Tools for the Analysis of New Biotherapeutic Drug Modalities: A Sponsored Virtual Symposium

Experts divulge where the industry is outsourcing most frequently, when companies should outsource more, and when performing tasks in-house might be best.

Deciding Where to Outsource in Biopharma Development and Manufacturing

Automation enables intensification of downstream processes.

Developing Automation Downstream

Successful scale-up and BLA filings for products manufactured by microbial biomanufacturing require strategic planning.

Outlining Successful Steps for Scale-Up and BLA Filings

This article provides an overview of solutions to address key challenges facing vaccine developers or CDMOs wishing to build their own commercial-scale mRNA production facilities.

Overcoming Engineering Challenges to Enable Commercial Scale mRNA Vaccine Manufacturing

Automation of pharmaceutical packaging saves costs and time, say contract packagers./

Automating Packaging of Bio/Pharmaceuticals

Manufacturers are taking the first steps toward process intensification.

Continuous Processing for Viral Vectors

The growth in demand of viral-vector-based gene therapies drives continuous efforts to improve viral vector manufacturing.

Driving Manufacturing Improvements Through Viral Vector Advances

 Washington Editor, jillwechsler7@gmail.com.

Ten years ago, FDA launched an initiative to expand the role of patients in informing regulators and sponsors on most harmful effects of a disease and primary goals of treatment that can lead to desirable medical approaches. To further advance and refine the process, FDA is highlighting how the Patient-Focused Drug Development (PFDD) program has encouraged active involvement of many patient groups in bringing their perspectives into the process of developing, testing, and approving new medicines.

The primary goal of PFDD is to “better incorporate the patient’s voice in drug development and evaluation,” says Theresa Mullin, associate director for strategic initiatives in the Center for Drug Evaluation and Research (CDER), who has led this effort since its establishment. The initial program featured FDA-led meetings but was expanded to include a wider range of sessions organized by patient groups themselves, Mullin explained in a webinar sponsored by the 

The PFDD initiative was established by legislation reauthorizing the 

 (PDUFA) in 2012. It called for FDA to organize 20 meetings each year to discuss patient experiences, which symptoms have the most significant impact on daily life, and how well current therapies treat those symptoms. The program grew to 24 meetings in the first year as more groups recognized the benefits of hearing patient perspectives on disease severity and on potential effects of treatment. Then in 2016, PFDD further expanded to include discussions organized by patient groups. There have been 55 of these externally led meetings, which follow the format of the agency-organized meetings in being open to the public and producing detailed reports to post on the FDA website.

Every one of these meetings “was very powerful,” Mullin commented, noting that physicians at FDA said that they had never heard these perspectives and that reviewers found that patient comments helpful in assessing the risks and benefits of a new therapy. And even though patients did not advocate for any particular therapies, they discussed the impact of available treatments and where new therapy could have an impact, a process that supported the development of new treatments for sickle cell disease complications.

To further improve the PFDD process, FDA has issued a series of guidance documents to advance the reliability of PFDD tools and methods. Two final guidances address how to 

collect comprehensive and representative input

how to assess which aspects of disease burden and treatment

outlines processes for selecting, developing, or modifying clinical outcome assessments. Still to come is a fourth guidance on incorporating clinical outcome assessments into endpoints to support regulatory decision making. These advisories aim to encourage manufacturers and researchers to utilize high-quality methods for collecting patient experience information that will be useful in regulatory decisions and to move away from multiple differing efforts for developing clinical assessment measures and tools that repeat the PFDD process with every manufacturer.

In looking to the future, Mullin envisions some kind of “national catalog” of standard core clinical outcomes and assessments based on patient perspectives. This would provide reliable measures of clinical outcomes for disease areas that are meaningful to patients and could gain support from FDA’s Standard Core Clinical Outcome Assessments Grant Program. The overarching aim is to ensure that clinical trial protocols are reasonable and accessible for patients and that clinical outcome assessment measures compare things that people care about.

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FDA is highlighting how the Patient-Focused Drug Development program has encouraged active involvement of many patient groups in bringing their perspectives into the process of developing, testing, and approving new medicines.

