Europe’s pharma companies join with non-profit group in push to measure outcomes of medical treatments

Europe’s pharma companies have signed up to a three-year deal with the International Consortium for Health Outcomes Measurement (ICHOM), in a move that adds significant heft to the push to implement value-based healthcare.

The alliance sees the European Federation of Pharmaceutical Industries and Associations (EFPIA) join forces with ICHOM to make the case for an outcomes-centred approach in Europe, showcasing examples of the model in action.

“There are many examples of collecting data for isolated surgical procedures, where there are easy-to-measure discrete outcomes,” said Thomas Allvin, executive director for strategy and healthcare systems at EFPIA. “We need to do more for conditions where medicines are central to disease management, including chronic conditions such as diabetes.”

“One of our key objectives is to get EU health to measure in a standardised way,” said

Allvin. “Only then can we work to design financial incentives to pay for outcomes.”

There are some powerful one-off examples of how outcomes can be collected and analysed, and an increasing number of pay-for-results deals are being struck between pharma companies and payers across Europe and in the US.

But different reporting standards and definitions make it hard to compare health data across Europe and a universal approach is needed to ensure the outcomes being measured are comparable from one setting to another.

Even countries with registries of surgical outcomes do not always collect high-quality, standardised data. “This is ICHOM's raison d’etre – they bring a lot of knowhow to the table,” said Allvin.

ICHOM aims to deliver outcomes that matter to patients at the lowest cost. From its formation in 2012, it has developed standard sets of patient outcomes for 23 disease areas.

These have been applied in some pockets, showing improved results for patients. However, there remains enormous variation across Europe in attitudes to, and awareness of value-based healthcare.

The first step for the EFPIA-ICHOM alliance will be awareness-raising, through workshops with policymakers, hospitals and health technology assessment bodies.

The two organisations aim to showcase examples of how to use the ICHOM model to improve outcomes, and may support the implementation of a standard set at a selected healthcare site. They will also explore ways to integrate the collection of standard outcomes data into electronic health records.

Pay-for-performance

Advances in biomedical science – often driven by publically-funded research – mean a wave of better and more effective drugs is reaching the market.

But rather than being a source of optimism, this innovation is further stirring controversy about drug pricing and patient access.

Even for drugs that show clear demonstrations of cost-effectiveness, the budget impact can mean Europe’s hard-pressed healthcare systems cannot provide them for all eligible patients.

A prime example is the use of nucleoside analogue drugs, which are in effect a cure for Hepatitis C infection, a long-term, chronic condition that can result in liver failure.

Despite the benefits for individual patients, the potential cost savings for healthcare systems and the possibility of drawing the cycle of infection to a close, countries across Europe have had to take the decision to phase introduction of these drugs, to avoid having to make cuts elsewhere.

Now healthcare systems need to prepare for advanced therapies, based on delivering cells, gene or tissues, which are starting to reach the market.

These products hold the promise that a one-off treatment will provide a cure – often for a rare disease where the only alternatives are palliative, or in cancer, when all other treatment options have been exhausted.

But they are expensive. Examples include Novartis’ CAR-T cell cancer immunotherapy Kymriah, approved in the US last year by the FDA and now in the European Medicines Agency’s (EMA) approval system. The US price is $475,000 per treatment (excluding the costs of administration and care).

Similarly, Gilead’s (formerly Kite Pharma’s) CAR-T cell product Yescarta, approved by the FDA in October and now in front of EMA, is priced at $373,000 per patient in the US, while Spark Inc’s Luxturna, a gene therapy for treating an inherited form of retinal dystrophy, a cause of blindness, also heading for EU approval, costs $850,000 per patient for treating both eyes.

Unlike Hepatitis C drugs, these treatments are for very small patient populations and, in the US, Novartis and Spark have agreed they will not be reimbursed for patients who do not respond. Even so, the number of advanced therapies in the later stages of clinical development will present a challenge for payers in the near future.

According to the industry body, the Alliance for Regenerative Medicine there are 82 advanced therapies in the final stages of clinical development currently.

Catalysing change

Against the backdrop of medical science breaking new ground and the accompanying controversy over prices and clamour for access, the tie-up between ICHOM and Europe’s biggest pharma industry body to work together on standardising patient outcomes is a significant development.

Both say there is no risk of compromising ICHOM's independence. “ICHOM's standard sets identify outcomes that providers should strive for, but are agnostic about how you get there,” said Allvin.

This will not change, said ICHOM chief Cristina Akerman. “We see ourselves as a catalyst in this area – a neutral, not-for-profit body focused on standardisation and bringing organisations together,” she said. “The life sciences are a very important stakeholder. That’s why we were eager to work with EFPIA.”

The collaboration will focus on developing infrastructure for data collection and implementing standard sets, rather than on selecting standard outcomes to be measured. “We have a very strict policy on engaging with the life sciences sector,” Akerman said. “Companies cannot fund the development of a specific standard set. Nor can they participate in working groups.”

The only opportunity for industry to provide input on developing standards is at roundtable meetings of stakeholders, held before working groups begin their work, and during an open review period before ICHOM reports are published.

All healthcare bodies can have a say during these phases. “There is a clear line in the sand,” Akerman said.

Reward results

ICHOM and EFPIA say defining and measuring outcomes should ultimately lead to the redesign of funding systems to reward results.

But Allvin does not believe the holy grail of value-based payments is imminent. “We think it’s important to start linking financial incentives to outcomes across the board, not just in pharma but for all services in healthcare. But there may not be a one-size-fits-all approach.”

Akerman agrees. “The long-term hope is to have systems where payment is focused on what is achieved,” she said. “But we should be careful about moving too fast. We have to get the basics right first. We can’t get to paying for results before we have a strong system for collecting and analysing outcomes.”

One of the benefits of working with a lobby group with EFPIA's clout may be the opportunity to push outcomes-based care up the EU agenda.

ICHOM is already working with the OECD on defining and collecting patient-based outcomes.  

“The EU has a role to play in sharing best practices between countries to show how this approach can work,” Akerman said.

While there is a lot of variation in Europe, publically-funded and single payer systems are well-suited to the outcomes-based healthcare approach, Akerman added. “Forming a strategic alliance with [EFPIA] can help us move forward in Europe. This could also inspire others.”

ICHOM was co-founded by Michael Porter, a Harvard economist, to develop standard sets of patient outcomes which could be used by healthcare providers around the world. Advocates say this can improve results and reduce variation between hospitals and health systems.

The best examples of the use of standard sets are in planned surgical procedures, such as prostate cancer  and hysterectomy. Dutch hospitals are paid more for heart operations when they get it right first time and Wales is exploring how to use outcomes data when buying lenses implanted by cataract surgeons.

In Europe and the US, some companies and payers are experimenting with reimbursement contracts that pay for drugs based on results. If a drug fails to deliver, the payer recoups some of the costs; if patients do well, the manufacturer receives the full price.