Guest Column | October 4, 2019

Developing Novel Policy Proposals To Support Access To Gene And Cell Therapies

By Anthony Barron, principal, Life Sciences Practice, CRA


The decades-long focus on more “personalized” medicines has paved the way for the recent and anticipated advances in gene and cell therapies and progress in many other areas. The first generation of gene and cell therapies has the potential to transform the way we treat some diseases and can bring new hope to patients with many rare and serious conditions. Many gene therapies are designed to be effective after a single cycle of care (one-off transformational or potentially curative treatments), which brings many obvious administration and patient benefits. Given the way medicines are currently paid for, this often involves large one-time payments for these drugs, although the clinical benefits may not be observed for several months or even years. Given this, advanced therapies such as gene and cell therapies will present a number of challenges for healthcare systems. If left unaddressed, these challenges could result in the adoption of such therapies being restricted and ultimately limit patient access. Without new approaches in healthcare delivery, management, and funding models, treatment may be out of the reach of many patients and hinder the commercial success of these technologies.

On behalf of the American Chamber of Commerce to the European Union (AmCham EU), the Life Sciences Practice at CRA recently analyzed a range of emerging trends and challenges associated with access to the use of new integrated technologies, including gene and cell therapies (the full report is available online here). The goal of this analysis was to better understand the factors that could limit patient access to transformative technologies and promising new therapies and identify opportunities to improve healthcare policies to help expand access. This analysis, which included a review of current policies as well as interviews with policymakers, patient advocates, trade associations, and other industry experts, highlights a clear need for new policy solutions that are specifically engineered to overcome cost and budgeting issues, address limitations with reimbursement and funding models, and rethink value assessments and outcomes measures to help the maximum number of patients access new therapies while keeping health systems viable over the long term.

Overcoming Critical Challenges

As healthcare systems focus more on how to manage spending and implement new cost containment policies, one challenge will be the impact of growing pricing pressure and budgeting issues on the clinical development and commercial potential of gene and cell therapies. Although these therapies are likely to deliver significant direct cost savings in overall healthcare service delivery over their lifetime, there may be some additional costs associated with supporting care around the delivery of gene therapy in addition to product costs. Nevertheless, brought together, these will create per-patient up-front cost challenges that health systems may not be structured to support. In response, many countries are using a variety of different mechanisms to fund and support the introduction of new therapies to their markets, increasing the burden on pharmaceutical companies seeking to provide global access to these new therapies.

In parallel to this, we also observe trends toward the use of multi-indication medicines and combination therapies in oncology, as well as medicine-device combinations and digital medicines such as bioelectronics. While these technologies and unique treatment approaches have the potential to significantly improve disease management and patient care, they also place new pressure on health system funding.

There is now broad consensus among academics and policymakers alike that the pace of change has brought with it an urgent need to rethink strategies in healthcare funding and budgeting, including addressing issues with funding siloes or separate reimbursement systems and pricing models.1 Experts call for efforts to adapt and evolve existing pricing and reimbursement models and develop regulatory strategies to ensure that speed of access to new treatments is not compromised.2

Many experts also agree that the current frameworks that drive health technology assessment (HTA) decisions are too fragmented and not suited to the nature of emerging technologies including gene and cell therapies. Conventional HTA approaches also struggle with assessing cell and gene therapies, as the level of clinical trial evidence is often based on single-arm trials or relatively small patient populations — which HTA bodies often question. Data packages submitted to different HTA bodies often receive very different outcomes that are not systematically aligned when translated to patient access. As a consequence, the benefit of a personalized medicine may not be recognized with current HTA methods.

The introduction of gene and cell therapies can also radically change how healthcare providers and systems deliver patient care. In many cases, these therapies must be supported by a specialized healthcare infrastructure and use of advanced diagnostic testing. Real-world evidence (RWE) collection tools will also be required, such as patient registries for follow-up studies to capture the combined value of a companion diagnostic and medicine in relation to outcome measures. This can in turn lead to an increase in the amount of data generated and collected through the treatment cycle. Data systems will need to be able to speak to each other (i.e., horizontal interoperability) and healthcare systems will need to take measures (robust data protection and security) to establish trust in these rapidly expanding data systems and protocols.