FDA Expands Patient Input on Drug Development 

The Massachusetts Justice department announced on Sept. 26, 2022 that Biogen had agreed to pay approximately $900 million to resolve allegations concerning submissions of false claims to Medicare and Medicaid. The suit, filed and litigated by former Biogen employee, Michael Bawduniak, under the whistleblower provisions of the False Claims Act, alleged that the company paid kickbacks to physicians to induce them to prescribe Biogen drugs.

According to the department’s press release, the suit alleges that from Jan. 1, 2009, through March 18, 2014, Biogen held programs through which it offered and paid remuneration, such as speaker and consulting fees, to health care professionals to induce them to prescribe three of their drugs: Avonex (interferon beta-1a), Tysabri (natalizumab), and Tecfidera (dimethyl fumarate). These actions would be a violation of the federal Anti-Kickback Statute, which prohibits exchange (or offer to exchange) anything of value to induce or reward the referral of business reimbursable by federal healthcare programs (1).

“Bawduniak doggedly pursued this matter on behalf of the United States for over seven years,” said General Brian M. Boynton, principal deputy assistant attorney and head of the Department of Justice’s Civil Division, in the release. “The settlement announced today underscores the critical role that whistleblowers and their attorneys play in utilizing the False Claims Act to combat fraud affecting federal healthcare programs.”

Under the terms of the settlement, Biogen will pay approximately $844 million to the US and approximately $56 million to 15 states. The US will pay Bawduniak a share of the federal recovery. The claims resolved by the settlement are allegations only and there has been no determination of liability.

United States Department of Justice, District of Massachusetts

1. American Society of Anaesthesiologists, “Anti-Kickback Statute and Physician Self-Referral Laws (Stark Laws),” 

Biogen has agreed to pay $900 million to settle federal and state claims that it paid kickbacks to physicians to encourage them to prescribe its drugs.

Biogen to Settle Kickbacks Lawsuit for $900 Million

The National Institute of Health (NIH) recently unveiled a new process for revealing key details of choroideremia, a rare genetic disorder that leads to blindness. Found through a study by the National Eye Institute (NEI), the approach combines traditional eye imaging techniques with adaptive optics, a type of technology that enhances imaging resolution.

According to an agency press release from Sept. 13, 2022, this approach was developed by Johnny Tam, head of the Clinical and Translational Imaging Unit at NEI. By combining adaptive optics with indocyanine green dye, the team was able to view live cells in the retina, including light-sensing photoreceptors, retinal pigment epithelium (RPE), and choroidal blood vessels. This allowed the researchers to see the extent to which choroideremia disrupts these tissues, information which can be used to design effective treatments for the condition.

The major findings of the study concerned the retinal pigment epithelium (RPE), a layer of pigmented cells needed for the nourishment and survival of photoreceptors. Patients with choroideremia had RPE cells that were significantly enlarged, with some enlarged as much as five-fold; by contrast, photoreceptor and blood vessel layers had less disruptions. According to the release, this suggested to the researchers that RPE disruptions may play a significant role in the disease.

“It’s not obvious at first, but using an existing tool in the clinic, we can monitor and track the cellular status of the RPE layer,” said Tam in the release. “This could prove valuable in identifying which patients would benefit the most from therapeutic interventions.”

A combination of eye imaging techniques and adaptive optics has revealed key details of choroideremia.

Novel Imaging Approach Reveals Key Details About Rare Eye Disease

Actylis, a global manufacturer and sourcing expert of raw materials and performance ingredients, announced its debut on Sept. 26, 2022. The new company combines Aceto and 10 industry specialists into an integrated global ingredient powerhouse; A&C, A&C Bio Buffer, Biotron Laboratories, Cascade Chemistry, Finar, Inter-Actifs, IsleChem, Pharma Waldhof, Syntor Fine Chemicals, and Talus.

