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FDA Issues Final Guidance on "Emerging" Drug Manufacturing Technologies

The FDA has issued final guidance for biopharmas seeking to incorporate what the agency deems “promising emerging and novel technologies” in their drug manufacturing processes. The final guidance, titled “Advancement of Emerging Technology Applications for Pha

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The FDA has issued final guidance for biopharmas seeking to incorporate what the agency deems “promising emerging and novel technologies” in their drug manufacturing processes.

The final guidance, titled “Advancement of Emerging Technology Applications for Pharmaceutical Innovation and Modernization,” offers recommendations to companies that are interested in participating in the FDA’s Emerging Technology Program.

Through the program, biopharmas begin and maintain a dialog with the FDA before filing regulatory submissions related to new drugs when their manufacturing process incorporates what the agency calls “innovative and novel technologies that have the potential to improve drug product safety, identity, strength, quality, and purity.”

Dialog occurs as biopharma representatives submit questions and proposals to officials on the FDA’s Emerging Technology Team (ETT), with the goals of discussing, identifying, and resolving potential concerns regarding the development and implementation of novel technologies in advance of regulatory submissions.

“We’re at an inflection point in the practice of medicine, not only in terms of novel therapies, but also when it comes to the advanced technology being used to manufacture these products,” FDA Commission Scott Gottlieb, M.D., said in a statement. “The FDA is committed to continuing to foster advances in innovative pharmaceutical manufacturing, which ultimately has the potential to improve drug quality and safety.”

Michael Kopcha, Ph.D., R.Ph., wrote in a September 11 post on the agency’s blog FDA Voice that two biopharmas have engaged with the ETT before implementing continuous manufacturing (CM) processes. One is Janssen Therapeutics, which last year won FDA approval to change its manufacturing process for from HIV-1 treatment Prezista® (darunavir) from batch to CM. The other company, Vertex Pharmaceuticals, has used CM for its cystic fibrosis drug Orkambi® (lumacaftor/ivacaftor) since it was approved by the agency in July 2015.

The final guidance came a week after the agency’s September 21 deadline for public-docket submissions about issues related to the science, technology, and best practices of CM. “FDA’s goal is to provide a framework of principles that clarify our expectations, while still encouraging companies to innovate and implement CM,” Dr. Kopcha stated.

‘Emerging’ Examples

In issuing its final guidance yesterday, the FDA also offered several examples of emerging technologies it would potentially consider, which fall under three categories:

  • Small molecules: Includes CM of drug substance; CM of drug product; model-based control strategy for CM; continuous aseptic spray drying; 3D printing manufacturing; and ultra-long-acting oral formulation.
  • Biological molecules: Includes controlled ice nucleation for lyophilization processes; advanced process control, such as predictive modeling for process monitoring and closed-loop bioreactor control; multiattribute method; next-generation sequencing; CM for an upstream process; and “pharmacy on demand,” a small-manufacturing platform for continuous bioprocesses.
  • Multiple products: Includes closed aseptic filling system; isolator and robotic arm for aseptic filling; novel container and closure systems for injectable products

“Emerging technology should be novel in the context of the pharmaceutical and related industries and it should have the potential to modernize the pharmaceutical manufacturing body of knowledge related to product quality,” according to a footnote within the guidance. “Emerging technology will be new to FDA in the context of pharmaceutical quality, with limited prior experience and knowledge.”

The FDA envisions interested biopharmas participating in the Emerging Technology Program before a wide range of submissions reviewed by the agency’s Center for Drug Evaluation and Research (CDER). These include beyond Investigational New Drug applications (IND), original or supplemental New Drug Applications (NDA), Abbreviated New Drug Applications (ANDA), Biologic License Applications (BLA), or application-associated Drug Master Files (DMF). DMFs were not included in the draft version of the guidance, issued by the FDA in 2015.

“Based on experience gained during the program, FDA intends to develop guidance and standards, as necessary, on emerging technologies and approaches to encourage and facilitate the innovation and modernization in pharmaceutical industry,” the final guidance states.

Connected reading

Helpful context for this guide

Source-derived material selected through this article’s indexed topics.

