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Setting A Higher Bar

hose who came to DIA Europe 2018 in Basel last month expecting EMA Executive Director Guido Rasi to detail the logistics of the Agency’s move from London to Amsterdam in 2019 were in for a surprise.

Without dismissing the importance of the move and its impact on approximately 800 families of EMA employees, Director Rasi chose instead to emphasize what matters most: the uninterrupted productivity of the Agency (as both gatekeeper and enabler) with regard to the timely assessment of medicines for the benefit of patients. His views were echoed by others (national regulators, patient advocates, industry representatives) at the meeting, who ironically now regard Brexit as an opportunity for the 27 post-Brexit EU Member States to compensate for the anticipated “exit” of the influential British MHRA by hiring more local experts and assessors, thereby raising their own bar of regulatory expertise. The mood was cautiously optimistic as Brexit becomes a reality in March 2019.

oles and responsibilities in the Canadian healthcare system are shared between different levels of government.

The federal government’s role includes setting and administering national principles, while direct health and social service delivery for most citizens is primarily the role of the provinces and territories. In 2016, Canadian provincial, territorial, and federal health ministers committed to work together to improve the affordability, accessibility, and appropriate use of prescription drugs.

he Certificate of Pharmaceutical Product (CPP) is the primary document of the World Health Organization’s (WHO) Certification Scheme on the quality of pharmaceutical products moving in international commerce.

The Initial Stage: 1980s to 2010

In the 1980s, Professor Chen Jie of Fudan University was the first person to bring the idea of HTA to China. Since then, he has been pushing for the development of HTA in China and raising awareness about HTA with health policy makers and health-related workers. With the expanding influence of HTA, the research unit at Fudan University was nominated as the ‘Key Laboratory’ by the Ministry of Health (MoH) in 2000. The lab conducted a series of academic HTA training workshops that introduced the HTA concept to professionals in China for the first time.

his month’s Translational Research discussion features imaging biomarkers, their essential role in predicting clinical trial outcomes, and new approaches which may accelerate drug development and protect patient safety.

This article summarizes progress from the initial imaging biomarkers developed to evaluate tumor shrinkage in response to cancer therapeutics, to the challenges posed by contemporary treatment paradigms which may produce new patterns of response. Current avenues toward meeting this challenge, including volumetrics, radiomics, kinetic modeling, and artificial intelligence, are reviewed. It focuses on computerized tomography (CT) because it is the workhorse of contemporary oncology, generating datasets required to develop novel approaches and creating immediate translational potential. However, the techniques discussed may enhance analysis of any quantitative imaging modality, including FDG-PET and/or MRI. The review of historical progress suggests factors that drive improvement independent of technology: increasing reproducibility, refining conceptual models, quantifying tumor phenotype, and learning from experience.

hat exactly is a medical insight? How does one identify and collect meaningful medical insights, and apply them to the healthcare product business?

And once one has gathered these insights, how does one continue the engagement with the thought leader or healthcare professional beyond the initial interaction? These questions were addressed at the Fishing for Insights and Extending the Engagement Beyond the Congress Workshop at the 2018 DIA Medical Affairs and Scientific Communication (MASC) Forum. The purpose of this workshop was twofold: 1) to share perspectives on engagement from those in the pharmaceutical, medical device, and professional association fields; and 2) to explore strategies and tactics to extend the engagement beyond the conference venue.

Key Takeaways

• Traditional insight gathering and engagement approaches can be supplemented with other methods, such as simple targeted surveys
• Engagement tactics that are mutually beneficial to both parties lead to stronger relationships
• When discussing insight generation and engagement, it is important to consider collaboration with internal stakeholders.

ach year, DIA is assisted by hundreds of volunteers to plan and organize the DIA Global Annual Meeting, including members of the Annual Meeting Planning Committee (AMPC). The AMPC considers and helps develop topics for each track featured in the Meeting.

As some of the newly appointed Patient Engagement (PE) Track members, we are sharing our thoughts about what DIA and its members can learn about Patient Engagement at the 2018 DIA Annual Meeting.

urrent digitization of patient health information via electronic medical records, data from clinical genomics, biomedical imaging, lifestyle and activity monitoring using wearables, social media, patient registries, emerging technologies of data analytics, and blockchain opens new opportunities to engage patients as important stakeholders in the biopharmaceutical drug discovery and development process.

Traditionally, the drug discovery and development process has engaged patients as participants, but not as partners. With its mix of advocates and industry members DIA’s Patient Engagement Community (PEC) offers thought leadership and practical experience in integrating patients as full partners.

n recent years, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has taken several steps towards innovating the regulatory process to deliver safer and more effective medicines and devices to patients with life-threatening conditions. Restructuring efforts have targeted the very core of the organization, seeking to improve PMDA’s performance efficiency throughout the regulatory review process.

To that end, PMDA has implemented various innovative, fast-track regulatory initiatives as part of its current 5-year Mid-Term Plan, including the sakigake Designation System and the most recent Conditional Early Approval System.

The result: PMDA scored a major success with the speedy approval of a “titanium bridge” (NPC-17) for type II thyroplasty – a device whose approval would have remained in limbo considerably longer without the sakigake fast-track review system. With the recent regulatory initiatives, PMDA has met the major targets of its Mid-Term Plan, achieving shorter approval times, greater review service quality, and enhanced safety measures. By prioritizing innovative products and companies that have not filed for approval elsewhere, the ministry of health hopes to provide such products earlier to patients.

or the first time in history, Japan’s new Clinical Research Act, enforced in April 2018, legally defines the term “clinical research” as clinical trials designed to establish the efficacy and/or safety of drugs, medical devices, or cellular and tissue-based products.

However, the new Act may add another bureaucratic step for researchers seeking to advance clinical trials.

The definition excludes clinical trials related to the approval of new medical products that are defined and conducted in accordance with the provisions of the Pharmaceuticals and Medical Devices Act (“Chiken”). The new legal definition is not in line with the global consensus on clinical research, which covers a wider area, including observational studies. Therefore, it might become necessary to refer to clinical research defined by this act as “clinical research-in-law” to distinguish it from our conventional understanding of clinical research.

his year, a remarkable national organization, NPS MedicineWise (previously known as the National Prescribing Service) celebrates its first 20 years of service to the Australian people.

The NPS was founded in 1998 by the Australian Government to promote Quality Use of Medicines (QUM), the centerpiece of Australia’s National Medicines Policy. The underlying premise of the QUM policy is that better outcomes from medicines use can only be achieved by inducing a ‘behaviour change’ of, and ‘partnerships’ between, patients, families, and caregivers along with health care practitioners and their organisations, the pharmaceutical industry, and governments. Critical to the QUM strategy is that the patient should occupy the centre, and crucially, the Consumers Health Forum (the umbrella body for consumer health organisations in Australia) was instrumental in driving the government and stakeholders towards the National Medicines Policy, the QUM national strategy (1992), and ultimately NPS MedicineWise.