What life science companies need to know about pre-approval access

Pre-approval access is evolving at a rapid pace. Mark Corbett, Executive Vice President of Medicines Access at Inceptua, gives you an overview of the key benefits of utilizing pre-approval access programs in your market strategy while helping more patients get access to medicines.

Pre-approval access to medicines is a hot topic. The majority of major oncology and rare disease medicines which have come to the market over the last few years were provided to patients in need prior to licensing via some form of pre-approval access program.

With most drugs being launched first in the US, a US Expanded Access Program (EAP) is usually the first step in any pre-approval access to a new medicine. Typically, medicines are provided free of charge, and the duration of the EAP is usually a few months, from filing until commercial availability.

Support global commercialization

However, the US EAP component of pre-approval access is just the tip of the iceberg in terms of potential patient numbers. Most companies with an eye to global commercialization extend the pre-approval access scope to encompass most of the major markets in Europe. This has multiple benefits. Firstly, due to the way the filing and reimbursement process works in the EU (where despite centralized EMA approval, in-country pricing and reimbursement negotiations can take years), pre-approval access in the EU is a crucial route to allow patients in need to gain access to a promising medicine which has been approved and is available in the US. In addition to this direct benefit for patients who have explored all existing treatment options in their country, it allows companies to gain early experience in their key markets.

Beyond these key launch markets, pre-approval access is also utilized by many companies to allow patients access to their medicines in countries where they never intend to commercialize either due to patient numbers not warranting a specific country launch, or where the company has no commercial footprint to support commercial launch.

The regulatory environment for pre-approval access is evolving at a rapid pace, with many new developments in recent years designed to improve and simplify the process of access. The majority of countries are supportive of pre-approval access, seeing them as a way to allow patients in their country to gain access to promising medicines.

As part of this evolution, the collection of clinical outcomes data is becoming more commonplace in the pre-approval setting. Although such data does not have the robustness of clinical trials data, it is a good early benchmark of how a product is performing in a patient population more reflective of the commercial setting.

No need to be concerned

Many companies still have reservations about pre-approval access. The most commonly cited reasons for a reluctance to open a pre-approval access program are concerns about cannibalizing ongoing clinical trials, and concerns about revealing unexpected safety signals prior to licensing.

Both of these concerns, however, don’t come into being in reality. Firstly, physicians know they should enroll a patient in an ongoing clinical trial (if they are eligible) before exploring the pre-approval access route. In fact, pre-approval access programs often help recruitment into clinical trials by making physicians aware of suitable trials within their countries.

Safety/adverse event concerns are not based on any demonstrable data. In fact, a recent analysis has shown that clinical holds imposed based on AEs identified during EAPs in the US are far less frequent than those imposed during normal commercial development of a drug (0.2% vs 7.9%).

In order to reassure companies about this, the FDA issued clarification in October 2017 about how it handles adverse event data from EAPs. It stated that it was not aware of instances in which adverse event information from an EAP had prevented the FDA from approving a drug. It went on to say that FDA reviewers of such adverse event data understand the context in which the EAP use was permitted and will evaluate any adverse event data obtained from an expanded access submission within that context.

For all products meeting a high unmet need, a key part of your market access strategy should encompass pre-approval access. The benefits vastly outweigh the risks.

Learn more: For more information about and/or advice on pre-approval access to medicines, you are welcome to contact the author Mark Corbett: mark.corbett@inceptua.com.

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CEO and Board Member

Stefan Fraenkel

Stefan Fraenkel is the CEO of the Inceptua Group and is also on the Inceptua Board. He has extensive experience in the pharmaceutical industry. Before joining Inceptua, he held senior management positions at Sobi (Swedish Orphan Biovitrum), Sweden’s largest publicly listed specialty pharmaceutical company, leading Marketing & Sales and serving as Head of Corporate Development and Strategy.

Previously, Stefan worked at Pfizer and Wyeth in international commercial leadership roles across Europe and the USA, gaining deep insights into global pharmaceutical markets and strategies. Stefan has also spent part of his career in management consulting. Stefan holds a PhD in International Economics, an MBA, and a BSc in Engineering.

