Friday 6 March 2015

Working with Pharma. Ed Owen's US blog part 2

As well as meeting the Cystic Fibrosis Foundation in the US last week, the Trust’s Chief Executive, Ed Owen, and Director of Research & Care, Dr Janet Allen, also dropped in on two pharmaceutical companies of importance to cystic fibrosis, Novartis and Vertex. Here is Ed’s second blog explaining what they find out:

Much is said about the pharmaceutical industry. Some of it is fair, some less so. But the fact remains that only pharma companies have the size, expertise and capability to develop and produce the drugs that can make a difference to people with cystic fibrosis. So it is vital that we have strong relationships with key companies, like Vertex and Novartis, and work with them for the benefit of our community. 

The Vertex story is an extraordinary one, and one in which our sister charity, the Cystic Fibrosis Foundation (CFF), has played a central role. It also tells us much about the industry, its weaknesses and strengths, limitations and potential.

Long frustrated by the lack of industry interest in cystic fibrosis in the late 1990s and early 2000s, the CFF began funding a number of commercial programmes, including one led by a biotech company called Aurora Sciences, later bought by Vertex, to find compounds to develop drugs to tackle the genetic causes of cystic fibrosis.

A decade and a half on, and Vertex is a pharmaceutical trailblazer. Ivacaftor (Kalydeco), is transforming the lives of those with the G551D mutation and a combination therapy of ivacaftor and lumacaftor has been submitted for approval for use in people with two copies of the F508del mutation.

Being a trailblazer however comes at a price, and the high price of ivacaftor has been controversial – with many questions about the likely price of the combination therapy if it is granted approval from the regulators.

With a cash-strapped NHS, and a system of drug appraisal that in England, at least, is ill-suited to assess particular issues relevant to conditions like cystic fibrosis, there is a real risk that this combination therapy is given regulatory approval in Europe later this year for use in four out of 10 people with cystic fibrosis but will not reach those that need it here in the UK for a considerable amount of time, if at all.

Our focus must be to ensure that approved therapies get to those who need them as quickly as possible - and we are discussing these issues now with all key players.

But, when we met them last week, Vertex continued to be tight-lipped about future pricing in advance of regulatory approval. Along with other pharmaceutical companies, the company argues that the price of drugs reflects both the high cost of investment that has been made over many years to develop and trial therapies (estimates vary, but the average cost of producing a new drug is usually well above £1bn) and the relatively small numbers of patients with ‘rare diseases’ like cystic fibrosis.

Like every other pharmaceutical company, Vertex has shareholders who demand an appropriate financial return on their investment. The long-term prospects of the cystic fibrosis ‘market’ means that Vertex is continuing to invest in both their immediate pipeline of potential therapies and in longer-term research to develop what they describe as ‘second generation’ drugs that they believe could be even more effective in future years.

We are delighted they are doing so, and are keen to see other drugs companies invest in cystic fibrosis research too. One such company is Novartis. It already produces cystic fibrosis drugs, most commonly Tobramycin. But we have had concerns about its future commitment following its decision to move its wider respiratory research base from Sussex in the UK to Boston in the US in late 2013.

We were therefore pleased to meet the Novartis team in Boston last week and to receive their reassurance that their cystic fibrosis-related drug programmes were alive and well.

For the first time in decades, the pharmaceutical industry is investing heavily in cystic fibrosis. As President Obama said in January, the condition is at the cutting edge of new advances in ‘precision medicine’ with new therapies being developed focused on defined patient groups with particular genetic mutations.

That investment will only continue for as long as companies believe that health systems will pay for the drugs that follow. Therein lies the challenge for the NHS, ourselves and all those wishing to see innovative treatments that transform the lives of people with cystic fibrosis accelerated as quickly as possible.

High-cost drugs are the future. How they are paid for is the key question that we all need to address. 

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