EMA finds development challenges for medicines targeting CNS disorders in new report

The European Medicines Agency (EMA) released a report on Tuesday that analyzed over 100 applications submitted for medicines in the fields of psychiatry or neurology between 1995 and 2014. In the report, EMA concludes that various challenges can arise in the development of medicines targeting central nervous system (CNS) disorders and stresses the importance of appropriate design of early-stage clinical trials for future development success.

Complexity of research for CNS medicines adds to the challenge of successful drug development; EMA cites a “higher rate of failure during the clinical development of these products compared to other fields of medicine” in their report. Of the 103 applications received by EMA’s Committee for Medical Products for Human Use (CHMP), 57 were neurology products and 46 were psychiatry, and 29 were rejected or withdrawn leaving 74 that received approval. Submitted drugs targeted schizophrenia, MDD, Alzheimer’s disease, epilepsy, and more.

EMA’s claim that appropriate design of early-stage clinical trials is not ungrounded in fact. The analysis explains that “over 50% of the applications that had major problems with the outcome confirmatory study (in terms of efficacy and/or safety) also had issues in the early clinical development.” The chart below visualizes the report’s findings and the differences in the types of issues raised for neurology medicines and psychiatry medicines.

EMA report data analysis

Appropriate design of early-stage clinical trials is vital for resulting in efficacious results, and Pearl Pathways is here to help. Please contact us to set up a discussion with our team of experts to assist with your drug or device clinical trials.

The 21st Century Cures Act passes through the House with resounding support

The 2st Century Cures Act, a nearly 1,000 page bill that details changes for the Food and Drug Administration (FDA) and National Institute of Health (NIH), passed by a vote of 392-26 today in in the House of Representatives. A Senate vote could come as soon as next week.

Assuming the bill prevails in the Senate, the NIH will be the recipient of $4.8 billion in funding over the next 10 years, though money allocation must be reauthorized each year. This money is “earmarked for the three landmark big science initiatives of President Obama’s time in office: the cancer moonshot, the BRAIN Initiative, and the Precision Medicine Initiative” (FierceBiotech).

The passing vote of 392-26 came as a shock to some critics of the bill, “showing a bipartisan spirit that has been rare in recent years” (statnews.com). If the bill passes, it would also “gives states $1 billion to fight the opioid crisis, and deliver an additional $500 million to the FDA.”

Experienced project manager joins Pearl Pathways

shawn-knoppPearl Pathways is pleased to announce the hiring of Shawn Knopp, PhD as an Advisor serving medical device and biopharmaceutical life science companies.

Knopp brings to Pearl Pathways over 15 years of experience in multi-disciplinary life science roles including project management, product development, regulatory, engineering, and manufacturing.

Diana Caldwell, President and CEO shares, “Our clients will benefit from Shawn’s extensive leadership experience navigating a variety of regulatory compliance challenges in large multi-nationals and small startups… His past experience working as in independent consultant in the industry allows him to adapt to the dynamic needs of the biopharmaceutical and medical device companies we serve. We are so pleased to welcome Shawn to the team.”

To view the full press release, please click here.

FDA’s list of emerging tech requests topped by continuous manufacturing and sterile production innovations

The US FDA’s Emerging Technology program was established as a catalyst for new technologies to help modernize pharmaceutical development and manufacturing in areas where the FDA has limited review or inspection experience. Thomos O’Connor from the FDA’s Office of Pharmaceutical Quality explains that “this is technology with the potential to modernize the body of knowledge associated with pharmaceutical development to support more robust, predictable, and cost-effective processes or novel products… our vision is to have a maximally efficient, agile, flexible pharmaceutical manufacturing  sector that reliably produces high quality drugs without extensive regulatory oversight ” (in-PharmaTechnologist.com).

O’Connor is a member of the committee formed to facilitate the program and assist the industry with applications. The committee, known as the Emerging Technology Team (ETT), has already received 20 applications this year, doubling the number requested last year. The in-PharmaTechnologist article breaks down the submissions: continuous manufacturing technologies for drug product, substance, and biologics represent 30% of all requests, another 30% consists of the use of robotics and container closure systems for sterile injectable production, biotechnology processes and analysis represent 22%, and the final 17% include other areas of innovation such as new dosage forms and 3D printing.

What implications, if any, do these numbers say about the future of the life sciences industry? One could look at the breakdown as a compass for where future demand (and funding) will be. Though continuous manufacturing and robotic/container closure technologies led the way with a majority share of submissions, the numbers represent a fairly even and robust spread of emerging technologies. If your firm is looking to participate in the Emerging Technology program and need guidance with your IND, NDA, ANDA, or BLA application, our dedicated team of experts at Pearl Pathways is here to help.

FDA Halts Finalization of Lab-Developed Test Draft Guidance

Last Friday, the FDA halted the finalization of guidance that would have changed the way lab-developed tests (LDTs) are regulated. The reason? The presidential election. The current administration now enters its waning hours and the FDA decided to suspend guidance until the new regime takes office.