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Chris Hasslinger

Chris Hasslinger is a Partner of Vesey Street Capital Partners and serves as a member of the Firm’s Investment Committee. He joined VSCP in 2023 and is responsible for sourcing and evaluating new investment opportunities and general portfolio company management. Mr. Hasslinger has nearly three decades of experience in healthcare and technology strategy and deal-making within the industry as well as in investment banking and private equity. He has extensive M&A experience, having closed over $20 billion in transaction value, and has established and structured a number of large commercial partnerships.

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Alan Raffensperger

Alan Raffensperger has a robust leadership background in the pharmaceutical industry, having served as CEO of Inceptua and COO of Sobi (Swedish Orphan Biovitrum), along with significant international executive roles at Amgen, Roche, and Pharmacia.

He has also been CEO for venture capital-owned medical device companies, showcasing his versatility in healthcare leadership. Alan’s early career as an advanced life support paramedic provided a strong foundation in emergency healthcare.

He holds an MBA and a BA in Emergency Health Care Management.

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Blake Goodner

Blake Goodner is a founder member of the investment firm Bridger Capital. He previously worked as a healthcare analyst at Tiger Management and a healthcare investment banker at Morgan Stanley. He currently serves as an advisor and board member for a range of healthcare companies. Mr. Goodner has been a member of the Trinity College Board of Visitors and the Duke Annual Fund Executive Committee. He is a current advisory board member with The Duke Margolis-Center for Health Policy.

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Adam Feinstein

Adam Feinstein is the Managing Partner of Vesey Street Capital Partners. Mr. Feinstein has 30 years of investment experience exclusively in the healthcare services sector. Prior to founding Vesey Street Capital Partners in 2014, he was a Managing Director on Wall Street and a healthcare industry executive. He held the position of Senior Vice President of Corporate Development, Strategic Investments, and Office of the Chief Executive Officer at LabCorp. Before his tenure at LabCorp, he spent 14 years as the Managing Director in Equity Research at Lehman Brothers/Barclays Capital. Mr. Feinstein is the Chairman of VSCP’s investment committee. He is also actively involved in working with portfolio company executives and sourcing new investment opportunities. At the same time, he oversees all of the firm’s investment activities and employees.

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Larry Marsh

Larry is a General Partner of Vesey Street Capital Partners and serves as a member of the Firm’s Investment Committee. He joined VSCP in 2016, and currently sits on the Board of QualityMetric, Safecor Health, and HRGi. He is responsible for portfolio company management and for evaluating new investment opportunities. Prior to VSCP, Larry was EVP, New Market Development & Chief Strategy Officer at Fortune 10 AmerisourceBergen. Prior to that, Larry was the #1 ranked Healthcare Technology & Distribution analyst on Wall Street for over a decade, at Barclays, Lehman Brothers, Salomon Smith Barney, and Wheat First Butcher & Singer. Larry worked with Adam, Bryan, Dan, and Joe at Barclays and Lehman. Larry received a B.S. in Economics & Management as well as an M.B.A. from the University of Richmond, and an M.P.H. from Columbia University.

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Heyward Donigan

Heyward Donigan is a seasoned healthcare CEO, Board Member, and Private Equity Advisor with broad industry experience and a track record of profitable growth. From 2019 to 2023, Ms. Donigan served as Rite Aid’s president and chief executive officer, making her one of the few women CEOs of a Fortune 500 company. While at Rite Aid, Ms. Donigan led the company to through a major brand, merchandise and technology transformation, debt reduction/refinancing, while also leading the company through a pandemic. Ms.Donigan is currently a strategic advisor to Vesey Street Capital Partners, Arima Health, and a board member of OnMed.

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Doug Cook

Doug Cook is the Executive Chairman at Inceptua. Mr. Cook began his career driving the early success of Livingston Healthcare, leading to the purchase by UPS to become what is today UPS’s Global Healthcare business. After Livingston Healthcare, Mr. Cook moved to AmerisourceBergen (now called Cencora/COR) in 1998 and had an impressive career overseeing most of Cencora’s high growth, manufacturer-facing business. Mr. Cook ultimately served as the Executive Vice President, President Commercialization and Animal Health for COR.

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