In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
Introduction
Significant scientific advances have created a rich clinical research pipeline, with innovative and potentially curative cell and gene therapies already available (eg, Kymriah® for acute lymphoblastic leukaemia, Luxturna® for inherited retinal disease and Zolgensma® for spinal muscular atrophy). Over the next 10 years, more of these therapies are expected to enter the market for other disease areas and larger patient populations. Potentially curative cell and gene therapies represent a key shift in patient care and will have considerable impact across the patient journey, affecting the healthcare system as we know it and the key stakeholders involved. Their launch is also met with significant debate over the uncertainty of outcomes’ durability and safety over the longer term; and the impact of treatment prices on affordability as more treatments enter the market. We draw from the characteristics of treatments in the pipeline and discuss the implications these will have on elements of the healthcare system and stakeholders that are most likely to be disrupted. Specifically, we discuss the impact on provision of care, delivery and supply chain, pricing and market access and value demonstration — given the difficulty of proving a durable, curative effect.
Are you looking to explore how lipid formulations in softgels can enhance drug absorption and bioavailability. Register for our upcoming webinar to find out!
3 September 2025 | 3:00 PM BST | FREE Webinar
This webinar will delve into the different types of lipid formulations, such as solutions, suspensions, emulsions, and self-(micro)emulsifying systems. Applications span diverse therapeutic areas including HIV therapy, oncology, immunosuppressants, and emerging treatments like medicinal cannabis (eg, CBD).
What You’ll Learn:
Lipid formulation development and screening tools for optimisation
Key steps in scale-up and industrialisation to ensure consistency and efficiency
Impact of lipid-based softgels on drug delivery and patient outcomes.
Can’t attend live? No worries – register to receive the recording post-event.
To understand the extent of the disruption posed by anticipated curative cell and gene therapies, we assessed the current pipeline for curative treatments. Approximately 55 percent of trials for curative cell and gene therapies are in Phase II, hence, we would expect an increasing number of launches in the next approximately five to 10 years.1 Some of the disease areas with a significant number of assets in development include oncology, rare diseases, blood disorders, infectious diseases and cardiovascular diseases (CVDs); and launches into the more prevalent conditions, such as CVDs, would lead to a larger systemic impact on healthcare structures.
Implications of potentially curative cell and gene therapies for healthcare systems
The positive health and other indirect benefits from potentially curative cell and gene therapies will require significant changes to healthcare systems at a global level. Drawing from the implications in three distinct disease areas with significant expected launches — rare diseases, cardiovascular diseases and haemophilia—potentially curative cell and gene therapies affect the patient journey throughout the healthcare system.
Diagnosis: the need for earlier and targeted diagnosis
To realise the full benefits of curative treatments, patients need to be diagnosed early. This will require continuous support and broader access to newborn screening in disease areas with strong genetic links, such as beta thalassemia, and a more significant shift towards targeted genetic testing in diseases with later symptomatic onset, such as CVDs.
More than 80 percent of rare diseases have a monogenic cause and are biologically attractive targets for cell and gene therapies, as by re-coding the defective gene or altering gene expression they can be cured.2 With a potential beta thalassemia cure on the market, diagnosis through screening could offer the opportunity to identify and treat patients early so they could enjoy better health through their adulthood. This also may lead to significant cost offsets for care and increased productivity gains from patients’ economic and societal participation.
Alternatively, CVDs are heterogeneous and multifactorial, from demographic to lifestyle-based, with only a portion of CVDs having clear genetic factors.3 Diagnosis is often confirmed later in life and diagnostic medical testing is driven by lifestyle risk factors and personal or family medical history. Cell and gene therapies in development for CVDs rely on current diagnosis methods. Biomarkers are becoming increasingly important and there is potential for curative therapies paired with targeted testing to motivate earlier diagnoses and avoid later-stage healthcare costs.4,5
Contact with HCPs: need for expanded healthcare expertise
In the presence of curative therapies, the required level of healthcare expertise will change. Healthcare professionals (HCPs) involved in the current care process will either need retraining or moving to serving patient populations not targeted through cell and gene therapies. In addition, curative treatments often depend on complex processes of preparation and administration, requiring a new set of specialists to be trained.
there will be significant adjustments to current healthcare systems affecting diagnosis, treatment expertise and infrastructure…”
Looking at the current treatments for CVDs, these range from initial preventive treatment for those at high-risk of developing CVDs, to oral therapies prescribed in the General Practice (GP) setting (also referred to as ‘Family Practice’), to in-hospital procedures. As CVDs often eventually require long-term treatment through a mix of primary and secondary settings, the introduction of potentially curative cell and gene therapies could disrupt disease management and have implications for practitioners. In a world with CVD curative therapies available, GPs are less likely to treat/care for CVD patients who have early targeted genetic testing/diagnosis and specialists are likely to play a larger role in patient care. For example, standard treatment for coronary artery disease currently consists of generic oral products such as statins, which can be prescribed by the GP. However, a gene therapy in development for this disease, Generx® (currently in Phase III trials), requires administration through a balloon catheter by a specialist cardiologist.6 In addition, curative therapies could reduce the requirement for cardiologists to perform as many traditional CVD surgeries, if they are effective in stopping or reversing disease progression.