The Path To Novel Policy Solutions

Given these challenges, new policy frameworks will be needed. The introduction of new financing arrangements to address challenges with “silo” budgeting and traditional pay-per-service models can help healthcare systems develop appropriate cost projections and better plan to accommodate many gene and cell therapies as they reach commercial launch. One approach could consist of combining funds from different organizations through “pooled” budgets (e.g., primary care vs. secondary care providers, national vs. regional healthcare budget holders, etc.). In the more specific case of gene therapies, this could focus on ensuring more fluid funding between healthcare services. For example, resources saved by the healthcare system from greater efficiencies as a result of hemophilia patients no longer requiring weekly or monthly hospital visits for transfusions could be transferred to fund advanced therapies.    

Beyond new approaches to funding, novel pricing models also will need to be considered, and they may need to be based in part on the number of patients eligible for treatment. Despite the fact that costs for individual patients are often high, the total budget impact of these therapies on a health system may nonetheless be limited given the relatively small number of patients that these therapies apply to, especially for rare diseases. Both pricing by indication and outcomes-based payment models may need to be developed and involve collaboration among payers, industry, and health systems. 

To ensure equitable access to many gene and cell therapies, the structure and implementation of HTAs may require the introduction of new procedures and benchmarks to provide the most comprehensive assessment of value possible. In order to develop a new HTA framework, stakeholders will need to be flexible, trying novel approaches to assess a product’s clinical and economic value based on available clinical data. A more holistic approach is needed when conducting HTAs, including efforts to include a wider range of social and economic benefits. Many systems still focus primarily or exclusively on direct healthcare costs, often limited to short-term costs and without consideration of societal costs and longer-term benefits. Assessments of longer-term impact will be both essential and possible as the collection of real-world efficacy and safety data and long-term patient monitoring are established for many gene and cell therapies, especially for those that are administered to patients in a single dose and that offer transformational or potentially curative benefit. It may also be advantageous to have HTA discussions during the early clinical development stages for new drugs so that agreement can be reached on evidence requirements needed to demonstrate that the therapies would be expected to provide added value to healthcare systems, patients, and their families/caregivers.

Ensuring widespread access to cell and gene therapies will require reform of the healthcare system. Management of patients being treated with these advanced therapies can involve a range of new and often complex technologies at every stage, from administration and dosing to monitoring side effects and data capture. Healthcare providers must have broader awareness of these technologies, which may require customized training programs for care teams (sometimes including family members). There also must be clarity around legal issues associated with data privacy. While payment models for many new therapies may require collection of data related to patient outcomes, providers must be able to establish and maintain clear procedures to address data sharing restrictions and understand the appropriate balance between data security and the level of information that can be shared with other healthcare professionals or with pharmaceutical companies.

Effective plans to address these issues will be essential to support faster adoption of cell and gene therapies in order to meet target standards in patient safety and quality of care.

The Path Forward

Moving forward, the pace of innovation will demand that health systems dedicate significant time and resources to upgrade business models in terms of delivery of patient care and strategies for funding and pricing novel health technologies. From there, they will need flexibility to ensure new approaches to health policy are adopted that can optimize patient access within a viable long-term model of sustainability. Many health systems will need to work closely with multiple stakeholders to address the challenges new therapies create for data security, reimbursement, and other issues. Success will be driven by how quickly and successfully they can implement these plans.


  1. OECD (2015) “Fiscal Sustainability of Health Systems: Bridging Health and Finance Perspectives.”
  2. Corbett, M. S., Webster, A., Hawkins, R., and Woolacott, N. (2017) “Innovative regenerative medicines in the EU: a better future in evidence?” BMC medicine, 15(1): 49.

About The Author:

Anthony Barron is a principal in the Life Sciences Practice of Charles River Associates. He has a master’s degree in health economics from The London School of Economics and has been working on European and international pharmaceuticals policy issues for more than 10 years. Barron regularly advises clients on health financing and pharmaceutical market access policy issues and has conducted a number of research projects on access to medicines and industrial policy in the pharmaceutical sector in Europe for EFPIA, PhRMA, IFPMA, EuropaBio, and the European Commission.  This includes research regarding barriers to access to medicines, pharmaceutical pricing policies, health technology assessments, the value of healthcare biotech, and recent market access projects.

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