From post-aging performance testing to container closure integrity, robust design controls extend far beyond “constituent part” requirements.

With the increase in complexity of some combination products and co-packaged kits, the need remains for ensuring that the patient gets the right drug at the right dose at the right time. While it is acceptable to make improvements to products as more information becomes available, it’s important to recognize that a change to a co-packaged kit can result in a warning letter if design controls are not properly implemented.

In her presentation at the 2017 PDA Annual Meeting in Anaheim, CA, Tracy TreDenick, Head of Regulatory and Quality Assurance and Founding Partner at BioTechLogic, explained that design controls for co-packaged kits include requirements for individual constituent parts, but also include inter-component dimensional and functional specifications, system integration verification testing and shipping of aged components. “FDA is asking for dimensional and functional specs for the final finished form,” she said.

This is not just about the closure of a prefilled syringe, but about the biocompatibility of all parts combined. She noted, “The challenge is understanding the constituent parts and the system integration requirements.” Continue Reading Article

Combination products are a fascinating area of the pharmaceutical industry and present great future promise. The segment is projected to reach $115 billion in global sales by the end of 2019. It has grown solidly at a rate of 7.9% CAGR since 2013, and is projected to continue at that rate through 2019.¹

Some of the key factors driving this growth include: higher levels of patient compliance, demand for minimally invasive surgeries, opportunities for precise pain relief, quicker healing and governments and non-governmental organizations (NGO) embracing combination drugs for their ease of administration.

Combination products defined in 21 CFR 3.2(e)² are therapeutic and diagnostic products that are composed of any combination of a drug, device, or biological products, with the intention of creating safer, more effective, precisely targeted and easier to administrate therapies.

While the technologies and innovations driving the combination product market deliver a great deal of value to patients and to the medical community, the novelty of these products is often challenging for drug developers and regulatory agencies. The marriage of two different disciplines – drug and medical device – creates a complex regulatory process that must be well managed. In addition, evolving regulations as the combination product segment matures can present challenges for older, legacy combination products. Continue Reading

SEE ALSO: The Formation of the Combination Products Policy Council to Improve Regulatory Efficiency

The issue of pharmaceutical data integrity is more important than it has ever been. Data is both the backbone of CGMP compliance and the fuel of the digital economy. Secondly, global regulatory bodies have become increasingly focused on data integrity and have issued many actions of varying severity.

The following collection of resources are the leading guidances and resources for pharmaceutical data integrity.

FDA Draft Guidance: Data Integrity and Compliance With CGMP Guidance for Industry

The purpose of this guidance is to clarify the role of data integrity in current good manufacturing practice (CGMP) for drugs, as required in 21 CFR parts 210, 211, and 212. Part 210 covers Current Good Manufacturing Practice in Manufacturing, Processing, Packing, or Holding of Drugs; General; part 211 covers Current Good Manufacturing Practice for Finished Pharmaceuticals; and part 212 covers Current Good Manufacturing Practice for Positron Emission Tomography Drugs. This guidance provides the Agency’s current thinking on the creation and handling of data in accordance with CGMP requirements. Access Guidance

MHRA GxP Data Integrity Definitions and Guidance for Industry

The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) guidance on data integrity is one of the globe’s leading resources on the topic. Access Guidance

Elements of a Code of Conduct for Data Integrity in the Pharmaceutical Industry

The Parenteral Drug Association’s (PDA) Elements of a Code of Conduct for Data Integrity in the Pharmaceutical Industry outlines key elements necessary to help ensure the reliability and integrity of information and data throughout all aspects of a product’s lifecycle. It is intended to be used in whole or in part to guide a company’s internal practices, create or modify an existing data integrity code of conduct, or in developing agreements with outsourcing partners or other suppliers. The elements identified throughout this document are intended to reinforce a culture of quality and trust within the pharmaceutical industry. Access Data Integrity Code of Conduct

Guidance: Good Practices for Data Management and Integrity in Regulated GMP/GDP Environments

Pharmaceutical Inspection Convention/Pharmaceutical Inspection Co-Operation Scheme (PIC/S) – Good data management practices influence the integrity of all data generated and recorded by a manufacturer and these practices should ensure that data is accurate, complete and reliable. While the main focus of this document is in relation to data integrity expectations, the principles herein should also beconsidered in the wider context of good data management. Access PIC/S Guidance

Library of Data Quality, Records Management & FDA Recordkeeping Laws

Be compliant with US regulations and statutes on data integrity and recordkeeping in order to avoid stiff penalties and preserve corporate value. This robust collection of resources will prove to be quite helpful. Access Library

As defined in the ICH Q10 guideline, a control strategy is “a planned set of controls, derived from current product and process understanding, that assures process performance and product quality” (1). Every biopharmaceutical manufacturing process has an associated control strategy.