According to a company press release, Actylis aims to address the major unmet need for better and more dependable access to critical raw materials and performance ingredients needed for the manufacture of highly regulated products in key industries. The combination of the many specialists will enable Actylis’ hybrid manufacturing and sourcing model, which is designed to provide various benefits to its customers in high-growth end markets.

“Actylis unites multiple industry specialists with a wide range of capabilities into a new, global enterprise with a unique hybrid approach that is greater than the sum of its parts,” said Gilles Cottier, CEO of Actylis, in the release. “This consolidation enables us to offer customers across diverse locations and industries highly flexible, customized solutions addressing their specific needs, while assuring reliable on-time delivery of the high-quality ingredients essential to their success.”

Actylis combines Aceto and its 10 acquired specialists into a singular global brand.

Actylis Debuts as an Integrated Global Specialty Ingredients Manufacturing and Sourcing Provider

Octagon Therapeutics, a preclinical-stage biotechnology company, announced a partnership with Novo Nordisk on Sept. 20, 2022. The research collaboration will use Octagon’s functional target discovery approach and novel chemistry strategy in disease areas in which Novo Nordisk has specific expertise.

The program selectively targets a population of pathologically activated B cells that contribute to autoimmune diseases like systemic lupus and vasculitis. According to a company press release, this approach can be applied in other disease areas to target disease-driving processes while retaining healthy immune functioning. A recent pilot project, initiated through Novo Nordisk’s Co-creation Greenhouse Accelerator program, demonstrated proof-of-concept in identifying atypical characteristics of other immune cells during disease; this ultimately led to the discovery of new therapeutic targets.

"Octagon has a unique platform technology to look into functional biology in a novel way.It makes use of clinical samples and primary immune cells to identify new drug targets,” said Uli Stilz, vice-president, Bio Innovation Hub, Novo Nordisk, in the press release. “Combined with our disease understanding in the cardiometabolic space and Octagon´s approach in targeting specific lymphocyte populations that drive disease progression, it will be exciting to see what therapeutic discoveries the collaboration can lead to."

"Novo Nordisk has a strong expertise in diabetes and related cardiometabolic conditions like [nonalcoholic fatty liver disease] and kidney disease." said Isaac Stoner, CEO, Octagon Therapeutics, in the press release."This collaboration will enable Octagon to expand our technology into these disease areas and to further leverage the impressive clinical sample collection that Novo Nordisk has available."

The collaboration will use Octagon’s functional target discovery approach and novel chemistry strategy in disease areas in which Novo Nordisk has specific expertise.

Novo Nordisk and Octagon Therapeutics Enter Inflammatory Disease Research Collaboration

Alcami, a US-based pharmaceutical contract development and manufacturing organization (CDMO), announced the addition of a 12,000 ft² solid-state characterization laboratory to its Morrisville, NC laboratory on Sept. 22, 2022. According to a company press release, the expanded laboratory is designed to increase the company’s NC footprint and complement the company’s existing sterile and oral solid drug product development, manufacturing, and analytical capabilities for products in all phases of development.

"We look forward to supporting our clients in the discovery of novel polymorph, salt, and cocrystal forms and in the development of robust crystallization and formulation processes," said Elliott Franco, vice-president, Laboratory Operations, Alcami, in the press release. "We have the technical expertise and technologies to provide our clients with a seamless transition from initial characterization through [good manufacturing practice] manufacturing and analytical release.”

The dedicated solid-state research and development suite is designed to promote discovery of novel polymorph, salt, and cocrystal forms.

Alcami Adds Solid-State Characterization Capabilities to North Carolina Laboratory

Pfizer announced on Sept. 22, 2022 an agreement with Global Fund to supply up to 6 million treatment courses of its COVID-19 oral treatment, PAXLOVID as part of its COVID-19 Response Mechanism (C19RM). The C19RM has been the primary vessel for providing grant support to low-and-middle-income countries to purchase COVID-19 tests, treatments, and personal protective equipment.

