Co-authored by Olasile Abolade, Associate Consultant
Pricing pressure and limited health care budgets have sparked interest in innovative contracting. Outcome-based contracts are innovative contracting plans used to link the outcome or health benefits of a drug to its price or overall cost of treatment. These contracts represent a parting from the traditional “fixed cost” reimbursements and are implemented in order to better manage the risks around public coverage of drugs whose clinical effectiveness and the effects of budgets are uncertain.
There are several challenges for pharma and MedTech companies, health authorities, payers, and pharmacies regarding pricing and reimbursement of pharmaceuticals/medical devices. For payers, the real question is – how can I pay a fair price for a new drug (or device) when under a cloud of uncertainty related to cost-effectiveness, safety profiles, long term and comparative effectiveness? Outcome-based contracts, sometimes referred to as value-based contracts, are mechanisms for tackling these issues.
They are the most transparent, reproducible and data driven models. So far in Europe, Italy is leading the implementation of an outcome-based model. To facilitate the implementation of outcome-based contracts in the country, patient (or drug monitoring) registries are used for evidence collection and tracking of health-related outcomes. A patient registry is a collection of standardized data about a population defined by a particular disease or condition and that serves one or more predetermined purposes. Patient registries are disease (or condition) based and are therefore applicable to biotech and MedTech companies. While there are similarities between drug and medical device registries, there is also some differences to be aware of related to registry design, data collection and analysis, including information on a device’s performance.
Why patient registries matter?
We can start by asking how many registries are out there? It is rather difficult to put an official count on the number of registries. But, in the rare disease category, for example, there are significant numbers, and the most represented countries are France, Germany, Italy, Spain and the United Kingdom.
Reviewing an inventory of registries compiled by the European Medicines Agency (EMA), one can see that a significant proportion of them are patient registries owned by or affiliated with government or non-profit organizations (NPOs).
Source: The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP)
When properly designed and implemented (either by government or private entities), patient registries should provide a real-world view of its clinical practice, patient outcomes, safety and long-term effectiveness of medicine. The registries track patient eligibility, evaluate utilization in clinical practice, collect epidemiological and safety profiles, including data that may be missing at market authorization.
In order for a patient registry to fully serve its purpose, there has to be a concerted effort from all participants to effectively collaborate on establishing, use, and management of the registry. This calls for alignment in several areas including data structure, scientific method, data sharing and privacy, and harmonized protocols.
The single most important key to a successful registry is planning. Planning includes defining what type of registry will be developed, following appropriate guidance, understanding how the data will be used once it’s collected, and developing a strategy that involves all relevant stakeholders.
The Italian Registry Experience
The success and prevalence of outcomes-based contracts in Italy comes down to the use of patient registries in implementing these procedures. The Italian Medicines Agency (Agenzia Italiana del Farmaco – AIFA), a national body responsible for pharmaceutical regulation and health technology assessment, owns and maintains a system of drug-product registries. How is the system successfully created and managed by AIFA?
The first factor to consider is the national legislation (Law no. 135/2012 and Law Decree 19/06/2015) passed in Italy that ensures country-wide use by different participants and the application of Managed Entry Agreements (MEAs) to drugs monitored by the system. The law enforces mandatory data collection requirements by various participants (e.g., physicians, pharmacists) and ownership of data is the responsibility of AIFA, although maintenance costs are shared with market authorization holders. There are other aspects to consider that have led to the success of this system, including:
- Digitalization: Integrating the AIFA registries with the Italian National Health Service IT system enables all activities from data collection to aggregation, and report generation to a single unified electronic platform. Digitalization also enhances information exchange and simplifies the tracking of analysis outcomes related to clinical and economic assessment of innovative drugs.
- Incentives to collaboration: Cross-functional use of registry data serves many purposes, such as drug regulation, public health, research, and health technology assessments. Effective collaboration between stakeholders at the regional and national level has improved data integration and quality. As a result, there are benefits for different participants such as patients (safe and timely access to innovative therapies), pharma (or MedTech) companies (access to relevant data such as pharmacovigilance, clinical, etc.) and health authorities (balance between costs and efficacy).
- Governance: A governing principle is defined and established. This is supported by various registry teams which include, at a minimum, project management, the scientific committee, legal (or data privacy) and quality assurance teams. Their roles, among others, include ensuring that the registry meets its targeted objectives in addition to overseeing that data access and privacy policies are strictly observed in accordance with local or international regulations.
Ultimately, outcome-based contracting has to be more value driven in order to be attractive to pharma (or MedTech) companies. This starts with the review of existing health care policies that could act as a barrier to open communication and then an effort by both pharma (or MedTech) companies and health authorities regarding the creation, use and maintenance of registries.