Related questions

01When to see a doctor

If you or your child has symptoms of cystic fibrosis — or if someone in your family has CF — talk with your healthcare professional about testing for the condition. Make an appointment with a doctor who has skills and experience in treating CF. CF requires regular follow-up with your healthcare professional, at least every three months. Call your healthcare professional if you have new or worsening symptoms, such as more mucus than usual or a change in the mucus color, lack of energy, weight loss, or severe constipation. Get medical care right away if you're coughing up blood, have chest pain or trouble breathing, or have severe stomach pain and bloating. Call 911 or your local emergency number or go to the emergency department at a hospital if: You're having a hard time catching your breath or talking. Your lips or fingernails turn blue or gray. Others notice that you're not mentally alert.

Source: www.mayoclinic.org ↗
02What you can do

You might want to take a friend or family member with you to the appointment to help you remember information. Before your appointment, make a list of: Symptoms and when they started. Include anything that makes symptoms worse or better. All medicines, vitamins, herbs and supplements that you or your child take. Include the doses. Family history, such as whether anyone in your family has cystic fibrosis. Treatment you or your child have had for CF, if any. Include what the treatment was and if it helped. Any other medical conditions and their treatments. Questions to ask your healthcare professional. Questions to ask may include: What is likely causing these symptoms? What kinds of tests are needed? What treatment do you recommend? I or my child have other health conditions. How will cystic fibrosis affect them? Are there any limits needed? Feel free to ask other questions during your appointment.

Source: www.mayoclinic.org ↗
03What Is Cystic Fibrosis?

Cystic fibrosis (CF) is a genetic disorder, which means you get it from your parents at birth. It affects your lungs, pancreas, and other organs. CF changes the way chloride (salt) moves through the cells of your body. This causes the mucus (which should be thin and slippery) in various organs to become thick and sticky. Over time, this thick mucus builds up inside your airways, making it hard to breathe. The mucus traps germs and leads to infections and inflammation. It can also cause severe, long-term damage to the lungs and lead to respiratory failure (inability to breathe normally) and death. In the pancreas, the thick mucus caused by CF prevents the release of digestive enzymes when you eat. This leads to malnutrition and poor growth. CF can also cause liver disease, reproductive problems, and cystic fibrosis-related diabetes (CFRD). More than 40,000 people in the U.S. live with CF. Doctors diagnose about 1,000 new cases each year. Today, more than half of the CF population is aged 18 or older, and new treatments have expanded the life expectancy by decades.

Source: www.webmd.com ↗
04Is There Any Cost Assistance Available?

There is a savings coupon available from the drugmaker that may allow you to pay as little as $0 for your prescription. Whether you are eligible depends on whether you have prescription insurance and what type of insurance you have. You can find out more at Vertex GPS™ Patient Support Program | ALYFTREK™ (vanzacaftor/tezacaftor/deutivacaftor) .

Source: www.webmd.com ↗
05What is it used for?

A sweat test is used to diagnose cystic fibrosis (CF).

Source: medlineplus.gov ↗
Research context

Read sources and limitations before applying a claim.

Research and Statistics: Who Has Cystic Fibrosis?

About 40,000 people are living with cystic fibrosis in the United States, and there are approximately 105,000 people with CF worldwide. (3) More than 75 percent of people with the disease are diagnosed by age 2, and more than half of all people living with cystic fibrosis are 18 or older. CF occurs predominantly in white populations, at a rate of 1 in 2,500 births. Between 2 and 5 percent of white people are carriers of the CFTR gene variant but have no overt clinical signs of disease. The disease is less common among African Americans, occurring at the much lower frequency of approximately 1 out of 17,000 births. (15) CF gene variants are most prevalent in persons of northern and central European ancestries or of Ashkenazi Jewish descent. They are rarely found in Native Americans, Asians, or native Africans. (16) CF is equally common among men and women, but women patients fare significantly worse than male patients with the disease. The median survival age for female CF patients is about three years younger than it is for men, but the reasons for the poorer survival rates among women are not completely understood. (17)

Source: everydayhealth.com ↗
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