Reg Vision

A painless look inside the most impactful regulatory trends and actions from 2017

By Karen Langhauser, Chief Content Director

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Keeping up to speed with the ever-changing global regulatory environment is enough to make anyone’s head spin — yet it’s vital when it comes to ensuring ongoing compliance, as well as making the right decisions for pharmaceutical organizations.

The benefits of proper regulatory intelligence are vast, especially at a time where speed-to-market is increasingly important. Successfully implemented regulatory intelligence can shorten time from filing to approval, increase the likelihood of marketing approval and help identify new opportunities in drug development. It can also help pharmaceutical organizations plan ahead, aiding in better prediction of regulatory review times and helping to proactively avoid potential compliance pitfalls.

What follows is what we hope will be a helpful contribution to your regulatory intelligence efforts: a brief discussion of some of the most impactful regulatory initiatives from the past year and how they play into current trends in pharmaceutical manufacturing.

The personalized medicines market — treatments tailored to the individual patient — is growing rapidly, with revenue predictions as high as $5,208.68 billion by 2022.1 Regulatory agencies play a large role in shaping the infrastructure that enables developments in personalized medicine.

On Aug. 18, 2017, the President signed into law the Food and Drug Administration Reauthorization Act (FDARA). This new law includes the reauthorization of the Prescription Drug User Fee Act (PDUFA), intended to provide the FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products.

Aside from the more visible changes to the fee structure and fees, PDUFA VI also aims to do more to integrate patient perspectives into the development and regulatory review of new medicines. Launched as a new initiative in 2012, as part of PDUFA V, patient-focused drug development (PFDD) incorporates the patient’s voice into the development and review process. The FDA has committed to hold 24 disease area-specific PFDD meetings with individual patients and patient groups over the course of PDUFA VI.

PDUFA VI has the potential to strengthen the FDA’s ability to advance the science of patient input with actions, such as placing dedicated experts into review divisions to engage with patients, patient advocates and sponsors during drug development.

21st Century Cures Act
The 21st Century Cures Act (though technically signed into law in December 2016 - a little early for our 2017 recap) is intended to provide the FDA with tools aimed at modernizing regulatory programs. In July 2017, the FDA announced a detailed work plan for the steps the agency is taking to implement different aspects of Cures, which included elements that further the goals of the personalized medicines initiative, including:

• The Center for Biologics Evaluation and Research (CBER) implementing the Regenerative Medicine Advanced Therapy (RMAT) designation, enabling the FDA to facilitate an efficient development program for, and expedite review of, new regenerative advanced therapies.

• CDER, working with CBER, outlining a plan for the development of patient-focused drug development guidances.

“PDUFA VI and the Cures Act work hand in hand to bring the patient into the drug development process and ensure that drug development is actually working to the benefit of patient outcomes,” notes Lawrence Liberti, VP executive director for the Center for Innovation in Regulatory Science (CIRS). CIRS, a non-profit subsidiary of Clarivate Analytics, brings together regulators, pharma manufacturers and health technologies assessment (HTA) agencies for the purpose of advancing regulatory and HTA policies and processes used to facilitate access to medicines.

The past year saw significant progress in the ongoing quest for global alignment of regulatory expectations. Harmonizing regulations across the world would greatly reduce the complexity of the drug development process, ultimately bringing new drugs to market faster.

ICH Guidances
A key organization when it comes to global alignment is the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH’s mission is to achieve greater worldwide harmonization in the production of medicines by developing guidelines via a process of scientific consensus between global regulatory agencies and industry experts.

“ICH’s aligned guidances allow pharma companies and agencies to have greater clarity surrounding regulatory expectations,” says Liberti. “In the last year, we’ve seen an increase in the number of participants that are formally recognizing ICH as an important way forward. Having alignment across growing markets will bring further clarity and predictability to the regulatory and development processes.”

Notable new members approved over the past year include regulatory agencies from Brazil (ANVISA), Korea (MFDS) and China (CFDA), while regulatory agencies from Cuba (CECMED) and South Africa (MCC) were added as observers.

As part of the ICH process, draft guidelines are transmitted to the regulatory authorities of the ICH regions for internal and external consultation. In 2017, the U.S. FDA released several draft guidances of ICH harmonized guidelines in various stages of the ICH process, including revised ICH S5 Guidelines, an addendum to E9(R1) “Statistical Principles for Clinical Trials” and a Q&A on Q11 “Development and Manufacture of Drug Substances.”

“ICH really sets a good level playing field, and I think adherence to ICH will be a key factor in promoting global alignment,” says Liberti.


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