The U.S. Food and Drug Administration (FDA or Agency) recently announced the final guidance titled “Pre-Launch Activities Importation Requests (PLAIR)” (Final Guidance). The Final Guidance outlines FDA’s policy for the importation of unapproved finished dosage form drug products an applicant is preparing for a U.S. market launch based on anticipated approval of a pending new drug application (NDA), an abbreviated new drug application (ANDA), or a biologics licensing application regulated by FDA’s Center for Drug Evaluation and Research (CDER). The guidance describes the procedures for making such a request to FDA before final approval of the application and provides factors the Agency will consider in granting such requests. FDA offers the following procedural information in the Final Guidance: (1) what information should be submitted to FDA in a PLAIR; (2) when and how to submit a PLAIR; and (3) the circumstances under which the Agency intends to grant a PLAIR.

The Final Guidance finalizes and updates the draft guidance of the same title issued on July 24, 2013. Click the link below for a summary of the major provisions in the Final Guidance, as well as our takeaways to assist industry in preparing and submitting PLAIRs.

Read more here.

First autopsy-validated, highly accurate, and minimally invasive DISCERN™ test is now available to support a clinician’s definitive diagnosis of Alzheimer’s Disease versus other, possibly curable conditions, giving individuals an opportunity to get the right treatment plan sooner—while giving families greater peace of mind. 

May is Older Americans Month (OAM) and a great time to focus on the needs of older Americans to better help them remain in their homes and live independently for as long as possible. Optimal health is an important concern for many older adults, especially for those facing the symptoms of dementia.

When dementia symptoms develop, it’s important to know if an individual is exhibiting Alzheimer’s disease (AD) or some other condition that can be curable or managed with different interventions. An early diagnosis of AD can help to alleviate worry for older Americans and get them on the right AD therapeutic journey sooner.

Fortunately, there is good news: the first highly accurate, minimally invasive skin test supporting a clinician’s definitive diagnosis of AD versus other forms of dementia, even in early disease, is now available. This test is designed to accurately assess the loss of synaptic activity in the brain, where memories lie, due to AD.

Right Diagnosis

Typically, AD can take years to diagnose. The condition tends to progress slowly and affects people in different ways. On average, a person with AD lives four to eight years after diagnosis, but can live as long as 20 years, depending on other factors.

Without a definitive diagnosis, many families are left with a frustrating and expensive uphill battle in the care journey.

Early Diagnosis

An early AD diagnosis allows patients to get treatment sooner, saving time, money and the hopelessness that patients and caregivers often feel when they don’t know what to do. Early diagnosis also gives patients the chance to have a say in their care.

Now that there are more treatment options becoming available for people with AD dementia, having an accurate diagnosis of AD earlier in the disease journey is of growing importance to older Americans.

During Older Americans Month, we hope that more people will take the time to consider how to improve quality of life for the older people in their lives, including new ways to help them age in place and get the best possible healthcare.

To learn more visit here.

As Executive Vice President, Chief External Innovation, Medical Safety and Global Public Health Officer for The Johnson & Johnson Family of Companies (Johnson & Johnson), Dr. Bill Hait talks about Johnson & Johnson Innovation’s mission to source innovations both inside and outside of its walls that have the potential to transform human health. He discusses how collaboration in the innovation ecosystem has enabled access to key healthcare data and deep insights that alter the way we look at addressing rapidly changing human health challenges.

The COVID-19 pandemic inspired scientists around the globe, from government, academia and industry, who collaborated to address our generation’s greatest healthcare challenge. Beginning with the rapid reporting of the nucleotide sequence of the SARS-CoV-2 virus to the discovery and development of new vaccines, monoclonal antibody therapies and small molecule antivirals, the speed by which our industry responded was breathtaking.

The pandemic also magnified the shortcomings of our healthcare systems, where people living in certain countries, or even in certain regions of the same country, did not have adequate access to lifesaving vaccines. The fundamentals of public health were never more important or more challenged by the imbalance of resources and the unpredictability of human behavior.

