All routes to market are littered with chicanes and hairpin bends that confound the best intentions. The journey is imprinted on the psyche but then you look up and find yourself in a dead-end street. Room for manoeuvre has evaporated and, in the costly world of drug development, that could spell disaster.
But now there is a new framework claiming to have satnav qualities to guide development programmes to a better destination.
BEACON is a new evaluation framework that aims to refocus the fundamentals to put payer needs at the heart of decision-making to help multinational companies maximise the chances of patient access and success.
It is a big ask. Even the most scientifically impressive medical breakthroughs are not immune to the payer challenge and achieving customer access. Witness Gilead’s hepatitis C drug sovaldi (sofosbuvir) which has glowing efficacy results yet is having a troubled transition into the market place.
The UK’s NHS is reluctant to fund its estimated £1bn cost for 20,000 people treated and the French health minister Marisol Touraine has suggested international collaboration to tackle patient access roadblocks on the drug’s progress.
Destructive
Given that Pfizer had to pull its powder-based insulin drug Exubera after it had returned just $13m in sales from an 11-year $2.8bn development project, it seems that help is needed across the spectrum.
BEACON, designed by Mundipharma International and the result of a trawl through oceans of health technology assessment (HTA) paperwork, makes it easier to identify issues before they become destructive and damage a drug’s chances of reimbursement.
It was summarised in a paper published in the peer-reviewed journal PharmacoEconomics: “Both pharmaceutical companies and payers are under increasing pressure to meet the growing healthcare needs of society. To ensure that innovative pharmaceuticals are reaching the market, and to ensure timely access for patients, pharmaceutical companies need to focus on intelligent innovation by instilling payer decision-makers’ needs through the entire development process.”
The company has tested the model and believes it can be a vital support at a time when development costs and returns appear to be heading in opposite directions. The bench-to-shelf journey was recently assessed as 12 years and £1.15bn per drug by the Association of British Pharmaceutical Industry. A gloomy study in Nature Biotech in July 2014 reported that only 32% of drugs will make it to phase III trials and only 10% to market.
The inhospitable climate is deepened by extra layers of local cost scrutiny even after approval by the European Medicines Agency (EMA) or the the US Food & Drug Administration (FDA).
‘Too expensive’
“We needed the BEACON framework because there are so many guidelines and changing payer perspectives around the world which can be difficult to incorporate into a global pharmaceutical development programme,” says Will Dunlop, head of market access for Mundipharma International, who has been developing the project over the last five years. “Drug development programmes have a large number of stakeholders, such as drug safety, patent, legal, business development, commercial and R&D leads, and even though common sense tells you that one of the key purposes of a drug is to meet that payer need, it is very easy for it to get lost in the process.
“The extreme case is that drugs don’t get reimbursed. A company can invest billions and at launch a government says: ‘Sorry, it is too expensive and you have not provided the right data.’ That is a huge waste.”
BEACON – with its six principles of Burden, Environment, Affordability/Value, Comparator, Outcomes and Number of Studies/Evidence – was borne from a company-wide review of assets which identified a need to optimise the value of drugs. A deep literature search of existing frameworks and guidance from national bodies was commissioned with a panel of international experts.
“There are thousands of pages of guidance around the world, a daunting amount. But, on close examination, fundamental principles come through regardless of the country and regardless of the particular body,” says Dunlop. “Although we deal with a lot of health systems some very common themes came through the research and it became clear that no-one had summarised them, at least not in a formal way. The concepts are not new but no one has locked them down before.
Elements
“Most of the guidance is written by government agencies and there is a danger of information paralysis as you try to incorporate them into very complex internal decision-making systems. What we are trying to do is simplify that down to six elements so you can have conversations and cross-functional working groups.
“A big reason that drugs don’t go forward is that they don’t meet customer payer needs because sometimes that focus is lost along the way. If you concentrate on these six fundamental elements, we believe your chances are much higher and patient access is improved.”
The framework is anchored to a concept of ‘When science is not enough’, an approach Mundipharma used to emphasise the need to couple practical business decisions to the scientific excitement of a new molecule or proof of concept.
“Traditional pharma companies are all about natural science and drivers such as: ‘How can we do the most impressive natural science and develop the great drug.’ Our challenge to that is you also need social sciences to make sure any new drug is appropriate for the health economy because the patient loses out if it isn’t,” adds Dunlop. “You may have a very interesting drug scientifically but if does not meet the customer needs – and the budget holder is the most important part of the system – then it may not be any good.
“If pharma doesn’t look through a fresh lens like BEACON then we could see more drugs failing at huge costs and companies not getting a return on investment. In the long term, companies will either have less money to invest in new drugs or not exist. Everyone wants to see drugs that have true value for all customers because that will create a stronger industry and a better chance of more patients getting the drugs they need.”
The framework is designed to be an ever-present metric in a drug’s development and allow easy and quick recalibrations along the way.
“BEACON assessments can be done as rapidly as required – from 30 minutes in some cases to a more in-depth two weeks – and be revisited regularly to optimise the value of the drug from a payer point of view,” says Dunlop. “It should allow companies to keep developing an asset and have confidence that it will get to the patient. In extreme cases, you may even decide that the drug is never going to fit the payer needs and could then stop a project to save waste further down the line.
Customer focus
“It is about having the right guiding proposition and the right data package to optimise the value of a drug.”
The framework was published with open access in a peer reviewed journal to communicate that Mundipharma, a global leader in pain medicines with a growing presence in other therapeutic areas, is serious about addressing hurdles to patient access.
Dunlop believes the framework has a role in spreading harmony across the different departments and functions of a multinational company so that the development focus is not clouded.
“If you are a lawyer working on a patent then you may not know about the payer needs and budget constraints in a particular health system,” he adds. “BEACON makes it easier for different stakeholders to communicate during a drug’s development.
“It allows everyone and every department to have a regular sense check and make slight adjustments if needed. Too often, the excitement around the product leads to a ‘We’ve started so we’ll have to finish’ approach and customer focus gets lost along the way.
“BEACON allows you to start to align patient needs with the payer needs from the start and build the right clinical data packages to meet those needs.”
Drug development will continue to be a cost-heavy process with high attrition rates but Mundipharma believes its framework can drastically reduce the amount that fail for commercial reasons.
“It is about keeping drug development on track and if we do that then industry can save money and work more efficiently, and payers will get the drugs they want at the value they want while patients get the drugs they need,” says Dunlop.
“Sometimes science isn’t enough.”