The challenge of attracting gene and cell therapy makers

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When it comes to gene and cell therapies, Australia appears to be suffering from the combined consequences of a bad global reputation when it comes to reimbursement and pricing, a small commercial market and poor exchange rate.

Australia is well down the list of launch countries for many companies with gene and cell therapies, behind larger developed countries and even some developing countries with no reimbursement systems but large private markets. 

The upshot is patients could be waiting years for local access to what could be curative life-changing treatments.

The added irony is that gene and cell therapies being developed by local emerging biopharmaceutical companies are probably more likely to be launched in other markets before Australia because of simple commercial realities.

Companies with an established long-term presence in Australia, like Novartis and Roche, have the institutionalised capability to bring their emerging gene and cell therapies to this market.

However, large global biopharmaceutical companies like these Swiss giants are not responsible for the bulk of innovation in this area. The majority of gene and cell therapies currently in late-stage development are being commercialised by small to mid-size biotherapeutic companies.

Most of these companies are headquartered in the US or Europe and few have any presence in Australia, let alone a disclosed commercial plan to bring their therapies to this market or even a capability to deal directly with the government.

Industry sources have told PharmaDispatch that, in some cases, Australia is not even on the list of launch countries and patients are likely to be waiting several years as companies establish and grow their commercial models in other markets.

US-based Alnylam Pharmaceuticals is commercialising the first RNA interference therapeutic for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy. 

The company's ONPATTRO (patisiran) has been approved in the US, Europe and Japan. The company is in the process of deploying 250 employees in customer-facing roles and has disclosed plans to launch ONPATTRO in Canada and Brazil.

The company has clinical trial sites in Australia but it has no disclosed plan to bring ONPATTRO here for the around 150 people with hATTR amyloidosis.

Companies with gene and cell therapies approved in other markets include Bluebird Bio and Sarepta Therapeutics.

It is understood there has been some discussion with potential Australian joint venture partners.

However, the US companies have baulked given costs associated with establishing even a limited local presence to manage a joint venture and navigating the regulatory and reimbursement pathways. The cost challenge is complicated by the small market, the time to funded access, the pricing environment and the negative impact of the exchange rate.

It seems few of these companies appear willing to redirect limited resources from other potentially more lucrative countries to a relatively small market associated with so many challenges and significant uncertainty.

Can we expect these companies to invest the millions required to commercialise one of these therapies in Australia when they realistically confront a wait of several years and a process they will expect to end with the lowest price in the world? 

Health minister Greg Hunt and the recently re-elected Coalition government have focussed significant attention on investing in R&D related to gene and cell therapies, highlighted by its $80 million commitment to the Melbourne-based Peter MacCallum for its Centre for Excellence in Cellular Immunotherapy.

Yet the policy environment underpinning the commercial reality for biotherapeutic companies is still characterised by significant confusion and uncertainty.

The traditional challenge associated with navigating Australia's reimbursement and pricing process is made even harder by the early and limited nature of evidence associated with gene and cell therapies. Australian processes have traditionally struggled with virtually any level of uncertainty, even for traditional small molecule medicines with trials based on thousands of patients.

Gene and cell therapies have the potential to be curative and effectively restore several decades of healthy lifespan. Yet they often treat rare disorders where early evidence is based on small early-stage trials. This means uncertainty, and history shows it is the one thing Australian decision-makers dislike virtually more than any other.

This is creating confusion and conflict in the policy environment because the government clearly wants to support patient access to gene and cell therapies.

Mr Hunt's announced funding for Novartis' CAR-T therapy KYMRIAH (tisagenlecleucel) before the recent election. 

KYMRIAH was funded for the small patient group of paediatric and young adult patients up to 25 years old with B-cell precursor acute lymphoblastic leukaemia (ALL). However, the Medical Services Advisory Committee is yet to approve KYMRIAH for the much larger group of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL).

The data for KYMRIAH is early and uncertain. It is yet to navigate the official approval process but the federal government is sending DLBCL patients overseas for CAR-T therapy. It is the least cost-effective and arguably most inequitable way of funding access given overseas travel is not an option available to all patients.

In other words, while the federal government's official channels are using their traditional approach and grappling with the uncertainties of gene and cell therapy, it is paying to send people overseas for treatment based on other sources of advice.

The government clearly already accepts the efficacy and value of CAR-T - well, part of it does - it must be frustrating for Mr Hunt.

Yet the worrying reality for Australia is that, even if the federal government is able to establish a clear and timely funding pathway for gene and cell therapies, it may not be enough to overcome in the short to medium term a poor reputation, market size, and the fact many of the relevant companies are currently focussed on establishing the viability of their commercial models in the eyes of investors in larger and more lucrative markets.

More direct action may be required - pull factors - if patients in Australia are to gain funded access to gene and cell therapies in a way comparable to those in other developed countries.