Building an appropriate hospital infrastructure and the role of cross-border care
The infrastructure required for the preparation, delivery and administration of curative treatments is highly specialised. This will likely lead to a clustering of care expertise and infrastructure needed for certified Centres of Excellence (CoEs) where care is delivered, with multidisciplinary teams providing integrated diagnosis, initial care and treatment maintenance services. It is also likely that these select centres may not be available in every country or region.
For rare diseases, treatment is largely provided at targeted CoEs that pool the necessary infrastructure and expertise. Although most rare diseases require life-long care, current standard of care treatments range from oral therapies to procedures and surgery. Potentially curative cell and gene therapies represent a complete shift in treatment, from chronic maintenance to a one-off or discrete course of treatment. In the example of beta thalassemia, emerging treatments would require extended stays in highly specialised facilities, which are scarce (eg, there is only one in Germany). Following the procedure, while patients will continue to be monitored by haematologists, cured patients would no longer need to visit clinics every two to four weeks for blood transfusions. This would represent a significant relief in burden and increase in quality of life for patients, plus reduce the specialist resources required for chronic treatment. Moreover, patient reliance on specialised treatment centres for chronic care would also decrease over time. Severe patients or those for whom the efficacy of curative treatments has waned likely would constitute most patients for these centres via on-demand factor replacement. This change in patients served would affect the market models, reimbursement opportunities and the portfolio of services that these chronic care specialist centres offer to patients.
Appropriate assessment of value and pricing and reimbursement
Given the long-term health and indirect benefits provided by curative treatments, health technology assessment (HTA) bodies and payers have already started to include the longer-term impact and broader benefits in their cost-effectiveness assessment modelling. They have started to explore more flexible approaches and thresholds to allow the value of curative therapies to be understood and tested over time. The degree of shift in methodology will vary by disease area and available treatment options.
For rare disease treatments, a flexible approach encompassing special provisions or pathways is implemented in many countries to allow for a higher price and address limitations in evidence given the small patient populations. In more prevalent disease areas, with multiple treatment options and, in some cases, generic alternatives, HTA or other evaluating bodies will require a more significant shift in approach to allow for curative treatments’ benefits and costs to avoid suboptimal comparisons to existing therapies. For example, flexibility in comparator analysis will be required for CVDs, where standard-of-care is consistently oral treatments (eg, beta blockers, acetylcholinesterase inhibitors). Considering long-term cost offsets is also key. The need for ongoing surgeries and/or heart transplants, which particularly affects progressive diseases such as heart failure, could be replaced with curative medicines that could potentially reverse the progression, such as Revascor®, a cell therapy in Phase III for chronic heart failure.7,8 This may lead to indirect health benefits by reducing co-morbidities and increasing gains from productivity later in life.
Dealing with uncertainty and importance of data and registries
A key component that requires close consideration is how HTA bodies and payers deal with uncertain efficacy and safety data. Curative treatments have only recently launched and will require long-term studies, as well as monitoring and collection of real-world data (RWD) to demonstrate durability of effect and safety.
There is already a trend towards the development of RWD registries for collecting ongoing data on specific products, where registries are managed by pharmaceutical companies or national medical agencies, such as the Zolgensma® registry required by the Federal Joint Committee (G-BA) in Germany.9
Alternatively, novel pricing and payment models can be used to facilitate access while managing payer uncertainty. Such is the case with bluebird bio’s Zynteglo® instalment payment plan over five years, where payers only continue to pay the instalments if the beta thalassemia patient continues to respond successfully to treatment.10
Addressing budget impact through novel access and payment models
One of the key challenges for payers and budget holders is assessing not only the extent of benefit but also affordability. This is particularly an issue as curative cell and gene treatments would lead to a direct budget impact at the time of provision, while patients, payers and society at large would reap the benefits over time. This has triggered a debate on the need to develop novel access and payment models that distribute the impact on the budget holder across several years. Payments can also be linked to outcomes to address clinical uncertainties, for instance in outcomes-based contracts in the US and payment-by-results agreements in Europe.
