Adding CAR-T to Cart: What are the hurdles impacting patient access to cell and gene therapies?

Woman lying in hospital bed. Image credit: Sharon McCutcheon on Unsplash

Innovative and curative treatments continually push the boundaries of treating known medical conditions. However, as the world changes gears, shifting from small molecules to cellular and gene therapies, now is an excellent time to take stock. With the increasing complexities of manufacturing and delivering cellular and gene therapies to patients, now is the time to interrogate emerging CAR-T therapies and ask: what does it take to access these lifesaving treatments?

Australia is an innovative country. With inventions from the Cochlear implant to the HPV vaccine all originating from our proverbial backyard, Australian scientists have made a significant impact on the health and wellbeing of millions around the world. With our innovative spirit driving our transnational contributions to medicine, it would make sense for us to also be on the receiving end. And yet, Australians have lamented about lack of access to some of the most innovative therapies in the world. While there is a special access scheme under the auspices of the Therapeutic Goods Administration (TGA), some might argue that patients’ desire to access innovative medicines is more akin to the controversial ‘Right-to-try’ access scheme, previously covered in Signals. The ‘Right-to-try’ scheme allows terminally ill patients the opportunity to access experimental therapies that have successfully concluded Phase 1 safety trials.

Compounding this issue is the difficulty of enrolling in international clinical trials or lack of a local site in Australia as a means to access novel treatments. This has been a major obstacle for Australian patients, but the situation has been improved due to the efforts of lobby organisations such as MTPConnect and, in no small part, the attractiveness of the Federal Government’s Research and Development Tax Incentive. Despite this, there is a long way to go to bring new and emerging therapies to our shores.

One such treatment is Chimeric Antigen Receptor (CAR) T-cell therapy. Currently one of the most prominent of the cancer immunotherapies, CAR-T therapy is attracting the interest of both healthcare providers and investors, especially after the FDA’s approval of Novartis’ Kymriah and Kite Pharma’s Yescarta, respectively. To ensure that Australian patients benefit from this lifesaving treatment, the two leading Australian political parties, the Australian Labor Party and the Liberal Party of Australia, both pledged their commitment before the 2019 election to provide cancer patients access to innovative cancer treatments. At the time of writing, Australians can soon access CAR-T therapy at the Peter MacCallum Cancer Centre and at the Royal Children’s Hospital, both in the state of Victoria.

Importantly, facilitating patient access to novel treatments is not merely a political issue but also an economical one. With CAR-T therapies, the hurdle of cost immediately comes to mind. Kymriah is estimated to be more than $AUD500,000 or less if indication-based pricing is practised. This means that the ability to deliver Kymriah at the Peter MacCallum Cancer Centre has required an $AUD80 million investment from the Australian Government. It is reassuring that both federal and state governments have repeatedly reaffirmed their commitment to providing Australians access to the latest therapies.

However, we should consider the supporting systems, infrastructure and reimbursement schemes that make it commercially attractive for biopharmaceutical companies to register their therapies in Australia. These initiatives would act in concert with health policy, reimbursement agencies guided by health technology assessments, and the TGA to lower barriers and expand Australian access to new, potentially life-saving drugs. To this end, we can imagine electronic medical records being an agent of change by acting as a catalyst and enabler of more creative reimbursement models.

Electronic medical records would not only track clinical outcomes associated with novel therapeutics, but they could also gather and collate the pharmacovigilance data required to differentiate between any medium to long term adverse reactions from other acquired afflictions during the remainder of a patient’s natural life. With this, we can accurately match the cost of the therapy against its benefits and thereby be in a stronger position to shape, update and reinvent reimbursement models.

Having said that, electronic medical records have been tried and tested globally to mixed results. The Australian Government’s MyHealth Record was met with skepticism. Over 2.5 million Australians have opted out over reported concerns about cybersecurity and potential misuse of data. Such criticisms of the system were understandable and well-founded. A Victorian hospital was hacked in early 2019 and, more recently, approximately 37,000 medical histories belonging to Australians may have been exposed after security vulnerabilities within an Australian company offering clinical trial matching services were exploited. Nevertheless, if new forms of reimbursement are necessary to subsidise high cost treatments, it is then necessary to rethink the opportunities of electronic medical records.

Innovation is not only restricted to the therapies themselves. There is a need for more innovative funding models and regulatory frameworks, otherwise carrying the financial cost of these treatments will simply get too expensive. To avoid negatively impacting patient eligibility, changing how we pay for novel therapeutics will be of paramount importance to maintaining and even improving access.

Our blog is just one of many covering the topic of “What are the hurdles impacting patient access to cell and gene therapies as part of Signal’s fourth annual blog carnival. Please click here to read what other bloggers think about this.


Image credit: Sharon McCutcheon on Unsplash

About CCRM Australia:

CCRM Australia is an Australian not-for-profit organisation supporting the development of foundational technologies to accelerate the commercialisation of regenerative medicine products and therapies. CCRM Australia’s focus is to bridge the commercialisation gap through a network of scientists, entrepreneurs, academic institutions and industry partners and address bottlenecks in the industry. CCRM Australia is modelled on the highly successful CCRM in Canada and is legally separate to CCRM. As a member of the Global CCRM network, CCRM Australia is a partner to a leading-edge industry consortium. CCRM Australia is supported by MTPConnect and the Victorian State Government.

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