ORPHA Executive Briefings

ORPHA Strategy Consulting Executive Briefings focus on news and current trends in early access, adaptive development pathways, transformative medicines, orphan drugs and designations, rare diseases, and real-world patient-centered evidence. They are published on LinkedIn and available here for download. Your comments, feedback and suggestions are welcome per email:  info@orphastrategy.com

Executive Briefing #8 - Adaptive Pathways and the Application of Real World Evidence
David Schwicker, March 2018

Timely patient access is at a crossroads: the speed of biopharmaceutical development is accelerating, yet the faster drug development and approvals move ahead, the more challenging the sustainability debate and pricing & reimbursement become. Three experts: Nicola Bedlington, Ad Schuurman, and Rob Thwaites, representing three key stakeholders - patients, payers and industry - debate collaborative ways forward.
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Executive Briefing #7 - Early Market Access – Value Demonstration Based on Less Complete Data
David Schwicker, February 2017

Next to effectively “moving the needle” in addressing unmet patient needs, payers are finding that specialty, advanced and orphan therapeutics also can substantially move the needle in terms of cost. While the outlay for an individual orphan drug may be relatively modest, the cumulative impact of the rapidly increasing number of novel rare disease treatments enhances budgetary constraints. Consequently, there is an intensifying sustainability debate surrounding policies for rare diseases and orphan drugs.

This ORPHA briefing details the current opportunities and challenges for early value demonstration and market access in Europe, specifically for transformative orphan and specialty medicines.
ORPHA Strategy Executive Briefing #7.pdf
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Executive Briefing #6 - Early Access Programs – an Overview for the United States (FDA) and Europe (EMA)
David Schwicker, January 2017

The EMA’s Support for Early Access and the FDA’s Expedited Programs facilitate and accelerate development and marketing authorisation with the aim to foster patients’ timely access to new medicines that address unmet medical needs. The focus of early access, both in the United States and Europe, is on life-threatening and debilitating diseases with a major impact on quality of life, and on medicines with a credible promise of significant improvements in clinical benefit and patient-relevant outcome(s).

While the FDA’s programs are well established, certain of the EMA’s early access tools have been launched more recently.
This executive briefing provides an overview of the early access programs, which have notably similar features in both jurisdictions.
ORPHA Strategy Executive Briefing #6.pdf
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Executive Briefing #5 - Expedited Programs for Serious Conditions in the United States (FDA Guidance)
David Schwicker, January 2017

Expedited programs represent the FDA’s aim to facilitate and expedite the development of new medicines that address unmet medical need in the treatment of serious and life threatening conditions.

This ORPHA Strategy briefing summarizes the FDA guidance concerning the four expedited programs: fast track designation, breakthrough therapy designation, accelerated approval, and priority review designation.
ORPHA Strategy Executive Briefing #5.pdf
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Executive Briefing #4 - EMA Adaptive Pathways Workshop Summary from an Industry Perspective
David Schwicker, January 2017

Adaptive Pathways (AP) is an initiative developed by the European Medicines Agency (EMA) focussed on medicines expected to have a significant clinical impact in patient populations with high unmet needs.

The EMA has hosted an Adaptive Pathways workshop in December 2016 to discuss stakeholder input and questions. This ORPHA Strategy briefing summarizes the workshop from the perspective of the pharmaceutical and biotechnology industry.
ORPHA Strategy Executive Briefing #4.pdf
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Executive Briefing #3 - Early Access – The State of Play in Europe 2017
David Schwicker, January 2017

This executive briefing summarizes the current “state of play” of innovative early marketing authorization and market access in Europe for transformative medicines from the viewpoints of the major stakeholders: Regulatory/EMA, HTA bodies, patients, healthcare providers, as well as industry.
ORPHA Strategy Executive Briefing #3.pdf
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Executive Briefing #2 - EMA Support for Early Access
David Schwicker, January 2017

There is a growing realization with regulators and health technology assessment (HTA) bodies that transformative specialty, orphan and advanced medicines often defy traditional routes of market authorization and access, particularly in rare diseases. This provides new opportunities for adaptive and innovative development pathways and early marketing authorisation and market access, employing a broader set of data and an iterative, life cycle approach to evidence generation, including real-world and patient-centred evidence.

This briefing provides a summary of the current European Medicines Agency (EMA) Support for Early Access, focussed on medicines that address major public health issues and are eligible to the centralized EMA procedure. The tools are not mutually exclusive and can be used in combination.
ORPHA Strategy Executive Briefing #2.pdf
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Executive Briefing #1 - Strategy Development for Early Marketing Authorisation and Rapid Market Access
David Schwicker, January 2017

Developers and manufacturers with potentially transformative medicines in early development should consider developing early access strategies for these products – or an entire early portfolio – to inform, complement and enhance their strategic thinking on benefit/risk and value demonstration, and the planning of evidence generation.
An early access strategy is developed through innovative thinking and discussion in an iterative and interactive strategy development process. This executive briefing outlines the ORPHA Strategy development approach.
ORPHA Strategy Executive Briefing #1.pdf
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