PAXLOVID treatment courses will be available through C19RM, subject to local regulatory approval by the 132 grant-eligible countries determined by Global Fund based on income classification and disease burden. According to a company press release, Pfizer anticipates supply to be available beginning in 2022, pending regulatory authorization and based on country demand. All low and lower-middle-income countries will pay a not-for-profit price, while upper-middle-income countries will pay the price defined in Pfizer’s tiered pricing approach.

“After so much disruption and loss due to COVID-19, we must continue to accelerate access to PAXLOVID as a treatment option for high-risk patients in all regions of the world along with test and treat programs that help get treatment quickly to those in need”, said Albert Bourla, chairman and CEO, Pfizer, in the press release. “This agreement with Global Fund is a critical step that will boost equitable access for high-risk patients in low-and-middle-income countries.”

Pfizer is set to supply global fund up to 6 million treatment courses of its COVID-19 oral treatment, PAXLOVID, for low-and-middle-income countries.

Pfizer to Supply up to 6 Million PAXLOVID Treatment Courses for Low-and-Middle-Income Countries

Roche announced on Sept. 19, 2022 that the European Commission (EC) has approved Vabysmo (faricimab) for the treatment of neovascular or “wet” age-related macular degeneration (nAMD) and visual impairment due to diabetic macular edema (DME). These conditions are two of the leading causes of vision loss worldwide, affecting more than 40 million people, according to a company press release.

Faricimab is currently the only injectable eye medicine approved in Europe. The treatment will begin at intervals of up to four months for people with nAMD and DME, with the potential to require fewer eye injections over time. The treatment is a bispecific antibody designed to target and inhibit two disease pathways to restore vascular stability.

“Many people with nAMD and DME struggle to keep up with the monthly eye injections and physician visits, often associated with current standards of care, and unfortunately their vision may suffer as a result of undertreatment,” said Ramin Tadayoni, head of the ophthalmology department, Lariboisière, Saint-Louis and Rothschild Hospitals, Paris, France, and European Society of Retina Specialists (EURETINA) president elect, in the release. “For people in Europe living with these conditions, today’s approval offers the first new mechanism of action in over a decade; one which could improve and protect their vision with fewer injections over time.”

EC has approved Roche’s Vabysmo, the first bispecific antibody for the eye, for two leading causes of vision loss.

EC Approves Vabysmo for Two Leading Causes of Vision Loss

Roquette, a French excipient provider, announced on Sept. 20, 2022 that it would be fully acquiring Crest Cellulose, an Indian manufacturer of excipients. This move, which follows Roquette’s decision to purchase a majority stake in 2018, will make the company a wholly owned subsidiary of Roquette. According to a company press release, Roquette identified India as a major area of growth in the near future and purchased Crest to reinforce their presence in India and Asia overall.

“At Roquette, we believe true innovation can only be achieved in an environment where ingredient safety is completely assured,” said Paul Smaltz, vice-president, Roquette Pharma Solutions, in the press release. “There’s no room for compromise or shortcuts when tackling the world’s greatest health challenges. The team at Crest Cellulose share our conviction that quality is king, always striving to optimize processes and redefine what’s possible in drug delivery. As a global leader in health and nutrition, we’re excited to harness our newly expanded capabilities to support our network of customers and partners in India, Asia, and the rest of the world.”

Roquette’s acquisition of Crest Cellulose is designed to reinforce its presence in India and other countries in Asia.

Roquette Acquires Indian Excipient Manufacturer

As the deadline nears for enacting legislation to renew user fees to fund significant FDA operations, legislative leaders are looking to approve a bare-bones measure as part of a short-term government funding bill. Congress must enact a continuing resolution (CR) by Oct. 1, 2022, to prevent a federal government shutdown, and the legislators now have agreed to include a five-year fee reauthorization in that measure. Such a CR for fiscal year 2023 is expected to run through Dec. 16, 2022, after the upcoming mid-term elections.

A key point of debate is whether to include in the CR package provisions to continue and revise a number of FDA programs also slated to expire next week. These and a number of broader reforms were included in the user fee reauthorization bill that was approved by the House months ago and has been pending in the Senate for weeks. It would make significant changes in FDA programs and operations, including policies for how FDA regulates diagnostics, dietary supplements, and cosmetics.