As we prepare for the future, we at Johnson & Johnson feel our efforts will require new ideas, a new generation of entrepreneurs and the deployment of new tools for scientific inquiry to generate, aggregate and analyze data, develop insights and test hypotheses that could inform the development of new ways to prevent, intercept and cure disease.

It is with this sense of urgency that we at Johnson & Johnson source innovation wherever it originates. Our commitment to innovation is grounded in Our Credo, which details our responsibilities to the patients, doctors and nurses, mothers and fathers we serve, along with our employees, communities and shareholders. It calls on us to experiment with new ideas, carry out research and develop innovative programs. It is also our moral compass, calling us to operate ethically, safely and with integrity.

Johnson & Johnson Innovation connects the external world of invention to internal experts throughout Johnson & Johnson. We have several ways of collaborating, including licenses and collaborations through our Innovation Centers and Business Development groups, incubation space in JLABS, and equity investments through Johnson & Johnson Innovation – JJDC, Inc (JJDC).

Our evaluation criteria for early collaborations are straightforward¹ :

1. If successful, will the opportunity be transformational?
2. Are the people with whom we are investing likely to succeed?
3. Is there a “killer experiment” that allows de-risking the opportunity?
4. Are the deal terms fair to both parties?

We also consider the diversity of the potential partner’s team as another metric of potential success.

We feel our approach to scientific collaboration has created a wealth of opportunities to advance the development of innovative products, identify and address unmet medical needs, and support healthy longevity. For a few recent examples, please visit our Johnson & Johnson Innovation news page and watch this short video featuring members of the Johnson & Johnson Innovation network.

Our recent experiences have taught us that we can achieve healthcare “miracles” when we collaborate, innovate and ultimately celebrate our contributions for better health for all humanity.

¹ Hait, WN and Stoffels, P. A primer for academic entrepreneurs on academic-industrial partnerships.
Nature Comms. 12: 5778-5781, 2021

The record number of Biotech companies that have gone public in recent times is not only impressive in terms of the amount of money these companies have made but also the vast number of firms that have managed to achieve public status. However, while getting public status is a hard road to navigate, staying public can be an even bigger challenge!

Going Public Has Never Been Easier

It is important to note that while going public is a great achievement, it has become easier in more recent times in the US. This is mainly due to the amount of money that is up for grabs and because firms are changing the way they work towards going public. Some of the main changes include:

• Going Public Earlier – around ten years ago, a company would, on average, achieve IPO status in ten years. The current timeframe is just four!
• Having More Money – interestingly, new IPOs tend to have more than double to spend than their counterparts ten years ago, but they are also spending it at a faster and higher rate.
• Improved Valuation – IPO valuations have also gone up more than three times from ten years ago, with the average being around $500 million!
• Special Purpose Acquisition Corporations (SPACs) – these companies have boomed during the pandemic with a 320% increase between 2019 and 2020. This alternative way to become public is more streamlined and has been the driving force behind large numbers of biotechs going public.

While it may be easier and more financially beneficial to go public earlier, this change in dynamic has also reduced the covetable nature of the process, reducing it to just another step along the way.

Working Hard to Stay Public

It would be unfair to say that getting public status is simple without sharing the fact that remaining in that position is incredibly hard. For example, public exchanges have listing requirements that can make continued compliance challenging.

When you stop to consider that so many biotech companies have gone public, getting notoriety has become harder than ever before. The first two to three years of being a public company involve getting noticed by analysts, so investors pick up stock rather than becoming the company that suffers eternal obscurity. In reality, with so many different offers available, achieving a core position with institutional investors can be challenging.

Top Tips for Continued Success

There are lots of theories about how firms can work to stay successful; the following tips are sure to help you plan in advance and get the results you are looking for:

• Don’t Be Complacent – no matter how quickly a biotech goes public; you should consider the financial and operational frameworks necessary to enable continued success.
• Spend Only When Needed – having cash thrown at you can leave you tempted to go wild and start a myriad of new projects. However, taking a more frugal approach will help your company cope during tough times. Analyze each expense and only invest in what will improve your outcomes.
• Expect Hard Times – no matter how great your offer is, every firm will experience tough times. Planning for these times will not only help you manage them effectively but will also ensure that they don’t derail your company’s goals.