FDA’s 2011 guidance for process validation (2) describes process validation activities in three stages (Figure 1). A primary goal of stage 1 is to establish a strategy for process control that ensures a commercial process consistently produces acceptable quality products. Biopharmaceutical development culminates in the commercial control strategy, a comprehensive package including analytical and process controls and procedures. Stage 2 process performance qualification (PPQ) is needed to establish scientific evidence that a process is reproducible and will consistently deliver high-quality products. Stage 3 of validation and continued process verification (CPV) provides an opportunity to improve process control through the lifecycle of a product. Continue reading

SEE ALSO: 4 Steps for Managing Biopharmaceutical Manufacturing Projects

The BioPhorum Operation Group’s (BPOG’s) Container Closure Integrity Testing (CCIT) workstream would like to congratulate the United States Pharmacopeia’s committee for its latest revision to USP chapter <1207> Package Integrity Evaluation: Sterile Products. Generally, we believe it provides a comprehensive overview of the available methods for container–closure testing and outlines many important elements for consideration in establishing a successful CCIT strategy. We first responded to the USP <1207> draft when it was released for comment in 2014. And from our perspective, some of the changes that were proposed and concerns that were raised in 2014 clearly have been addressed. We are grateful to see that.

The purpose of this letter, however, is to highlight specific areas that cause us concern as a crossindustry group. We are aware that USP’s “informational” chapters are not compulsory. As end-users of such guidances, though — and as representatives of companies that receive the scrutiny of regulators — we recognize that informational chapters often evolve in practice to establish expectations. So any lack of clarity or any bias introduced toward specific methodologies is of concern. It is in this context that we would like USP to consider our further comments here.

These concerns center on the description, perception, and treatment of probabilistic and deterministic analytical methods — specifically dye and microbial ingress methods. We would like to see our concerns considered at the earliest possible opportunity, ideally precipitating an update to USP<1207>. Read article
SEE ALSO: 4 Steps for Managing Biopharmaceutical Projects

Interested in learning more about gene editing and its therapeutic applications? Download a new white paper from the American Society of Gene & Cell Therapy (ASGCT). The document, titled “Therapeutic Gene Editing: An ASGCT
White Paper” is intended as background information for policymakers, patients, and the general public to help them better understand gene therapy concepts and its related therapies.

The paper is designed to help people to better understand the ramifications of a soon to be released report on human gene editing by the National Academy of Sciences and National Academy of Medicine. This report, planned for release in early 2017, is expected to address the ethical, legal, and social implications of gene editing processes and suggestions for potentially needed policy.

Download White Paper

Biopharmaceutial project management is a complex process. This SlideShare walks you through the process:

• Challenges of biopharma project management
• Scope of biopharmaceutical project management
• Characteristics of great biopharmaceutical project managers – are we asking for unicorns?
• 4 Steps for managing biopharmaceutical projects
  • Step # 1 – Clearly Establish Project Definition and Impacting Constraints
  • Step # 2 – Project Execution Planning
  • Step #3 – Project Execution
  • Step # 4 – Project Completion

Despite active M&A activity by conventional standards, 2016 was a fairly quiet year compared to 2015. The U.S. government cracked down on inversion-based deal making that drove so much of the mega M&A activity in 2015. For instance, Pfizer expected to save about $123 million in taxes after merging with Ireland-based Allergan. It would have been the largest merger in pharmaceutical industry history.

When the law changed, the Pfizer and Allergan deal immediately fell apart as did other inversion-based deals. Instead, 2016 M&A activity focused on strategically managing product portfolios and leading companies buying the innovation needed for growth.

What will 2017 hold? With the incoming Trump administration it is hard to tell. Cash repatriation policies mentioned during the campaign might bring a great deal of off-shore money back to the U.S. and spark another round of M&A mania.

The following is some of the most notable activity from 2016. Happy 2017!

Sources:

• Mergr
• BioSpace
• Pharma Letter
• “Pharma Year in Review,” C&EN
• “Baxter Buys Claris’ Generic Injectables Subsidiary for $625M,” GEN, December 15, 2016
• “AbbVie to Expand Oncology Presence Through Acquisition of Stemcentrx and its Novel, Late-Stage Rova-T Compound for Small Cell Lung Cancer,” April 28, 2016, Abbvie press release
• “Pharma M&A Market: Latest Challenges and Opportunities,” Peter Young, Pharmaceutical Executive, December 14, 2016
• “Review of the year 2016: M&A,” George Underwood, Pharma Times, December 2016
• “Trump’s corporate tax holiday could spur pharma M&A,” Carl O’Donnell, Reuters, December 6, 2016

As 2016 draws to a close, C&EN takes a look back at 10 stories that shaped the pharmaceutical industry.
The year began with the expectation that Pfizer and Allergan would join to become the world’s largest drugmaker, but it was not to be. A proposed change in U.S. tax law prompted the firms to go their separate ways. Numerous smaller deals by both soon followed.

The human dimensions of the drug industry opened up in stories of the controversial approval of eteplirsen, a therapy for a rare disease that affects young boys, and of the fight against the Zika virus and its tragic impact on pregnant women.

Another story was of tech multimillionaires such as Sean Parker and Mark Zuckerberg getting into the drug discovery act with new institutes dedicated to curing cancer and other diseases.

Others were about drug pricing and FDA’s crackdown on shoddy practices at overseas companies.

The year ended with a story that could affect the drug industry for years to come: Donald Trump’s unexpected win in the U.S. presidential race. What that outcome means for pharmaceutical firms will start to unfold in the early months of 2017.

Read the top pharmaceutical stories of 2016. In addition, Top Research of 2016, the year in quotes, the year in tweets and more. Access C&EN’s 2016 Pharmaceutical Year in Review