Real-world data can help pharma companies to capture clinical and economic outcomes to bridge the gap between the development of medicines and what happens in real life clinical practice. Gathering the right type of data at the right time and using it in the right way can benefit patients as well as help pharmaceutical companies demonstrate the value of their products to healthcare providers, commissioners and payers.
Randomised controlled trials (RCT) will always play a key role in the process of establishing a drug's benefit-to-risk profile and bringing it to the market.
However, even the largest RCT cannot give a full and accurate picture of how that medication will be used and the outcomes that it will achieve when it reaches everyday practice. In recent years there has been growing recognition of the part that real-world data can play in filling these knowledge gaps and reducing the uncertainty that exists when a new medicine is launched. With pressure to bring drug costs down as national healthcare budgets are increasingly squeezed, more attention is turning towards how real-world data can help to demonstrate the value of a medicine.
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Real World Data Task Force has defined real-world data as 'data used for decision-making that are not collected in randomised controlled trials'. Through necessity, this is a broad definition that encompasses everything from real-world studies and registries to health surveys and the use of administrative data and medical chart reviews. The type of data sought and how it is collected will be driven by the nature of the disease and the drug, as well as the way in which the data will be used.
Integrated patient support services, such as homecare services, telephonic counselling, and convenient medication delivery services, provide an array of benefits to patients, as well as offering key points of interaction at which real-world data can be gathered. Integrated patient support services provide an ideal opportunity to capture real-world data in the patient's real life setting.
There are clear benefits to providing patients with supportive care services and gathering actual real-world data to demonstrate the value of a medication. These benefits include close monitoring of patient adherence, building up information about outcomes, ensuring the patient is gaining maximum benefit from the prescribed therapy and increasing real-world knowledge about the efficacy and safety of the medicine.
Consider adherence data as an example: one of the key gaps between clinical trial and clinical practice is patient compliance. Measurement and monitoring of medication usage in the real world enhances the patient feedback loop to care providers and optimises the value of therapy. In a nutshell, it can vastly improve the understanding of that medicine's value and enable medical teams to address gaps in patient care to ensure optimal outcomes.
Of course, gathering the data is only one half of the story and what is then done with that data is of critical importance. Four key uses are likely to be top-of-mind for any pharma team tasked with developing a real-world data strategy:
1. Better understanding of the effects of medications in a real-world setting
In a 2011 White Paper, the Association of the British Pharmaceutical Industry (ABPI) stated that: 'non-interventional real-world (RW) data cannot replace the safety and efficacy data generated by RCTs but can help to support those data by allowing actual versus expected efficacy and safety to be evaluated in the context of a real clinical setting. Hence RW data can reduce the uncertainty surrounding a new medicine at the time of launch by improving the information on benefits and risks and developing evidence on real-life effectiveness.'
Removing this uncertainty can be as beneficial to the drug's manufacturer as it is to prescribers and patients. Real-world data can, for example, demonstrate how a medication works in populations that were not studied in pre-approval RCTs, or under conditions that were not studied. It may also be able to show how one medication performs relative to another where head-to-head study data is not available. As well as providing confirmation of a medication's safety and efficacy, this type of data can give pharma marketers a better understanding of the patients in which a treatment delivers the greatest benefit. This insight can be used to hone and strengthen the marketing strategies as well as to provide further evidence of value.
2. Develop health economic data to defend price and secure market access
According to ISPOR, real-world data is essential for sound coverage and reimbursement decisions. With many countries adopting value-based pricing and requesting that pharma companies demonstrate the value of their medicines, generating data that can be used to defend pricing and justify a medicine's place in a competitive and cost-conscious market will become imperative. A major benefit of using real-world data to generate health economics data is the fact that it has the potential to enable payers and other decision makers to understand the economic impact of a medicine in a specific country, region or even hospital.
3. Understand and demonstrate the real economic value of a medicine to payers
Another factor that is likely to elevate real-world data on the pharma agenda is the 2010 pharmacovigilance legislation set to come into force in Europe later this year. This legislation contains a new obligation for companies to perform post-authorisation efficacy studies in certain circumstances. Furthermore, there is an anticipated move towards periodic benefit-risk assessment. These developments will herald an era in which continuous assessment of risks and benefits is required to defend a medication's place in the market and to communicate its value. In this scenario real-world data will not only be valuable; it will be essential.
4. Deliver smarter, more targeted communications to patients, payers and HCPs
Knowing more about the patients who are using a medicine helps to refine customer communications and ensure that the right messages reach the right healthcare providers (HCP), particularly GPs who play a key role in delivering patient care. There is another important benefit here too: it is increasingly common for pharma companies to develop information and services to support patients taking a particular drug.
If they can identify and understand the challenges that patients experience when they use the medicine, targeted communications can be developed to help patients to manage those challenges. The net result is a win-win situation for all parties: the patient gains the best possible chance of deriving maximum benefit from the therapy, the HCP is able to receive more comprehensive information and the pharma company can show that the medicine is providing maximum value.
Several factors in the European healthcare environment are increasing the emphasis on real-world data and its weight in the process of determining the safety, efficacy and value of medicines. Positively embracing this change will help pharma companies increase their understanding of their medications and, ultimately, to demonstrate the value that they bring to patients, payers and HCPs.
Dawn Sherman is chief operating officer at Medco International