It’s more than a year ago since Spark Therapeutics launched its revolutionary gene therapy Luxturna, a one-shot treatment that can hugely improve sight for people with a rare disease seriously causing impaired vision.
Luxturna is a single injection used to treat Leber’s congenital amaurosis, where people have a mutated RPE65 gene, meaning they are often unable to see at all at night, and have seriously impaired vision in daylight.
It works by delivering 150 billion viral vector particles containing a correct copy of the RP65 gene to retinal cells, restoring the patient’s ability to make the missing enzyme and dramatically improving their sight. But Luxturna is also hugely expensive – costing $850,000 to treat both eyes.
Meanwhile, CAR-T (chimeric antigen receptor T-cell) therapies are being used in cancer, potentially saving the lives of patients with advanced blood cancers who would have almost certainly died in the past.
Patients taking these drugs have their T-cells harvested by electrophoresis, then they are genetically modified to attack cancer cells before being injected back into the patient. The process can take weeks and results can be powerful, potentially saving the lives of the patients.
But these drugs are also phenomenally expensive. Gilead’s CAR-T Yescarta costs $373,000, while Novartis’ Kymriah costs up to $475,000 per patient, and patients can require constant monitoring and intensive care because of the extreme side effects the drugs can cause.
These drugs are seldom used as they are only approved in the sickest patients, but again these high up-front costs require new methods of reimbursement.
Novartis has recognised this and in the US the company itself reimburses the drug’s cost in full if it does not work – trial data shows around half of patients respond partially and around 32% achieve a complete response in patients with relapsed or refractory large B-cell lymphoma.
In the US many people pay some or all of their medicine bill, depending on their insurance arrangements, and many would simply be unable to find this kind of money.
The eye condition Luxturna treats is rare, affecting one in 50,000 people, but with US trade body PhRMA estimating there to be around 300 cell therapies in development for various diseases, healthcare systems and patients will increasingly face funding challenges for these therapies if existing reimbursement models don’t change.
Andrew Powaleny, director of public affairs at the US trade body PhRMA, insisted that these new technologies are affordable for healthcare systems if these value-based approaches to pricing are used.
Strategies such as those used by Novartis could make cell and gene therapies affordable despite their high up-front costs, he argued.
He said: “As these therapies grow in prevalence, one key challenge is in reimbursement for these medicines. An example of this is CAR-T treatments which are reimbursed under Medicare Part A, which results in hospitals receiving a much lower amount than the cost of the treatment.
PhRMA cited US government figures predicting that spending on retail medicines will increase 5-7% annually, while retail medicine spending is projected to remain at around 10% of total national health expenditure.
Powaleny said the US healthcare system regulates drug spend as older drugs go off-patent and cheaper generics and biosimilars become available.
Therefore, PhRMA argues that with $140 billion of US brand sales facing competition from generics or biosimilars between 2017 and 2021, there is plenty of headroom for this new generation of cell-based drugs.
But not everyone is as optimistic. In Europe, the patient group EURORDIS is calling for action to ensure patients get access to rare disease therapies.
Last year the organisation published a position paper calling for a ‘four pillared approach’ to ensure patients can access the orphan medicines and therapies that are approved for their conditions.
European regulators approved a gene therapy for a rare disease as long ago as 2012, granting a licence for uniQure’s Glybera (alipogene tiparvovec) to reverse lipoprotein lipase deficiency, a rare inherited disorder that can cause severe pancreatitis.
But at a price of around 1 million euros, uniQure withdrew the drug in 2017 after it was reportedly used in just one patient.
In light of this and the growing pipeline of cell and gene therapies for rare diseases, EURORDIS is calling for a rethink of how these therapies are developed.
R&D needs to be revamped to be less expensive and faster and there needs to be early dialogue between developers and payers to establish a therapy’s value.
There should also be better communication between European healthcare systems over price, and price discounts early in a drug’s lifetime if there is uncertainty over clinical evidence.
With billions of dollars being invested in these new therapies, it is vital that healthcare systems and pharma work together to ensure patients can get access. But it will require joined-up thinking and new levels of cooperation between pharma and healthcare.
PhRMA’s Powaleny urged healthcare systems to realise the significant benefits that these therapies can bring.
Richard Staines is Senior Reporter at pharmaphorum. He has been a journalist since the 1990s and has written for websites, newspapers and magazines. He has always had an interest in health and has been focusing on the pharma industry since 2010, interviewing industry leaders and covering stories on topics including regulation, mergers and acquisitions, and the latest clinical developments.