Category: Blog

Piggyback clinical studies for medical device and pharma companies

Piggyback clinical studies are a clever way to gather and analyze a wide range of data. They let us collect health economic data alongside clinical data, all at the same time. These studies have their own set of advantages and challenges, but they can be especially helpful for start-ups and companies with products that are still in the pre-market stage.

What are “piggyback” clinical studies?

Piggyback clinical trials collect health economic data alongside clinical data. These studies can offer several benefits to pharma and medical device companies, including:

Cost-effectiveness

These trials can be cost-effective by enabling collection of health economic data using the same trial infrastructure and resources that are already in place for the clinical trial. 

In addition, because the data is collected at the same time, the sponsors can gain real-time insights into the economic impact of the intervention under study. The cost-effectiveness data can be analysed and used to inform decision-making during the trial itself or shortly after its completion.

Timeliness

By collecting health economic data alongside clinical data, piggyback trials can ensure that the economic data is directly relevant to the specific patient population and intervention being studied. 

This can enhance the accuracy and relevance of the economic data, as it is collected in the same context as the clinical data, minimising potential sources of bias or confounding.

Enhanced data quality & integrated analysis

Piggyback trials make it possible to combine clinical and health economic data in one analysis. This approach gives us a more complete and overall understanding of the intervention’s value.

By using integrated analysis, we can better evaluate the intervention’s cost-effectiveness, budget impact, and other economic outcomes. This method considers both clinical and economic factors, resulting in a more thorough assessment.

The 4 main challenges of piggyback clinical studies

Designing piggyback clinical studies can be challenging due to the need to balance the collection of clinical and health economic data, ensure patient safety, and maximise efficiency.

  1. Because clinical trials are often artificial in nature – not reflective of real-world patient populations or patient treatment pathways – it is important to address areas of bias and risks to generalisability. For example, economic data may lack statistical power because the number of patients included in the trial is dependent mainly on clinical outcomes.

  2. In order to collect relevant economic and quality-of-life data during the clinical study, it is important to consider not only the study end points, but also time points for collection of the data.

  3. The variability in outcomes between patients and settings can be a significant challenge for conducting economic evaluations. The ability to extrapolate findings from a clinical trial to other populations or settings can be limited.

  4. Cost data collection: Collecting accurate cost data can be challenging, as it requires detailed information on resource use, prices, and other cost-related factors. This information can be difficult to obtain, particularly in low-resource settings or for interventions that involve multiple providers or institutions.

The benefits for medical device and pharma companies

Accelerated innovation

Piggyback clinical studies provide a means to gather comprehensive data and insights early in the product development cycle, helping to accelerate innovation and bring new products to market faster.

Cost savings

Pharma and medical device companies can achieve significant cost savings by collecting multiple types of data at once, rather than running studies separately.

Maximised success

These studies can provide valuable insights into the clinical and economic impact of new products and therapies, enabling companies to optimise their approaches and maximise their chances of success.

The combined data can help with marketing efforts by providing a more comprehensive picture of a treatment’s value proposition.

Faster reimbursement

Reimbursement applications and Health Technology Assessments (HTA) require data not only on effectiveness of new medical treatments and technologies, but also the cost-effectiveness. Companies must be able to justify the cost of their products and ideally, demonstrate value for money compared to competitors.

Collecting cost-effectiveness data early, during the clinical trial stage, can speed a company’s efforts to get reimbursement for their product. This can be a particularly important step for medical device start-ups and companies seeking DiGA status in Germany.

Piggyback Clinical Studies: Real-World Examples

Here are some recent examples of clinical studies, where health economic data was collected during the clinical trial.

Cost-effectiveness data were collected alongside a multicenter RCT on laparoscopic vs open gastrectomy for resectable gastric adenocarcinoma in 10 Dutch tertiary referral centers.

van der Veen et al. (2023)

An economic evaluation was conducted alongside a multicenter RCT at six medical centers performing corneal transplantation surgery in the Netherlands.

Simons et al. (2023)
Trial summary

A single-blind two-arm 1:1 RCT to investigate both the effectiveness and cost-effectiveness of a blended physiotherapy program for ST, including a smartphone application designed to increase exercise therapy compliance and provide tinnitus counselling.

Demoan et al. (2022)
Trial summary

An RCT assessing the clinical and cost-effectiveness of the Journeying through Dementia (JtD) intervention compared to usual care.

Wright et al. (2018)
Trial summary

An RCT designed to investigate the cost-effectiveness of exercise therapy in conjunction with the general practitioner’s (GP) care, compared to GP care alone, for patients with hip OA.

van Es et al. (2011)
Trial summary

“... it is important that the economic investigator contributes to the design of the study to ensure that the trial will provide the data necessary for a high-quality economic evaluation.”
Ramsey et al. (2015)

Best practices for designing piggyback clinical trials

Identify appropriate endpoints

When designing piggyback clinical studies, it is important to select endpoints that are relevant to both clinical and health economic outcomes, and align with regulatory requirements.

Maximise patient safety

Ensuring patient safety should always be a top priority when conducting piggyback clinical studies. This includes careful consideration of study design, informed consent, and data collection procedures.

Include experienced health economists

The inclusion of experienced health economists is critical to the success of piggyback clinical studies. Health economists can help to design studies that balance clinical and economic objectives and develop methodologies for data analysis.