The changing nature of a biotech company’s relationship with public markets is something for any conscientious executive to be aware of. A lack of understanding could mean making the wrong decision about when to go public and maintaining public status can be more difficult than achieving it.

Imagine you need to find a friend inside a crowded stadium, but you don’t have their exact location or a way to contact them. Examining every seat, restroom and food vendor would take hours or maybe days. What if there was a way to sort every single person at that stadium based on their visual traits (e.g., male, brown hair, glasses, beard), so that you could separate out those who do not fit that person’s description into one area and all that do into another area? Then the technology could pull up a high-resolution photo of each person identified as having similar traits, so that you could visually confirm the right person and their location. And you could perform this scan at a rate of 15,000 people per second — or in other words, you could find one person in a stadium of 100,000-plus in under 10 seconds.

For immunologists or functional genomics researchers looking to develop better cancer treatments or personalized medicine—who may often be looking for a needle-in-the-haystack type of rare cell among billions of cells—a new BD technology featured on the Jan. 21, 2022 cover of Science unlocks the potential to do what was previously impossible. In the newly published study, the European Molecular Biology Laboratory (EMBL) and BD demonstrated that this technology identifies at high speeds, not only if and how much of a biomarker is present in a cell, but also a biomarker’s location and other detailed information that could help scientists answer complex biological questions faster.

Why this novel technology represents a landmark advancement for the flow cytometry and cell sorter instruments these scientists rely on

The new technology has the potential to transform immunology, cell biology and genomics research and enable new cell-based therapeutic discovery. Until now, the flow cytometry instruments that researchers relied on to analyze and sort cells enabled them to characterize a large volume of cells for a limited number of traits at a time. For example, current technology allows a researcher to use a dye to make a biomarker on or in the cell that they want to study, such as a protein, fluoresce in a bright color relative to its abundance on or in the individual cells. The lasers that excite the dyes and the corresponding fluorescence emitted enables researcher to know whether the protein is present or not and how many cells had it. However, that was as specific as we had been able to get on this type of high-throughput instrumentation. Researchers seeking more detailed information about specific cells would need to look at individual cells under a microscope to manually identify each one, which is time consuming and is limited by your ability to sort each cell by visual inspection alone.

This breakthrough technology, in essence, marries the high throughput of a flow cytometer with the imaging capability of microscopy followed by cell sorting. By integrating cell imaging and image-based decisioning to sort cells at exceptionally high speeds, it’s now possible to identify, characterize and sort millions of cells in minutes, not hours (or days), based on the visual details of each cell and not solely on the type or quantity of biomarkers that are present. If you could apply this technology to the “find a friend” example, we would be able to sort the entire world’s population of 7.8 billion people in a few days—to learn exactly where on the planet that person was and confirm it was them with an image. The potential this technology has for accelerating research timelines unlocks new dimensions in research and opens the possibility of discovering life-saving drugs and other therapies to patients faster.

Building upon multidisciplinary technologies to create a breakthrough

This technology represents the culmination of more than a decade’s worth of work from a multidisciplinary team of optical, mechanical, electrical, biomedical and software engineers and scientists that aimed to provide researchers a differentiated and flexible capability for analyzing single cells. Taking inspiration from beyond the healthcare space, the R&D team leveraged cutting-edge methodologies from tech-forward fields, such as wireless telecommunications, to create state-of-the art capabilities.

By automating some of the traditionally manual aspects of precision identification, the R&D team from our Biosciences business created a technology that pushes the limits of speed and precision in flow cytometry. The technology can analyze more than 1,000 times the amount of data compared to traditional flow cytometry methods. Named BD CellView™ Image Technology, this technique can capture multiple images of individual cells flowing through the system at speeds up to 15,000 cells per second and also adds a previously impossible capability of sorting cells based on detailed image analysis of individual cells at this speed.

