Guest Column | May 15, 2018

What's The Right Pricing Strategy For Cell And Gene Therapies?

By Walter Colasante, VP, Life Sciences Practice, CRA


At the BIO-Europe Spring Conference in Amsterdam in March 2018, CRA hosted a special program where attendees from biotech, Big Pharma, healthcare provider/payer groups, and patient associations were asked to share their views on pricing strategies for innovative medicine, including cell and gene therapies. More than 60 attendees responded to a series of questions about drug pricing, with a focus on cell and gene therapies, and their responses were captured live through an instantaneous voting system during a panel session. Their responses highlight the diverse interests and range of opinions about pricing innovative and often high-cost therapies, but also demonstrate that most players in the sector see an urgent need for new pricing models to accommodate these products. Following is an outline of the survey questions and responses from participants in the meeting.

Will selling at a low price, maximizing access to patients, put the long-term viability of gene therapy at risk?

Targeting a lower price point than what an economic assessment could justify, with a view to maximizing access of innovative new therapies, is a simple and obvious solution recently used for advanced medicines reaching the market. The goal is to circumvent early challenges, including severe payer restrictions, access delays, slow uptake, and failure to meet commercial goals, associated with high prices for new therapies. Opinions are decidedly mixed, with only 43 percent of respondents endorsing the simple low-price option. But the majority of participants indicated that they see potential long-term risk for gene and cell therapies if originators prioritize access without considering other factors in pricing models. They call for the full range of stakeholders to be involved or considered in pricing and access decisions, with a focus on long-term, more complex solutions in cases where new products can present significant short-term challenges in costs to payers and healthcare systems.

Are EU health systems positioned to accommodate gene and cell therapy pricing, and can solutions be based on reassigning funds and negotiating contracts?

There is a strong feeling that EU health systems are not positioned to manage the costs of innovative high-cost therapies, with 70 percent of participants voting “No.” Among the key considerations, participants believe there are currently insufficient funds or flexibility in health systems throughout Europe to support patient access to many advanced medicines. Many participants see the solution as much more complex than reassigning funds or negotiating contracts. From an industry perspective, one could urge multifunctional internal stakeholders to consider product launch strategies and policy initiatives that can better support long-term sustainability of gene and cell therapy development and provide new sources of funding for access.

Most participants believe the solution must be a comprehensive approach that combines pricing strategy with policy activities up front to ensure that pricing decisions occur in an environment with sufficient funds available. Facilitating access to funding from third parties such as insurers, charities, and financial institutions for securitized loans could be one way to achieve target pricing and access levels.

Can engaging third parties (insurers, charities, and financial institutions) help to mitigate the funding and affordability issues hampering access to innovative therapies today?

The response to this question — with 83 percent saying “Yes” — highlights the strong support for development of new models in pricing and access that engage more stakeholders. To improve negotiation outcomes for high-cost therapies, industry insiders call for all stakeholders to acknowledge that access to many medicines, and especially many advanced medicines, is a goal where multiple stakeholders can bring innovative and mutually beneficial approaches that otherwise might not be possible with fewer parties at the negotiating table.

Can the biotechnology sector successfully develop, launch, and commercialize gene and cell therapies without the need to involve or partner with Big Pharma?

A majority of participants (64 percent) believe an innovative biotechnology company can often be positioned to launch a product independently rather than partnering with a larger biotech or Big Pharma. Financing and experience will continue to be the two biggest hurdles to overcome, but there are examples of nimble companies that have succeeded by developing or acquiring the range of resources and expertise necessary for success.

In all cases, the decision to commercialize a gene or cell therapy should be carefully considered to account for targets in patient access, product life cycle management, and consistency and synergy with other products in the company pipeline. In cases where these factors are aligned, the developers must then consider whether it has access to the short-term financing and internal capabilities that are essential to successfully launch a product. From there, with the right expertise in-house or in advisory roles, out-licensing to Big Pharma may not be necessary.

Is the commercial model for a gene or cell therapy the same as any innovative medicine?

One striking finding from the poll is that a strong majority of participants — fully 93 percent — agree that advanced gene and cell therapies are in a class by themselves when it comes to access and commercial strategies. Respondents to this survey overwhelmingly believe that the necessary commercial models for gene and cell therapies are not similar to other, often high-volume, innovative medicines. They reference multiple factors, including one-off dosing or curative efficacy, significant up-front financial costs, and limited sites of care that could restrict access for many patients, as considerations that will drive the need for radically different approaches to bring these therapies to market.

The specific commercial model will have to begin with examination of the similarities between cell and gene therapies and other innovative therapies to treat many rare diseases. Of particular importance are the communication of safety and clinical value, appropriate training around novel delivery models, and careful identification of patients, treating physicians, and treatment centers. From there, the scientific complexities and financial implications of gene and cell therapies can require detailed clinical conversations with clinicians and patients, often beyond what a traditional sales force can offer. To support adoption, these therapies will also require engagement by multiple stakeholders with regulators, payers, manufacturers, patient advocates, and the right healthcare providers. When it comes to innovative new therapies that represent important advances in medicine, industry insiders across the board caution that a one-size-fits-all model in development and commercialization will be insufficient.

About The Author:

Walter Colasante is a VP in CRA’s Life Sciences Practice. He has experience in both the pharmaceutical and consulting industries and across a range of different therapeutic areas including oncology, the central nervous system, and rare diseases. You can reach him at and connect with him on LinkedIn.

The views expressed herein are the authors and not those of Charles River Associates (CRA) or any of the organizations with which the author is affiliated.