Getting drugs to market has never been easy, with regulators rightly demanding high standards in terms of clinical effectiveness, safety and the value proposition. But the influence of health technology assessment (HTA) bodies in Europe, such as NICE in the UK and Germany’s AMNOG pricing system, mean that pharma companies have several barriers to overcome before they are able to launch.
And on the other side of the Atlantic, US payers are becoming concerned about the high price tags for drugs – which can also be a barrier to access as prescription costs will often be funded in part or entirely by the patient.
According to Rosanne Campbell, senior project leader for value, access and HEOR, and Andrew Thomas, managing director for PR UK, pharma companies must start thinking about matters relating to price early on in drug development to maximise their chances of getting the product to market at a good price.
They added that it’s not always as simple as NICE being intransigent, or pharma companies being greedy – often it’s a case of miscommunication that occurs because of a drug development culture where companies are rewarded on the basis of clinical milestones.
Thomas said: “We see this a lot when we work with smaller companies, like biotechs, who are very clinical-focused. They’ll have one product that they, understandably, put their heart and soul into, and they’re so focused on getting regulatory approval that they’re not thinking about the commercial side and getting access soon enough in the strategic planning process.”
This also does not fit well with NICE, which is increasingly looking for engagement with drug developers early in the clinical development process.
Much of early clinical development is overseen by smaller biotechs because of big pharma’s tendency to outsource this stage of research or get involved much later on in the clinical development process.
Consequently, the needs of HTA bodies are still often overlooked in the early stages.
Campbell said: “HTA bodies really want early engagement. If we look at NICE, they’re set up in such a way to encourage that. They have the Office of Market Access and Early Scientific Advice.
“They want you working with them early on so that you are developing the right evidence that’s going to help you to get access.”
According to Campbell, HTA bodies are becoming more confident and are clear in their demands about what they will and will not accept from pharma.
Their goal is to guide development so that drugs come in at the right price and come to the market as soon as possible.
While NICE is accommodating in terms of providing feedback during the clinical development process, it is rigid when it comes to enforcing its cost-effectiveness threshold.
“They’re saying, ‘Don’t mess us around. Don’t come to us with a QALY of £32,000, come to us with a QALY of £30,000’,” Campbell said.
Unfortunately, NICE’s approach is at odds with a trend towards clinical development where drugs are approved on the basis of earlier data.
Regulators in both the US and Europe are willing to accept findings from phase 2 trials and grant conditional licences if manufacturers commit to providing confirmatory data from large-scale studies.
But bodies such as NICE and Germany’s G-BA and IQWiG often rely on phase 3 data to measure how newly approved drugs perform compared with standard care.
NICE struggles with data from phase 2 trials which may often have a single arm, resulting in uncertainties following its number-crunching.
Thomas said: “It’s trying to get pharma companies to look beyond the regulatory stage, and towards the commercial end of things.”
Another challenge is to convince payers about the value of the innovative products. With so many companies trying to argue that higher prices are justified because of innovation, payers want to see hard evidence of any benefits.
Thomas said: “We have to innovate. We need to bring new products through, but it’s about specificity. Innovation for what purpose, for who and when? It now needs to be more quantifiable and qualifiable innovation.”
Campbell added: “You have to tick certain boxes, which can have advantages and disadvantages. Obviously, the advantage is that it can help you focus, in terms of you as a company and what you need to be working towards to really demonstrate the value.
“But then if your product is innovative, the rules may be restrictive, or it may be difficult for you to really demonstrate its value.
“That can cause problems and lead to undervaluation of your product. That’s not such a positive thing for pharma.”
In many cases the missing link can be the patients’ perspectives. They have the insight that can allow companies to make the case for their product and help to convince payers about its value. Campbell cites the example of cystic fibrosis drugs in Ireland, which were eventually funded as a result of campaigning by patients.
Campbell said: “It’s a super expensive drug. It was taking up a lot of the healthcare budget, but patients had a big voice there and it got in on the floor of Parliament. Then suddenly they were reassessing things. I do think the patient is getting more powerful. They’re a lot more informed now. They have a stronger voice.”
It’s worth noting that campaigning by patients in the UK has not been able to resolve the differences between health technology assessment bodies and Vertex for their expensive cystic fibrosis drug Orkambi – but there have been some notable successes.
The Scottish Medicines Consortium (SMC) earlier this month used its Patient and Clinician Engagement (PACE) in a process that ended with a funding recommendation for Akcea’s Tegsedi for the rare muscle wasting disease spinal muscular atrophy (SMA).
Although the SMC rejected Vertex’s latest cystic fibrosis combination Symkevi using the same process, this demonstrates that patient input can be a deciding factor when it comes to health technology assessment and market access.
Campbell said: “It’s the same in NICE committee meetings as well. The patients are there, they are represented, they have a place at the table.”
Making a compelling case about the value of the product will therefore require pharma companies and biotechs to be well coordinated internally, and to begin dialogues with organisations such as NICE as early as possible in the development process.
Thomas said: “Oftentimes you’ll still find people working within silos in a pharmaceutical organisation. We would always argue that there are huge advantages to be realised from greater integration – both in terms of resource allocation and intellectual efficiency.”
Campbell concluded that the key to success with organisations such as NICE is to be willing to accept advice from outside sources, and to start thinking about arguments on value as early in clinical development as possible.
“You need to have that back-of-mind when you’re preparing your clinical trial, when you’re getting ready for market access, when it’s approaching the commercialising stage.
“You have a better chance of getting your story across with a clear strategy in place.”
Rosanne Campbell is senior project leader for value, access and HEOR of Syneos Health Consulting, based in London. Rosanne brings more than eight years of experience in HEOR and PMA consulting to Syneos Health’s Pricing and Market Access practice in Europe. She has worked on a wide range of projects including economic models, literature reviews, reimbursement dossiers and market access strategy across Europe, Asia and the US. Rosanne studied in Ireland and London with degrees in Business, Economics and Health Economics.
Andrew Thomas is managing director of Syneos Health PR UK, based in London. He has more than 18 years’ experience of running national, regional and global communications campaigns for pharmaceutical and biotech clients as well as disease awareness initiatives for large NGOs such as the World Health Organization. He also has considerable client-side experience having held senior roles in Pfizer with responsibility for some of the organisation’s biggest brands and product launches.
Syneos Health is the only fully integrated biopharmaceutical solutions organisation. The company, including a contract research organisation (CRO) and contract commercial organisation (CCO), is purpose-built to accelerate customer performance to address modern market realities. Created through the merger of two industry-leading companies – INC Research and inVentiv Health – it brings together approximately 24,000 clinical and commercial minds to help its biopharmaceutical customers shorten the distance from lab to life. Learn more at syneoshealth.com