In summary

Piggyback clinical studies offer an innovative approach to comprehensive data analysis, allowing for the collection of both clinical and health economic data in parallel. These studies offer a range of advantages for start-ups and companies, but also pose challenges in study design and implementation. Careful planning, attention to detail, and early collaboration between clinical and economic researchers is crucial. Addressing these challenges can help ensure that economic evaluations provide meaningful insights into the costs and benefits of healthcare interventions.

Interested? Contact us and we will gladly tell you more.

Further information:

Erkki Soini
CEO, Health economist

erkki.soini(at)esior.fi

Market Access and price and reimbursement application

In general, ’Market Access’ can refer to a product’s access to the market. In the health sector, however, Market Access usually refers to bringing and keeping medicines, medical products or devices on the market and available for patients. Market Access in its entirety is vast and includes many aspects, such as whether the product is intended for outpatient or inpatient care. In the Market Access process for outpatient products, one of the most central tasks is to determine a reasonable wholesale price and to apply for reimbursement status. Market Access procedures for inpatient products, on the other hand, may require public tendering and possible risk-sharing agreements. Here, we focus on the core aspect of Market Access for outpatient products: the price and reimbursement application.

Price and reimbursement applications require a variety of information

The sale and reimbursement of outpatient medicinal products are strictly regulated. Medicines require marketing authorisations (MA) in order to be sold. For this, a manufacturer must demonstrate that a product is efficient, safe, and of acceptable quality.  To bring the medicine within reach of most consumers, the marketing authorisation is not enough.  With an MA alone, consumers must pay the entire cost of the medicine. For wider access, most products also need to have a reimbursement status.

In Finland, the Pharmaceuticals Pricing Board (PPB) is the authority that makes reimbursement decisions. The PPB objectively determines the price level at which the medicinal product can be included in the reimbursement system with public funding. Preparation of the price and reimbursement application requires comprehensive information about the product: its therapeutic value, position in the national clinical practice, and its economic impacts. When a new active substance or a significant extension of the reimbursement status is concerned, the application must be accompanied by a health economic assessment. 

A high-quality price and reimbursement application demonstrates value

The price and reimbursement application addresses the product’s impacts on treatment, the state budget, and the market. These aspects are meaningful for both individuals and society; the reimbursement decision can affect tens of thousands, even hundreds of thousands of people in Finland alone. Therefore, the application must demonstrate the value for patients, health, and society. The evidence is compiled from numerous sources – national registers, clinical data, published data, and other sources of real-world data. It is analysed, studied, modelled, and interpreted.

There are important benefits to a successful application process: patients have access to medicinal products with demonstrated cost-effectiveness and positive societal impacts. This access to effective medications can have an enormous positive effect on patients’ quality of life. At ESiOR, this motivates us to do better every day.

ESiOR on your Team

Are you looking for expert assistance in navigating the complex world of market access and pricing for your healthcare product? At ESiOR, our team of seasoned professionals have a proven track record of successfully navigating the market access landscape and securing favorable pricing and reimbursement for our clients. With extensive experience in a wide range of therapeutic areas, ESiOR is your go-to partner for achieving commercial success in the healthcare sector.

Interested? Contact us and we will gladly tell you more.

Further information:
Erkki Soini CEO, Managing Partner

erkki.soini(at)esior.fi

Why do we need Health Economics?

Let us cut to the chase. Health is important. It is without doubt one of people’s core values regardless of nationality or culture and a key driver of wellbeing. On the other hand, money and funding are important, as well, whether we like it or not. You cannot buy love and you cannot buy health, but nevertheless health and money are connected. But let’s leave the connection between money and love aside!

Unfortunately, modern medicine may not be able to work wonders, but together with a healthy lifestyle, it can help us live longer and stay healthier. Of course, medical treatment and health interventions come with a price.

What is cost-effectiveness research?

We are one step closer to the point now. Resources are scarce and limited, and we do not have endless pockets of money. So, we need to choose. How do we decide?

Consider you need to buy a car. What do you do? You list the possible options, hopefully keeping your budget in mind. You compare the options against each other – which car would benefit you the most and offer most value for money? In other words, you would do appraisals, research, and comparisons on different qualities of the cars. You would also consider how your purchase and its cost would impact you later. What would you have to give up choosing a specific car? How expensive would it be in the long run? How would it benefit you later?

That’s a typical predictive comparison. Now, what does your car have to do with health economics? If you think of the process, it resembles our work quite a lot. Instead of cars we compare health-related things: different treatment options, health technology, and medicinal products, for instance. We are specialists of cost-effectiveness research and produce predictive assessments of health investments and costs, and benefits.

Cost-effectiveness is key

It is crucial we apply our best knowledge into social and health economics outcomes research. The costs of healthcare are rising to a level we cannot afford. The trend is both national and global, and recent incidents such as COVID-19 are likely to catalyse the challenges with health and social care costs.

At the same time, we have treatments and practices that are not effective – they may not be cost-effective even at a zero price. Therefore, it is essential these existing treatments are also assessed carefully, so that we will be able to allocate our national or local healthcare resources in a cost-effective manner. Real-world data is needed for the task, which we can turn into real-world evidence.

To answer our title question: health economics is important because we cannot afford not to utilise its methods to assess cost-effectiveness. Cost-effectiveness is a way to produce health efficiently, and as much as possible. So, a lot is at stake. Our health.

Interested? Contact us and we will gladly tell you more.

Further information:
Erkki Soini, CEO and Health economist

erkki.soini(at)esior.fi