Also off the table is the proposed Cures 2.0 legislation, which was pulled from the main user fee package due to Republican opposition. That measure supports and revises a range of programs to advance biomedical research, but now its fate is uncertain.

Without reauthorization of new fees for drugs (Prescription Drug User Fee Amendments [PDUFA]), generic drugs (Generic Drug User Fee Amendments [GDUFA]), biosimilars (Biosimilar User Fee Amendments [BsUFA]), and medical devices (Medical Device User Fee Amendments [MDUFA]) before the current fee programs expire next week, FDA will lose its authority to collect fees from manufacturers, along with requirements for meeting certain timeframes and for utilizing agreed-on policies for assessing and approving new drugs and medical products.

FDA also would have to lay off thousands of staffers who are supported by fee revenues of potential layoffs, a prospect that has eroded agency morale and undermined efforts to recruit and hire needed employees. The agency has struggled for months to maintain and expand its professional workforce in a very competitive market for skilled biopharma experts, and the agency’s funding uncertainty has worsened the situation.

Medical product user fees generate approximately $2 billion in agency revenues, about 40% of FDA’s non-tobacco budget. While Congressional leaders on both sides of the aisle support the agency’s programs as important for ensuring the health and safety of Americans, the broader political debate over government funding has erected added hurdles for timely reauthorization of new user fee programs. And some consumer advocates have broadened their criticism of expanded user fee funding of FDA by regulated industry as leading toward bias and undermining agency decision-making. FDA Commissioner Robert Califf has said that the agency has sufficient carryover funds to maintain operations for about five weeks into the new fiscal year that begins Oct. 1, 2022, but FDA still will have to notify more than 3000 staffers of “impending employment actions” without user fee reauthorization by Congress.

Legislative leaders are looking to approve a bare-bones measure to fund FDA operations as part of a short-term government funding bill. 

Congress Slated to Reauthorize User Fees Without FDA “Improvements” 

Adam C. Fisher, Phd, is Director of Science Staff, Office of Pharmaceutical Quality, Center for Drug Evaluation and Research, FDA.

Pharmaceutical manufacturers employ sophisticated strategies to shorten the time to product launch, but how many consider adopting an advanced manufacturing technology as a strategy to do so? It turns out that manufacturers who have adopted continuous manufacturing have shortened their time to FDA approval and their time from approval to product launch. Might this strategy work for others?

When Stelios Tsinontides joined FDA as director of the Office of Pharmaceutical Manufacturing Assessment in October 2019, he saw the full landscape of global pharmaceutical manufacturing for the first time. He noted there were only six FDA-approved applications using continuous manufacturing for solid oral drug products. Despite his 29-year career in the pharmaceutical industry, he was puzzled as to why so few manufacturers had embraced continuous manufacturing. “I just assumed that many more pharma manufacturers would have embraced this technology given its widespread adoption in other industries,” he explains. Then he set out to understand why. “I heard through our engagements with manufacturers that they had concerns about FDA’s application review delaying their product launches as well as some concerns related to postapproval changes and inspections,” he says.

Now concerned himself, Tsinontides initiated an internal audit of applications to FDA’s Center for Drug Evaluation and Research (CDER), and the findings surprised even his most seasoned colleagues. Rather than delaying product launches, the audit found that FDA had approved applications employing continuous manufacturing three months faster than those employing traditional batch manufacturing. What’s more, these products also hit the market faster after approval. Based on the sales figures for these drugs, these continuous manufacturers enjoyed $100 million–$500 million of earlier revenue based on their shortened launches. “Once I saw these numbers, I knew based on my industry experience that this story needed to be shared broadly with pharma decision-makers,” he says.

Tsinontides wasn’t the only one curious about the slow adoption of continuous manufacturing for pharmaceuticals. In 2020, Clifford Rossi, a professor at the University of Maryland School of Business and a financial risk expert, began to explore the financial aspects of continuous pharmaceutical manufacturing. “For decades, it’s been significantly cheaper to manufacture overseas, especially in China,” he says. “I assumed that pharma manufacturers were not investing in continuous manufacturing due to the financial risks and economics, and perhaps even due to tax rates.”

