they mimic, are derived from living cells “with inherent variability and therefore are incredibly complex to manufacture.”2

Pearl Pathways employs a team of experts with decades of experience across all of the life science industries. Our team has helped several life science companies develop their products and navigate through the challenges detailed above. Please contact us today to begin a conversation.




Save the date: RAPS Indiana Chapter hosts a senior FDA representative to speak about drug safety lifecycle management

RAPS IndianaOn March 9th, the RAPS Indiana Chapter, chaired by Pearl Pathways COO Gretchen Bowker, will host an interactive presentation from a senior FDA representative. The FDA representative, Jill R. Bourdage, RPh, PMP, will speak about processes and procedures of the Office of Surveillance and Epidemiology (OSE) within the Center of Drug Evaluation and Research (CDER). The presentation will provide an overview of the OSE and its role in the continuum of drug safety lifecycle management. Anticipated updates from PDUVA VI will be covered and the audience will learn how the role of the OSE Safety Regulatory Project Manager and regulatory expert as point of contact for industry can help achieve optimal communication.

Presentation title: Optimizing Communications with the Office of Surveillance & Epidemiology

Date: March 9, 2017

Time: 5:30 – 8:00pm EST

Location: Purdue University: Seng Liang Wang Hall

West Lafayette, IN

Registration and more information about the content can be found here.

United States sees first gene edited CAR T-cell receive an IND approval

human T-cellThe United States Food and Drug Administration (FDA) approved its first Investigational New Drug (IND) submission for a gene-edited chimeric antigen receptor (CAR) T-cell product for clinical trials. CAR T-cell therapy involves using a virus to edit an immune cell (T-cell) extracted from an individual patient, instructing the cell to attack cancer cells within that patient. This autologous process requires individualized treatments for each new patient. Cellectis’ new product (UCART123), which the FDA granted IND approval, is an allogenic CAR-T immunotherapy, meaning it is non-patient specific or “off the shelf.”

Cellectis plans to initiate Phase 1 trials this year. UCART123 targets CD123, an antigen expressed at the surface of leukemic cells in acute myeloid leukemia (AML) and tumorous cells in blastic plasmacytoid dendritic cell neoplasm (BPCDN). AML is characterized by “uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues” and accounts for an estimated 19,950 new cases each year in the U.S alone.1 BPDCN, categorized by the World Health Organization (WHO) under AML, typically presents with features of both lymphoma and leukemia. It impacts bone marrow and blood cells but the most frequently involved site of disease is the skin.2

Pearl Pathways can help your company navigate the complex regulatory landscape towards a pre-IND meeting, new IND filing, 510k submission, and more. Please contact us today to schedule a conversation with our team of industry experts.




Indy Science Connect to host a Night of Networking

On February 23rd, Indy Science Connect will host a Night of Networking event from 6-8pm EST. The event will be held at The Tap in Indianapolis, Indiana.

Indy Science Connect is a professional networking group that was “started by a small group of friends that wanted to do more to bridge the gap between life sciences institutions.” You can learn more about the organization here. Stay tuned to our blog for updates regarding Indiana life science events.

China CFDA expedites regulatory reviews

brandwood biomedical CFDABrandwood Biomedical, our Asia Pacific strategic partner, reported this week in a blog post that the China Food and Drug Administration (CFDA) aims to speed up regulatory reviews. The draft policy change, released on December 29, 2016, will impact submissions for drugs and medical devices. The policy change will allow for certain types of drug and medical device submissions to receive direct approval by the Center for Drug Evaluation (CDE) or Center for Medical Device Evaluation (CMDE) without further review.

To learn more about the types of submissions within the scope of the new draft policy, please visit the Brandwood Biomedical blog post here. If your company plans to enter the Chinese market with your product, please contact the expert bilingual team at Brandwood Biomedical for help with your Chinese regulatory and clinical needs. For US / FDA regulatory support, please contact our staff at Pearl Pathways.

Now hiring: Clinical Research Associate

Pearl Pathways is pursuing a Clinical Research Associate to join our team! The CRA will be responsible for the coordination and conduct of clinical research activities either for the sponsor or at a research site. This role will include the day-to-day responsibility for clinical research ensuring studies are conducted in accordance with the protocol, relevant SOPs, and good clinical practices. Pearl Pathways is headquartered in Indianapolis, Indiana with a second office in Houston, Texas. Telecommuting may be an option as well. Please see the full job description, hiring details, and contact information here.

Annual Deloitte report indicates diminishing biopharma R&D returns

In case you missed it, Deloitte recently published its annual report measuring the return from pharmaceutical innovation. Unfortunately, Deloitte’s 2016 conclusions do not bode particularly well for the biopharma industry. The full report, which can be viewed here, contains sections on the ROI of R&D, increasing pipeline value, reducing cost to launch, and the external challenges the biopharma industry faces.

