June 2019

June 2019


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Executive Leadership | From the Editor-in-Chief

June 2019
The End of Chronic Therapies?
Alberto Grignolo

Alberto Grignolo, PhD
Global Forum
Fellow of DIA


ell, maybe not in the immediate future, but the end may indeed come.

The recent FDA approval of a gene therapy for Spinal Muscular Atrophy (SMA) raises fundamental questions about the future relevance of traditional medicines.

FDA’s press release announcing this approval states: “A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA.”

A single dose apparently delivers a cure. This is transformative.

Editorial Board

Content stream editors

Translational science
Gary Kelloff US National Institutes of Health
David Parkinson ESSA Pharma, Inc.

regulatory science
Yoshiaki Uyama Pharmaceuticals and Medical Devices Agency (PMDA)
Adora Ndu BioMarin Pharmaceutical, Inc.

Patient engagement
Deborah Collyar Patient Advocates In Research (PAIR)
Lode Dewulf Servier

Value & access
Edith Frénoy European Federation of Pharmaceutical Industries and Associations (EFPIA)

Editorial Staff

Alberto Grignolo, Editor-in-Chief Parexel International

Ranjini Prithviraj, Global Associate Director, Content Collaboration DIA Publications

Sandra Blumenrath, Science Writer DIA Publications

Chris M. Slawecki, Senior Digital Copyeditor DIA Publications

Regional Editors

David Mukanga Bill and Melinda Gates Foundation

Silke Vogel Duke-National University of Singapore Medical School

Richard Day University of New South Wales, Medicine, St. Vincent’s Hospital

Judith Glennie JL Glennie Consulting, Inc.
Megan Bettle Health Canada

Ling Su Shenyang Pharmaceutical University, Lilly Asia Ventures

Thomas Kühler Sanofi R&D

J. Vijay Venkatraman Oviya MedSafe

Kazuhiro Kanmuri Inter-Professional, Inc.

Inas Chehimi Novartis

Ebony Dashiell-Aje FDA

Young Professionals Editor

Kaley Weintraub Rutgers Post-Doctoral Fellow

DIA Membership

Bringing together stakeholders for the betterment of global health care.

Regulatory Perspective: Digital Health Technology Tools

Use in Clinical Investigations to Evaluate Clinical Benefit in Patients

Ebony Dashiell-Aje
Clinical Outcome Assessments Staff, Office of New Drugs
Sarrit Kovacs
Clinical Outcome Assessments Staff, Office of New Drugs
Leonard Sacks
Office of Medical Policy

t FDA, there is a focus on evaluating and considering valuable novel approaches and tools that can support our regulatory decision-making. The dramatic growth and incorporation of digital technologies into daily life has afforded new opportunities to use these tools to understand patients’ functioning and how it is affected by different diseases and treatments.

Former FDA Commissioner Scott Gottlieb has made statements regarding advances in the field of digital health innovation, including launching the Agency’s Digital Health Innovation Action Plan. Likewise, the US President’s budget includes a proposal to create a Center of Excellence for Digital Health. In the current landscape, the Agency is receptive to innovative, technology-derived, study endpoints in various therapeutic areas.


Califf: Digitization Will Return Humanity to Medicine
PREFER Focuses on Patient Preferences

Kristin Bullok
Benefit-Risk Management Scientist, Global Patient Safety
Eli Lilly and Company


atient engagement in medical product development has become a priority across patient groups, regulators, and industry. Presentations and communications proliferate about what patient preferences are and why it is important to understand them. But patient preference research needs practical steps to realize its goal; namely, the systematic inclusion of patient preference information and data into medical product decision-making. The Innovative Medicines Initiative (IMI) project Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) is helping in several major ways.