To investigate further, Rossi developed a stochastic financial model that also showed him something different than expected. Continuous manufacturing in new facilities provided economic benefits and even an economic advantage for US manufacturers as compared to batch manufacturers abroad. He found the contribution of continuous manufacturing to the expected net present value over the life cycle of a drug to be nearly $500 million. “When comparing investments in continuous or batch strategies for a new US facility, the results clearly suggest that the lower costs associated with continuous manufacturing should lead to more investment in it—even for generic firms,” he says. “It seems that continuous manufacturing may not only be worth the investment, but its adoption could also help to reshape global pharmaceutical supply chains.”

As the data from CDER’s audit and the University of Maryland study began to trickle in, Tsinontides started to wonder how FDA had approved applications with advanced manufacturing technology so fast. He reached out to his colleague Sau “Larry” Lee, PhD, senior scientific advisor to CDER’s Emerging Technology Program, to understand. “For those wishing to adopt a new technology, CDER created an Emerging Technology Program to allow early engagements with the agency, even prior to filing a regulatory submission,” informs Lee.

Formed in 2014, the program allows an organization to meet with FDA experts from review and inspection programs to discuss and resolve potential regulatory issues prior to filing a submission. It turns out that all of the FDA-approved continuous manufacturing applicants had engaged with CDER’s Emerging Technology Program. “We’ll engage with those adopting new technologies, and our early interactions do not even need to be product-specific,” says Lee.

The application to participate in the Emerging Technology Program is a straightforward document with a five-page limit that includes basic items such as a description of the technology and an explanation as to why it is substantially novel. “It’s very important that applicants to the program describe how their proposed new technology could potentially improve product quality,” Lee notes. “We try to develop familiarity and internal expertise on their technology so we can carry that forward to the application assessment and facility inspection.”

Pharmaceutical manufacturers that move toward adopting advanced manufacturing benefit from increased support from FDA in the short term and can reap the rewards of using an advanced technology in the long term. The Emerging Technology Program has not only been effective in helping manufacturers expedite their product launches, but it is also well-received by participants who rate their satisfaction with the program an 8.9 out of 10 in exit interviews. Of course, a technology cannot remain emerging forever. This high level of regulatory engagement on continuous manufacturing will not continue indefinitely as technologies mature and adoption becomes more widespread.

Engagements with the program may explain how continuous manufacturing applications were approved quickly, but Tsinontides still wondered how these products had hit the market faster after approval. Most pharma manufacturers employ batch manufacturing, a process in which material is manually moved from one unit operation to the next, and product is made one batch at a time. Drug manufacturing has largely been conducted this way since the middle of last century. “After approval, batch manufacturers typically need to complete scale-up transfer, which can involve qualifying larger equipment, updating processes, and completing performance qualification for the commercial-scale process,” explains Tsinontides. “All of this takes time and effort.”

Many industries such as commodity chemicals and food processing have embraced continuous manufacturing as their default means of production, due to advantages including shorter processing times, reduced waste, and a smaller footprint. Continuous manufacturing uses an unbroken flow of material from one unit operation to the next with no stops in between, bringing opportunities for improved efficiency and fewer errors. “Continuous manufacturing also allows for agility related to production timing and rate, and is not necessarily a 24/7 production,” says Tsinontides.

While multiple factors can determine the time between drug approval and marketing, manufacturing scale-up by increasing equipment size is often the bottleneck. Continuous operations, by design, require fewer process and equipment changes to reach commercial-scale operations. Changes to accommodate the commercial scale in continuous processing typically involve simply increasing the run time on the same equipment and with the same controls. The decrease in time to market after approval, alone, accounted for $50 million–$125 million of the early revenue for continuous manufacturers.