While individual rare diseases are uncommon and often not considered by payers to have large budget impact, the combined impact of all 5,000 to 8,000 rare diseases is significant. As more potentially curative cell and gene therapies reach the market for a greater number of rare diseases, and potentially more prevalent ones, the budget impact will be considerable. A pushback on prices is already evident with current gene therapies for rare diseases, including Luxturna®, Zolgensma® and Zynteglo®.11-13 Novel payment models, including annuity-based and subscription payments that spread the cost over time or create certainty of the cost expected, can mitigate affordability concerns, but can be resource-intensive to establish and maintain – and require a level of infrastructure which may not be present in all countries.14,15
Conclusions and implications
The advent of potentially curative cell and gene therapies poses significant disruption for healthcare systems. Implications must be considered to ensure that patients, HCPs and wider communities can prosper from the benefits of these therapies. Additionally, there will be significant adjustments to current healthcare systems affecting diagnosis, treatment expertise and infrastructure, value assessment and pricing and market access. Over the next few years, we expect the following issues to have implications for several healthcare stakeholders:
Healthcare disruption
Extent of disruption
Stakeholder implications
Dealing with uncertainty and importance of data and registries
Very High
It is critical that payers develop rules for RWD consideration and collection to curtail uncertainty around curative therapy durability. The establishment of a registry in some markets may be a condition for cell and gene therapy access at launch.
Addressing budget impact through novel payment model
Very High
Budget impact concerns may lead payers and manufacturers to develop novel access and payment models that allow for the spread of payment and impact on the budget holder across many years and as patient outcomes are realised.
Appropriate assessment of value
High
HTA bodies and payers will need to take a longer-term, broader-based, and more flexible approach as the long-term health and indirect benefits of curative therapies continue to be understood and tested.
Shifting role of primary and secondary settings
High
Specialised nature of curative treatments will lead to a clustering of care expertise and infrastructure needed for CoEs, where multidisciplinary teams provide integrated care. Primary care may require a shift in use towards other patients or possibly disease areas.
Building an appropriate hospital infrastructure
High
Extensive support for building hospital infrastructure for preparation, delivery, and administration of curative treatments will be required in dedicated centres or integrated within existing hospitals.
Need for earlier and targeted diagnosis
Medium
To realise the full benefits of curative treatments, patients need to be diagnosed early in life, requiring continuous support and broader access to new-born screening in genetic disease areas.
Need for new healthcare expertise
Low
HCPs involved in the current care process will either need retraining or shift to serving patient populations not targeted through gene and cell therapies.
Role of cross-border care
Low
Availability of many curative cell and gene treatments will require a renewed effort to clearly determine regulation attached to provision and reimbursement for cross-border care, given treatment may be provided only in select centres.
About the Authors
Artes Haderi is a Principal within the London office of the Life Sciences Practice at Charles River Associates (CRA). She specialises in public health and pharmaceutical policy and has considerable experience providing analytical solutions to biopharma companies and trade associations.
Anna Hillel is a Consulting Associate within the Boston office of the Life Sciences Practice at CRA. She focuses on pricing and market access engagements, with experience across US and EU.
Rebecca Steele is an Associate within the London office of the Life Sciences Practice at CRA. She works across strategy and policy consulting, with experience in rare diseases and the launch of gene therapies.
The views expressed herein are the authors’ and not those of CRA or any of the organisations with which the authors are affiliated. The authors wish to acknowledge the contributions of Lev Gerlovin and Neil Turner to this article.
Lara-Pezzi E, Dopazo A, Manzanares M. Understanding cardiovascular disease: a journey through the genome (and what we found there). Disease Models & Mechanisms. 5, pp. 434-443 (2012).
This website uses cookies to enable, optimise and analyse site operations, as well as to provide personalised content and allow you to connect to social media. By clicking "I agree" you consent to the use of cookies for non-essential functions and the related processing of personal data. You can adjust your cookie and associated data processing preferences at any time via our "Cookie Settings". Please view our Cookie Policy to learn more about the use of cookies on our website.
This website uses cookies to improve your experience while you navigate through the website. Out of these cookies, the cookies that are categorised as ”Necessary” are stored on your browser as they are as essential for the working of basic functionalities of the website. For our other types of cookies “Advertising & Targeting”, “Analytics” and “Performance”, these help us analyse and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these different types of cookies. But opting out of some of these cookies may have an effect on your browsing experience. You can adjust the available sliders to ‘Enabled’ or ‘Disabled’, then click ‘Save and Accept’. View our Cookie Policy page.
Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.