As RAPS reported, the press office for the FDA, Tara Goodin, defended this position because the “FDA believes that patients and health care providers need accurate, reliable, and clinically valid tests to make good health care decisions – inaccurate or false tests results can harm individual patients. We have been working to develop a new oversight policy for laboratory developed tests… and realize just how important it is that we continue to work with stakeholders, our new Administration, and Congress to get our approach right. We plan to outline our view of an appropriate risk-based approach in the near future.”

The agency released draft guidelines two years ago outlining a risk-based framework for regulating LDTs that would be phased in over nine years. LDTs have historically been regulated by the Centers for Medicare & Medicaid Services under the Clinical Laboratory Improvement Amendments. The FDA’s decision, though praised by some, creates uncertainty for the industry and may leave a lot of companies in limbo. To learn more about this topic, see our industry whitepaper. Need assistance navigating the regulatory guidance for IVDs or LDTs, contact us and our experts can help.

Paging Dr. Google: The growing use of digital channels in physicians’ daily practice

“Just Google it” has shifted from a colloquialism spoken within small circles to an expression used ubiquitously around the world, so much so that Google, Inc. pleaded the public ten years ago to only use their name when referring to the company to avoid losing its trademark status. Despite the company’s efforts, no one seems to be immune to the spread of Google as a verb rather than a noun, even physicians. Though the expression can sometimes be received with a negative connotation (I can recall frustrated accounts from classmates of professors telling them to Google the answer to questions), utilizing digital search engines is an effective means of accessing information quickly.

An annual survey by CMI/Compas breaks down just how frequently today’s physicians use search engines – most often Google – in their day-to-day work. FiercePharma provided a nice summary of the results.

  • More than 70% of physicians use search engines ≥ 1 time per day
  • Oncologists search most frequently, with 46% searching 4 or more times daily

CMI/Compas also analyzed information-gathering behaviors across the six different specialty areas of primary care, as seen in their chart below.

Physicians' likelihood of using different sources. (CMI Media)

Physicians’ likelihood of using different sources. (CMI Media)

Time appears to be the biggest factor influencing physician’s information-gathering behaviors. When faced with less pressing matters, physicians across the board resort to more “traditional” methods: medical journals, electronic databases, peers and colleagues, etc. However, when an answer must be produced within minutes, online search engines become the primary method of finding necessary information. Even when time isn’t an issue, digital methods still trump the physical options. The increasing availability of reliable clinical and empirical data through online search engines may be influencing this trend, as well as the general ease of use of the search function on a handy electronic device (phone, tablet, etc.) The influence this is having on the medical device and pharmaceutical industry are vast. What is the role of dedicated applications to enhance search on manufacturers’ products? How will physicians increasingly use data fed to them over the internet from their patients’ wearable tech? How do diagnostic companies develop solutions to help them diagnose over the internet?

To learn more, check out this resource.

Experienced quality analyst joins Pearl Pathways

Phillip BishopPearl Pathways is excited to announce the hiring of Phillip Bishop as a quality analyst. Bishop will help the Pearl team serve medical device and biopharmaceutical companies. Bishop brings 13 years of quality control (QC) laboratory experience to the Pearl Pathways team and recently held leadership roles in a good manufacturing practice (GMP) compliant QC laboratory at a top-tier contract manufacturer of sterile products. Diana Caldwell, President and CEO shares, “Phillip brings strong quality, manufacturing, and technical expertise as well as a deep understanding of validation documentation to the Pearl team. Our clients will benefit from his vast knowledge of quality compliance and quality assurance systems, CAPA, multiple test methods, and pharmacopeial work.”

You can read the full press release here.

Holiday season hours of operation for Pearl Pathways

As we enter the holiday season, please keep in mind of our operating hours. Pearl IRB will not hold a full board meeting on November 23rd but meetings will resume as scheduled the following week. The office will be open during normal business hours (8 AM – 5 PM EST) except for the following holidays:

  • Thanksgiving
  • The day after Thanksgiving
  • December 23rd
  • December 26th
  • December 30th
  • January 2nd

Effective January 1st: China FDA Priority Review Procedure

The China Food and Drug Administration announced that manufacturers will be able to request priority review and approval of Class II (limited to imported application) and Class III (both domestic and imported application) devices exported to China beginning January 1, 2017. RAPS reported that the device must have been enrolled in the National Science and Technology Major Project or National Key Research and Development plan, or if it can diagnose and treat:

  • rare diseases, and has significant advantages in clinical practice
  • malignant tumors, and has significant advantages in clinical practice
  • specific and frequently occurring diseases in the elderly, and where there is no effective way to diagnose or cure such diseases
  • specific and frequently occurring pediatric diseases, and where there is no other effective treatment
  • or can address an urgent clinical need, and the same type of device has not yet been marketed in China

If you need help navigating the process for a priority review, please contact us. Pearl Pathways’ expert guidance can help you with your device applications and expedited approval if your device meets the CFDA’s fast-track approval requirements.

Purdue professor to host free webinar for RAPS members: Emerging Issues in Manufacturing with Impact on Quality

raps indianaOn Thursday, November 17th, Purdue University’s professor of Medicinal Chemistry, Dr. Stephen R. Byrn, will host a free webinar exclusive for members of the Indiana Regulatory Affairs Professionals Society (RAPS) Chapter. Members may register for the webinar here.