Biosimilars, 21st Century Cures Act, Clinical Trial Regulations, PDUFA, Global Regulatory Strategy and a Preview of May

RAPSRAPS' Latest | 02 May 2017 |  By 

The following is a summary of feature articles posted over the past weeks with links and a preview of what's to come in May.

Feature articles over the past several weeks have highlighted biosimilar intellectual property issues and key strategies being adopted by the innovator companies. Other articles covered regulatory changes impacting how premarket applications for combination products will be reviewed by FDA, aspects of the revised European clinical trial regulation, key points and benefits of the Prescription Drug User Fee Act (PDUFA) and strategies for implementing IDMP standards. The establishment of a resource tool in Thailand is presented that was developed to assist health providers make decisions on product selection, improve drug procurement in a cost-effective manner and provide better healthcare service to patients.


Biological medicines are comprised of large, complex molecules produced by living organisms. Biological medicines are used for disease prevention or treatment and their entry into clinical practice has revolutionized healthcare in many challenging therapeutic areas. While it is relatively simple to reverse-engineer a generic version of a small-molecule drug using bench chemistry, the complexity of innovator biological products and the need for living cell cultures means it is impossible to reproduce them exactly. For this reason, generic versions of biologics have been termed 'biosimilars.' In their article, "Biosimilar Intellectual Property Issues in the US: An Update," Huml, Baum and Dawson provide an update on US biosimilar intellectual property issues including the complex and evolving areas of patent protection, market exclusivity, the 'patent dance' and lifecycle planning.

Over the past 25 years, biologic medicines have transformed how several chronic and life-threatening diseases are treated. Because of the great value of these innovative biologic medicines and the capital required to bring them to market, they are able to command high prices. For example, seven of the 10 top-selling pharmaceutical products in 2015 were biologics. However, the pending expiry of patents for many of these most successful biologic medicines presents an opportunity for developing biosimilars with potential to gain a share of the lucrative biologics market. "Strategies Adopted by Innovator Companies in Response to Biosimilars Competition" by Manaktala, Huml and Rulewski examine key strategies being adopted by the innovator companies and discusses the impact these strategies are likely to have on the future market for biosimilars.

Law and Legislation

Combination product premarket notifications (510(k)), Premarket Applications (PMA) and Requests For Designation (RFD) to the US FDA have increased substantially over the last decade with the number of regulatory submissions for this type of regulated product increasing annually. Cumming and Mathewson summarize regulatory changes impacting how premarket applications for combination products will be reviewed by FDA and procedures and reporting requirements for applicants and FDA as required by the 21st Century Cures Act in their article, "Amendments to the 21st Century Cures Act: Changing the Regulation of Combination Products."

Clinical Trial Regulation EU No 536/2014 will come into effect in 2018 and Schmitt, Bonacci, Lisinireport on aspects of the revised regulation and evaluate its potential impact on clinical trials conducted to assess medicinal products for human use. They discuss changes impacting the critical role of the Ethics Committees in the approval process for clinical trials as well as the planned, centralized data reporting channel, referred to as "the portal." While revisions to the regulation are still underway, readers will benefit from the assessment of its current status and anticipated developments.

When planning a New Drug Application (NDA) or other type of marketing application, regulatory professionals must consider many things, including when the data will come due, commitments made to the agency, working with agencies to plan the submission, internally planning timelines for the submission, coordinating all disciplines to contribute to the marketing application, working with authors to write sections in a specific way to stay on key messages, using pre-defined templates, keeping the whole process 'on target' in terms of publishing and submission timelines, and triaging any and all crises.

Fauvelle and Brown-Tuttle discuss key points and benefits of the Prescription Drug User Fee Act (PDUFA) for those filing a New Drug Application (NDA) and explain how PDUFA user fees have played an important role in expediting FDA's drug approval process in their article, "The Prescription Drug User Fee Act (PDUFA): A Primer."

To prepare a pharmaceutical company for the new IDMP standards, internal stakeholders and management need to be aware of the elements in the new standards, how they will impact the company and once the new standards become effective, how they will impact their products. After these considerations have been taken into account and after a project and a project team has been defined, the next step should be to examine the company's level of preparedness. Schlaps discusses the gap analysis and preliminary activities necessary to prepare a pharmaceutical company for the upcoming IDMP standards in his article, "Identification of Medicinal Products (IDMP) Standards, Part 3: Steps to Prepare for IDMP Implementation."

Global Issues

Thailand's health system is regulated by the Thailand's Ministry of Public Health (MOPH) and the Thai FDA. In "Thailand's Approach to Drug Quality Assurance: Creation of the Green Book," Patthamajinda discusses how MOPH and the Thai FDA in collaboration with a number of government and independent health agencies regulate drugs, both new and generic, as well as traditional, nonwestern medicines.

Feature Articles Coming Throughout May

Feature articles throughout the month of May will present upcoming changes to FDA's major User Fee Programs (PDUFA VI, MDUFA IV and GDUFA II) for fiscal years 2018-2022, various US and European harmonization initiatives and pediatric regulatory activities. Other articles will address conditional approvals, quality certificates and ICH Q12. Look for these feature articles throughout May.

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