Cookie
Description
cookielawinfo-checkbox-advertising-targeting
The cookie is set by GDPR cookie consent to record the user consent for the cookies in the category "Advertising & Targeting".
cookielawinfo-checkbox-analytics
This cookie is set by GDPR Cookie Consent WordPress Plugin. The cookie is used to remember the user consent for the cookies under the category "Analytics".
cookielawinfo-checkbox-necessary
This cookie is set by GDPR Cookie Consent plugin. The cookie is used to store the user consent for the cookies in the category "Necessary".
cookielawinfo-checkbox-performance
This cookie is set by GDPR Cookie Consent WordPress Plugin. The cookie is used to remember the user consent for the cookies under the category "Performance".
PHPSESSID
This cookie is native to PHP applications. The cookie is used to store and identify a users' unique session ID for the purpose of managing user session on the website. The cookie is a session cookies and is deleted when all the browser windows are closed.
viewed_cookie_policy
The cookie is set by the GDPR Cookie Consent plugin and is used to store whether or not user has consented to the use of cookies. It does not store any personal data.
zmember_logged
This session cookie is served by our membership/subscription system and controls whether you are able to see content which is only available to logged in users.
Performance cookies are includes cookies that deliver enhanced functionalities of the website, such as caching. These cookies do not store any personal information.
Cookie
Description
cf_ob_info
This cookie is set by Cloudflare content delivery network and, in conjunction with the cookie 'cf_use_ob', is used to determine whether it should continue serving “Always Online” until the cookie expires.
cf_use_ob
This cookie is set by Cloudflare content delivery network and is used to determine whether it should continue serving “Always Online” until the cookie expires.
free_subscription_only
This session cookie is served by our membership/subscription system and controls which types of content you are able to access.
ls_smartpush
This cookie is set by Litespeed Server and allows the server to store settings to help improve performance of the site.
one_signal_sdk_db
This cookie is set by OneSignal push notifications and is used for storing user preferences in connection with their notification permission status.
YSC
This cookie is set by Youtube and is used to track the views of embedded videos.
Analytics cookies collect information about your use of the content, and in combination with previously collected information, are used to measure, understand, and report on your usage of this website.
Cookie
Description
bcookie
This cookie is set by LinkedIn. The purpose of the cookie is to enable LinkedIn functionalities on the page.
GPS
This cookie is set by YouTube and registers a unique ID for tracking users based on their geographical location
lang
This cookie is set by LinkedIn and is used to store the language preferences of a user to serve up content in that stored language the next time user visit the website.
lidc
This cookie is set by LinkedIn and used for routing.
lissc
This cookie is set by LinkedIn share Buttons and ad tags.
vuid
We embed videos from our official Vimeo channel. When you press play, Vimeo will drop third party cookies to enable the video to play and to see how long a viewer has watched the video. This cookie does not track individuals.
wow.anonymousId
This cookie is set by Spotler and tracks an anonymous visitor ID.
wow.schedule
This cookie is set by Spotler and enables it to track the Load Balance Session Queue.
wow.session
This cookie is set by Spotler to track the Internet Information Services (IIS) session state.
wow.utmvalues
This cookie is set by Spotler and stores the UTM values for the session. UTM values are specific text strings that are appended to URLs that allow Communigator to track the URLs and the UTM values when they get clicked on.
_ga
This cookie is set by Google Analytics and is used to calculate visitor, session, campaign data and keep track of site usage for the site's analytics report. It stores information anonymously and assign a randomly generated number to identify unique visitors.
_gat
This cookies is set by Google Universal Analytics to throttle the request rate to limit the collection of data on high traffic sites.
_gid
This cookie is set by Google Analytics and is used to store information of how visitors use a website and helps in creating an analytics report of how the website is doing. The data collected including the number visitors, the source where they have come from, and the pages visited in an anonymous form.
Advertising and targeting cookies help us provide our visitors with relevant ads and marketing campaigns.
Cookie
Description
advanced_ads_browser_width
This cookie is set by Advanced Ads and measures the browser width.
advanced_ads_page_impressions
This cookie is set by Advanced Ads and measures the number of previous page impressions.
advanced_ads_pro_server_info
This cookie is set by Advanced Ads and sets geo-location, user role and user capabilities. It is used by cache busting in Advanced Ads Pro when the appropriate visitor conditions are used.
advanced_ads_pro_visitor_referrer
This cookie is set by Advanced Ads and sets the referrer URL.
bscookie
This cookie is a browser ID cookie set by LinkedIn share Buttons and ad tags.
IDE
This cookie is set by Google DoubleClick and stores information about how the user uses the website and any other advertisement before visiting the website. This is used to present users with ads that are relevant to them according to the user profile.
li_sugr
This cookie is set by LinkedIn and is used for tracking.
UserMatchHistory
This cookie is set by Linkedin and is used to track visitors on multiple websites, in order to present relevant advertisement based on the visitor's preferences.
VISITOR_INFO1_LIVE
This cookie is set by YouTube. Used to track the information of the embedded YouTube videos on a website.