Deloitte compared performance data of 12 leading biopharma companies against four mid- to large-cap companies. These are some of the some of the key findings from Deloitte:

  • Annual projected pharma R&D returns continue to decline to 3.7 percent
  • Peak sales per asset fall 4 percent year-on-year since 2010
  • Costs to bring a product to market stabilize, from $1,576 Million in 2015 to $1,539 Million in 2016
  • Smaller pharma companies have seen a decline in overall performance, but on average they continue to outperform their larger counterparts, generating returns up to three times higher

The return on investment of R&D remains an important indicator within the biopharma industry. Even if R&D costs are high, a strong sales forecast can attract investors. A company’s ROI also guides internal decisions about the allocation of funds to, or away from, the research and development department. A healthy R&D department allows organizations to continue researching new treatment options for various patient needs.

The report includes four main factors currently influencing R&D returns. Maintaining a consistent focus on a specific therapy area, the end of the blockbuster, smaller companies remain more effective, and the increase of M&A are each listed as either positively or negatively influencing returns. Deloitte offers some key lessons for companies to apply that may increase commercial success while reducing the cost to launch:

Increasing commercial success

  • Aim for therapy area focus
  • Target populations where value can be maximized
  • Adhere to robust target product profile
  • Generate evidence to support all stakeholder needs
  • Align end-to-end decision making across the organization

Increasing R&D productivity

  • Think small, win big decision making
  • Strike the right balance of staff and resource
  • Lift the burden of data complexity

The lessons outlined by Deloitte are issues that we discuss with our clients on a daily basis. Pearl Pathways’ team of experienced advisors remain up to date on industry trends to help life science companies both small and large meet their goals. Please contact us today to begin a conversation.

Clinical trials and pharmaceuticals adapt to growing digital health tech market

digital health wearableDescribing the rise of medical “wearables” in the U.S. and global marketplace as meteoric seems fitting given the rapid growth of the industry over the past decade. Wearable technology has been around for decades (check out an amusing history of some medical and not-so-medical wearables here), but mainstream adoption of the technology occurred more recently. Fitbit entered the scene in 2009 followed by several offshoots. Pebble kicked off the smartwatch craze just five years ago, in 2012. Now, one in six consumers in the United States currently use wearable tech such as the aforementioned fitness bands and smartwatches1. This trend will not slow any time soon, as the industry could see more than 3x growth in the next few years, expanding from $5.1 billion in 2015 to $18.9 billion in 20202. How will the expansion of medical wearables impact the life sciences industry?

An article on Insights, a division of Samsung focused on researching how their devices are used by everyday people in everyday life, reports on the adoption of digital health tech in clinical trials and pharmaceuticals. The article cites a survey by digital health technology vendor, Validic, on how digital health devices and data impact clinical trials:

  • “Sixty percent of respondents said they have used digital health technology to conduct clinical trials.
  • Ninety-seven percent of respondents said they will increase their use of digital technologies in clinical trials in the next five years.
  • Mobile apps and in-home clinical-grade devices are currently the most commonly used devices in clinical drug trials, but wearable activity trackers and sensors will be the focus of future use.
  • Seventy percent of respondents said patient-generated health data (PGHD) could have the greatest impact on improving treatments for chronically ill populations.”3

Current consumer-grade wearables are accessible and powerful, but a number of companies see the need to advance the use of clinical-grade devices as well. For example, the Insights article notes a collaboration between Validic and Sutter Health with the Office of National Coordinator for Health Information Technology. Their pilot project aims to determine how to best deliver patient generated health data (PGHD) to healthcare clinicians and researchers. The project “monitors and collects data from Sutter Health patients with Type2 diabetes through the SutterMpower app, which connects to devices measuring patient blood glucose, blood pressure, weight, and activity level.”3.

Medical wearables make remote monitoring of patients a powerful option for clinicians. The Insights article describes another pilot study that explores if patient hospitalization can be avoided through the combination of clinician visits to patient homes and advanced continuous electronic digital patient monitoring. In addition to improving an individual’s health, digital health tech could also reduce healthcare costs. Insights references a study published in The American Journal of Managed Care that analyzed anonymized medical and pharmacy claims data from over 1.2m patients with diabetes, high blood pressure, or high cholesterol. It was found that “payers could save approximately $38 million to $63 million per 100,000 members by deploying resources for improving medication adherence in specific patient populations.”3.

Digital health technology’s transformation of the health care and life science industries has just begun. Medical wearables bring opportunities for individual consumers to track and assess personal vital health information. Health care and life science professionals face the challenge of managing and analyzing this data while continuing to find innovative and safe ways to improve patient health while maintaining the confidentiality of private health data. Our team of experts at Pearl Pathways can support your company’s efforts to develop new digital health devices by navigate the changing regulatory landscape impacting these products.


1 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4737495/#pmed.1001953.ref002

2 https://ww2.frost.com/news/press-releases/clinical-grade-wearables-accelerate-growth-opportunities-internet-medical-things-iomt-solutions/

3 https://insights.samsung.com/2016/12/05/digital-health-tech-takes-off-in-clinical-trials-and-pharmaceuticals/