FDA’s audit also looked at the regulatory burden for those adopting continuous manufacturing, related to making postapproval changes and being subject to pre-approval inspections. The FDA audit found that there was not a substantial difference in the number of process changes reported by batch and continuous applicants. However, continuous applicants reported no changes related to equipment, processing conditions, or batch-size range. Tsinontides says, “This observation aligns with the fact that continuous processes are often designed to have inherent flexibility to tailor production to demand.”

An FDA pre-approval inspection was conducted for the majority of batch products and all of the continuous products. “While continuous applications had more process-related issues observed during inspections than batch applications, it stands to reason that as FDA’s confidence in continuous manufacturing increases, the need for pre-approval inspections and the number of process-related issues should lessen,” contends Lee. The most important finding for continuous manufacturers was that pre-approval inspections did not adversely impact application approval timelines, as all were approved in the first FDA review cycle ahead of the goal date.

Owing to his engagements with technology developers in the Emerging Technology Program, Lee found another issue faced by manufacturers wanting to adopt continuous strategies. Many had faith in FDA’s willingness and ability to approve continuous manufacturing applications but had doubts about that of other global regulators. Often manufacturers deal with dozens of global regulators for a single international product launch.

To address this problem, Lee initiated an international effort to describe harmonized scientific and regulatory considerations for continuous manufacturing. The result was an international guideline on continuous manufacturing dubbed ICH Q13. “The goal of ICH Q13 is to commit regulators to consistent adoption of consensus-based guidelines to realize the benefits of continuous manufacturing for patients as well as industry,” says Lee. The ICH Q13 guideline has undergone global public consultation and is expected to be finalized before the end of the year. FDA’s hope is that manufacturers will have more confidence in the global regulatory support for continuous manufacturing adoption.

As continuous manufacturing transitions from the innovators to the early adopters in pharma, it seems the question is when, not if, pharma will broadly embrace continuous manufacturing. The rapidly changing demand caused by COVID-19 displayed the value of manufacturing agility and the problems caused by long, tangled supply chains and time-consuming manufacturing technology transfers. Many saw continuous manufacturing as a potential solution to these problems.

As continuous manufacturing hit the spotlight in 2020, so did its funding. The federal government alone awarded nearly $500 million to companies developing domestic continuous manufacturing technologies. Some of these technologies now approach maturity and regulatory filings, leaving FDA in a challenging but promising position.

Adam C. Fisher, PhD is Director of Science Staff, Office of Pharmaceutical Quality, Center for Drug Evaluation and Research, FDA.

FDA backs the benefits of adopting continuous manufacturing, a strategy which has accelerated approval and launch timelines, and its Emerging Technology Program could be a helpful tool.

The Manufacturing Edge on Path to Market

FDA and the National Institutes of Health (NIH) announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership, on Sept. 14, 2022. The goal of this partnership is to advance the understanding and develop treatments for amyotrophic lateral sclerosis (ALS) and other rare degenerative diseases.

The Critical Path Institute (C-Path) will convene the partnership, bringing together rare neurodegenerative disease experts, including patient communities, advocacy organizations, private entities, and more. According to an agency press release, areas of focus will include patient-focused drug development, characterization of neurodegenerative diseases and their natural history, the identification of molecular targets for neurodegenerative disease, and increased efficiency, predictability, and productivity of clinical development of therapies.

“There is a crucial need to develop new treatments that can improve and extend the lives of people diagnosed with rare neurodegenerative diseases, including ALS. Collaboration across public and private sectors can accelerate the progress to address this urgent need,” said Hilary Marston, chief medical officer, FDA, in the release. “The partnership we are announcing today will leverage the shared expertise of all participants to create a path towards new breakthroughs in treating these diseases. We look forward to working with NIH, C-Path, and other public and private partners to carry out this important effort.”

“This public-private partnership will convene the entire ALS community to develop novel strategies and approaches to therapy development and clinical testing with the goal to finally produce a treatment that stops the tragic progression of ALS,” said Walter Koroshetz, director of the National Institute of Neurological Disorders and Stroke, part of the NIH, in the release.

The Public-Private Partnership for Rare Neurodegenerative Diseases will work to enhance understanding and develop treatments for rare neurodegenerative diseases.

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