Engaging patients and their representatives in medical product research through patient preference studies is considered important. But at the same time, complex questions remain regarding methods used to elicit patient preferences and how to integrate these preferences into making research decisions. Previous projects and initiatives, including those from the FDA and EMA – plus earlier IMI projects such as Pharmacoepidemiological Research on Outcomes of Therapeutics (PROTECT), the European Patients Academy on Therapeutic Innovation (EUPATI), and the Medical Device Innovation Consortium (MDIC) – provide a solid foundation for developing such tangible recommendations.
From Market Authorization to Patient Access: How Long is Too Long?
EFPIA Patients W.A.I.T. Indicator

Edith Frenoy

Director, Market Access
European Federation of Pharmaceutical Industries Associations (EFPIA)

Background and Scope


egulatory approval (marketing authorization) is necessary for drug launch but is not sufficient to guarantee patient access to a licensed medicine. There is a time lag between a marketing authorization and patient access. The question of how long that lag is matters a great deal to patients. EFPIA sought to answer this question with empirical data.

The EFPIA “Patients W.A.I.T.” (Patients Waiting to Access Innovative Therapies) indicator provides a benchmark of the rate of availability and waiting times for new medicines in European countries. For new medicines (defined as medicines that include a substance which has not been previously available in Europe) up to a specific cut-off date, the report shows the following within a (rolling) three-year or four-year cohort:

  • The rate of availability, measured by the number of medicines available to patients in European countries.
    • For most countries, this is the point at which the product gains access to the reimbursement list (see exceptions/explanations in callout box below).
    • In some countries, products are available with special reimbursement conditions (see exceptions/explanations in callout box below).
    • The rate of availability in a country does not necessarily indicate medicine uptake, as some medicines may be available in a market with no uptake (no sales or no volume).
  • The average time between marketing authorization and patient access, measured by the number of days elapsed from the date of EU marketing authorization (or effective marketing authorization in non-EEA (European Economic Area countries) to the day of completion of post-marketing authorization administrative processes. Waiting times reflected in the Patients W.A.I.T. Indicator include any delay, whether attributable to companies or to competent authorities.

The analysis is based on information gathered from EFPIA member associations, who either refer to information available from official sources or gather this information directly from member companies.

Around the Globe

Pharmaceutical Policy Developments in Canada
Judith Glennie
JL Glennie Consulting, Inc.

s Canada heads into a federal election in October 2019, the first half of 2019 has been a busy time for pharmaceutical policy-related announcements. Issues that have been topics of discussion for decades have finally risen to the top of the political agenda, impacting areas such as rare diseases, national pharmacare, and drug pricing, to name a few. These developments are particularly relevant to reimbursement policies.


New Sources Changing the Meaning of Evidence?

Around the Globe

New Rules Reinforce Clinical Trial Oversight in India

Anirban Roy Chowdhury
ARC Life Science Consulting Group
Executive Committee Member
Indian Society for Clinical Research


n March 19, 2019, the Union Ministry for Health and Family Welfare published the new Drugs and Clinical Trials Rules, 2019 (New Rules) which is expected to build a robust regulatory and clinical research ecosystem in the country and promote the conduct of ethical and quality clinical trials which, in turn, will benefit patients in India by providing early access to medicines for unmet medical needs.

These New Rules are applicable to all stakeholders like pharmaceutical companies, study sponsors, investigators, academic research institutions, and ethics committees who are involved in conduct of clinical trials, bioequivalence and bioavailability studies, and academic health research.

It is interesting to note that the word “oversight” appears several times in the New Rules. Although the number of mentions may not be that significant, it is the spirit of the oversight of research activities by stakeholders that gets a lot of attention in the New Rules.

Young Professionals’ Corner

Specialty Pharmacy:

Customized Approach to Drug Dispensing, Continued Pressure to Innovate

Carolyn Riedl
Rutgers Post-Doctoral Fellow

What is a Specialty Drug?


pecialty drugs are used to treat complex and chronic conditions. Though there is no standard definition, there are two main considerations for determining whether a product is a specialty medication: complexity and cost.

  • Complexity may be specific to the handling or administration of the product itself, such as an injectable for the management of psoriasis; it may also be due to the complexities of managing the disease state, such as an oral oncolytic with specific monitoring parameters.
  • High cost of most specialty products is in part due to the complexities of manufacturing the product. For instance, some specialty products are biologics which are manufactured or synthesized from a biological source. These specialty products may require cold chain management and subcutaneous administration. As a result, patients who are utilizing specialty medications also require a higher level of clinical management and specific drug and disease support services.
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