Unlocking new frontiers from research to advancing precision medicine

By enabling scientists to more rapidly view and isolate cells with specific, observable traits of interest, this technology can accelerate discovery research and unlock potential therapies or cures for disease in a broad range of fields, such as virology and oncology—and we’ve just scratched the surface of the types of analyses that are possible. The incredible precision and ease of use for the technology opens new possibilities for researchers in a variety of fields from HIV therapeutics to plant biology. While the full potential for this technology is still unknown, it could transform what’s possible in a wide range of disciplines from immunology to genomics research to cell-based therapeutics and beyond.

BD has been a pioneer in the space since the 1970s, when it became the first company to commercialize flow cytometry technology. Since then, we’ve continued building upon that unparalleled expertise and capabilities leadership, continuing to attract the best and brightest in the field to our R&D team, where they can leverage the breadth and depth of resources BD provides. This team has successfully advanced flow cytometry technology to fill a long-standing gap in research where no other company has. We look forward to working with the scientific community at-large to help them leverage it and advance both research and therapeutic discovery, ultimately advancing the world of health™.

Learn more about why Science chose the study from EMBL and BD as its cover story

Knowing the Difference: Alzheimer’s Disease vs. Other Types of Dementia 

Alzheimer’s disease (AD) is the most common type of dementia, but there are many other kinds of dementia, including Lewy body dementia, Huntington’s disease and non-Alzheimer’s dementias. Not a normal part of aging, dementia is caused by damage to brain cells that affects an individual’s ability to communicate, which can affect thinking, behavior and feelings. AD accounts for 60-80% of dementia cases and afflicts an estimated 5.8 million Americans aged 65 and older.

An early diagnosis of AD can help determine if a patient’s symptoms are truly due to AD or some other conditions that can be curable, such as infections, emotional distress or nutritional deficiencies, such as a vitamin B12 deficiency. What’s more, a definitive AD diagnosis allows patients to start clinical interventions sooner, providing a cost savings for payers, as well as saving time, money and the despair of not knowing for those involved. It has been estimated that early diagnosis of AD could save the health system $7.9 trillion (present value of future costs). Unfortunately, early diagnosis of AD has been costly with poor accuracy.

What is Alzheimer’s Disease?

AD is a type of dementia that affects memory, thinking and behavior. Over time, symptoms tend to increase and start interfering with individuals’ ability to perform everyday activities. In the early stages of AD, memory loss is mild but gradually individuals lose the ability to respond to their environment.

AD is a complex disease with plaques and tau tangles–both abnormal clusters of protein fragments, which many experts believe play a critical role in the clinical symptoms of AD by blocking communication among nerve cells and disrupting processes that cells require. While these are clinical findings associated with AD, there are other factors at work. This is evidenced by a recent paper that demonstrated centenarians with little or no cognitive decline were found to have high levels of plaque and tau at death, while another paper suggested that more than one-third of people studied with mild to moderate AD had minimal levels of plaque accumulation in the cerebral cortex.

Improving Accuracy in Early AD Diagnosis

Unfortunately, most patients with AD are diagnosed in the late stages of the disease, with very few being diagnosed in the preclinical stages when treatment can be the most effective.

Rather than focusing on clinical findings associated with AD, there is a new approach that assesses neural and synaptic loss to diagnose AD in people diagnosed with dementia.  DISCERN™, the first autopsy-validated, highly accurate, minimally invasive test for the definitive diagnosis of AD versus other forms of non-AD dementias and those with AD and other degenerative pathologies, gives patients and families the answers they need, enables providers to make a conclusive diagnosis, and allows payers to establish protocols and prior authorizations for prescribing and reimbursing treatment. It also helps pharmaceutical companies identify appropriate clinical trial participants.

DISCERN™ can be utilized as a tool to manage appropriate patient access to therapies approved in the future, in addition to the clinical and economic benefits of improved early, accurate diagnosis.