Risk Management, Policy Framework, EU MDR and EU IVDR, and Global Orphan Drug Regulations

Feature ArticlesFeature Articles
| 06 September 2018 | By Gloria Hall  | ©

Feature articles over the past weeks have focused on risk management, the global regulatory framework, including next generation cancer vaccines, drug delivery combinations and pediatric plans, the latest on EU MDR and EU IVDR implementation and global orphan drug regulations.

Risk Management

Recognized drug safety and risk management expert Elaine Morrato discusses the intersection of emerging implementation science and regulatory science innovation and how together they may advance pharmaceutical risk management in the US. In Pharmaceutical Risk Management in the 21st Century Cures Era, the author touches on risk management as an adaptive, rapid-cycle process embedded within a learning health system; dissemination design for better adoption and sustainability of the intended risk mitigation behavior(s); and framing risk management as de-implementation of low-value or harmful health services and practices.

The medical device and pharmaceutical industries have embarked on an advantageous, evolutionary journey focused on digital healthcare technology. In Risk Management in the Digital Healthcare Sector, Merck experts Darin Oppenheimer, Suraj Ramachandran, Jeannette Boyle and George Cusatis discuss the convergence of the information technology and the healthcare industries and the increased use of digital products connecting patients and healthcare professionals. The authors review issues surrounding risk management, including the risk potential for “telemedicine” devices, regulatory expectations and cybersecurity concerns. Unique challenges are detailed covering the effectiveness of risk management planning, regulatory expectations and cybersecurity.

The opioid crisis is far greater in the US than in other countries in the US with two-thirds of drug overdoses caused by opioids. FDA has used Risk Evaluation and Mitigation Strategies (REMS) to try to control the misuse of opioids. For several opioids, FDA has urged manufacturers to work together to create shared REMS to expedite management and assessment of the REMS requirements. In How FDA is Using REMS to Help Manage the Opioid Crisis, regulatory authority Edward Tabor provides a candid discussion on how the agency is trying to limit opioid misuse and abuse by restricting the availability of these important drugs to patients who really need them to treat intractable pain.

Policy Framework

Recent technical advances in the development of therapeutic vaccines—integrating immuno-oncology with precision medicine—are revolutionizing cancer therapy by implementing a strategy of utilizing the patients’ immune system to treat cancer. Regulatory research specialists Vidya Narayanaswami and Stephen Amato discuss advances in the development of “personalized” cancer vaccines in Overcoming the Challenges in Getting Cancer Vaccines to Market. The authors review challenges that need to be overcome to get new cancer vaccines approved and highlight FDA’s Regenerative Medicine Advanced Therapy (RMAT), a comprehensive policy framework aimed at providing oversight for cancer vaccines and regenerative medicine products, including novel cellular therapies.

For all new medicines seeking Marketing Authorization (MA) in the EU and US, pediatric plans must be submitted to ensure medicines for children are included in the mainstream drug development process.

Navigating the Development of Pediatric Plans details a practical overview of the information that should be included within a pediatric plan, focusing on EU regulations with a comparison to the legislation issued by FDA. Scientific writing and regulatory affairs professional Rajendra Wable examines the common mistakes and misunderstandings that can occur when submitting such documents.

EU MDR and EU IVDR: Next Steps

In recent years, biopharmaceuticals have revolutionized the treatment of many chronic, serious and life-threatening diseases. With few exceptions, because of their molecular size and structure, they have to be administered to patients by injection. Drug Delivery Combinations and new EU Medical Device Regulations discuss regulatory issues related to Drug Device Combinations (DDCs). European regulatory experts Mats Ericson, Blake Green and Andrew Lennard speculate on how these products will continue to be regulated as medicines under the new EU Medical Device Regulations and offer suggestions regarding considerations and changes that may need implementation prior to the end of the transition phase in the year 2020.

With the full application of the new In Vitro Diagnostic Regulations (IVDRs) fast-approaching, IVD companies planning to CE-mark their first product or continue to market their existing CE-marked products, should not wait until 2022 to transition. Companies should immediately begin developing and implementing strategies to be fully compliant with the changes. In vitro diagnostics and quality authority Karen Richards discusses processes and plans to be developed by IVD companies to address the new EU IVDRs in Complying With the new EU IVDR. The author explains the differences between IVD and IVDR, new IVD classifications, notified bodies, new clinical evidence requirements and when to begin transitioning to the new requirements.

Global Orphan Drug Regulations

A landmark European law introduced a set of commercial incentives in the European Union (EU) to encourage the development of products for rare (orphan) diseases—which until then, had been largely neglected by the pharmaceutical industry. In Orphan Drug Regulations in Europe, regulatory professionals Sharry Arora and Bhaskar Saxena define orphan drugs and provide an overview of the evolution of European orphan drug regulations. The authors address incentives, including orphan drug legislation and policy, the criteria for orphan status designation and the authorization process for gaining an orphan drug designation in EU.

It is estimated between 5,000 and 8,000 distinct rare diseases exist today, affecting up to 10% of the population in total with up to 250 million people suffering from a rare disease globally. In Regulatory Strategist’s Toolbox: Global Orphan Drug Regulations, Requirements and Designation Requests,

Regulatory intelligence expert Meredith Brown-Tuttle defines orphan drugs, outlines the requirements for orphan drug designation, discusses global orphan designation requirements, databases, processes and what new countries are exploring an Orphan Drug Act or regulation. The author also provides global orphan drug resources to aid in preparation of an application.

Canada does not have a specific regulatory framework for orphan drugs; however, Health Canada announced two initiatives to create a new approach for the authorization of orphan drugs and to release the Canadian arm of the international website orpha.net, a comprehensive database of information and services for rare diseases. In An Update on Orphan Drugs in Canada, regulatory expert Brenda Gryfe discusses the agency’s current approach to orphan drugs for rare diseases and how two recent initiatives to improve regulatory review may impact orphan drug registration in Canada.


For years, medical experts have called for a randomized clinical trial to investigate dietary salt’s links not only to high blood pressure but to heart attack or stroke. In Salt: Needed, Widely Used, Often Misunderstood, regulatory professional Max Sherman discusses dietary salt needs, the history of salt use and why its use should be studied to further investigate links to cardiovascular disease. The author cites a potential study on salt intake aimed at determining the unhealthy consequences of high salt intake and closes by reviewing recent studies suggesting that too much salt in the diet can lead to a variety of cardiovascular problems, including high blood pressure and the potential for heart attack or stroke.

Feature Articles Coming Throughout September

September will include a variety of articles on advertising, promotion and labeling, including compliance strategies for updating prescription drug labels, drug advertising in Canada, advertising and promotion regulatory reviews, optimizing labeling and advertising outcomes through targeted subject-matter expertise and off-label promotion. Look for these and other regulatory topics throughout September. If you have a suggestion for a feature article, contact me at ghall@raps.org.


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