Pre-approval access (early access, expanded access, named patient supply etc.) is a hot topic at present, especially with the high-profile discussions around Right-to-Try which are making the headlines around the world. Regardless of the merits of Right-to-Try, preapproval access to medicines is high on the agenda for many pharmaceutical and biotech companies.
Successful pre-approval access for biotech companies can be a game-changer. There are several crucial advantages for biotech’s who embark on pre-approval access in Europe. Firstly, it is possible in most EU countries to open a pre-approval access program whilst the product is in PII. Crucially, in most countries it is also possible to charge a commercial price for your product. Such programs also provide the opportunity to capture clinical outcomes data, to develop relationships with physicians, and to demonstrate demand for, and efficacy of, your product in a real-world patient population. Furthermore, an EU access program can continue indefinitely if you have no plans to commercialize outside of the US.
There are many examples of companies securing long-term benefits through the strategic use of a pre-approval access program. The opportunity to generate some early revenues, the implications of generating outcomes data from European patients, and the benefits of a positive physician experience of your product ahead of commercial availability are only a few of the many potential benefits of allowing pre-approval access in Europe, whilst you continue on your clinical development pathway.
Whilst at first glance the regulatory landscape in Europe can look daunting, it is relatively straightforward to navigate. All EU countries have adapted overarching EU legislation on pre-approval access to fit within the specific environment of their own healthcare systems, with the intention of making it as easy as possible for companies to provide access to patients in need.
The key thing is that you are in control. The regulatory environment allows you to open a preapproval access program in one, many or all EU countries. You decide what approach suits your organization best. It is possible to ‘test the waters’ by opening a program in a selected few countries initially and expand to others as demand dictates. You have full control over which patients and physicians you allow on to any program, and unlike the US EAP system, there is no requirement for monitoring, or for a protocol (with the exception of a few countries) to be developed. It is also possible to control the numbers of patients entering a program, until you have a more detailed understanding of demand.
Partnering with an experienced vendor relieves you of the burden of the day-to-day challenges of any pre-approval access program and allows you to focus on your key objectives of clinical and commercial development.
However, before embarking on an EU access program, it makes sense to validate in advance the pros and cons of such an approach, as a number of product- and disease-specific considerations can inform the go or no-go decision.
In order to understand whether it is feasible, or practical, to put in place a pre-approval access program a detailed feasibility analysis is recommended. A product-specific, per country feasibility analysis will allow you to understand the disease-specific and regulatory specific issues in countries of interest, allowing you to make informed decisions regarding whether or not to implement a pre-approval access program in Europe. The more detailed the up-front understanding, the better your decision-making.
Examples of the topics which should be considered in a pre-approval access feasibility analysis include:
- In what countries can we open a pre-approval access program at PII/PIII
- Does the data (efficacy and safety) support the commencement of a pre-approval access program?
- What existing therapies are available in different EU countries for our indication?
- Where can we charge for our product at PII/PIII?
- What is the awareness/demand for our product?
- What are the implications for supply chain?
- What is the existing treatment landscape?
- What competitor clinical trials and development strategies do we need to take into account?
- What are our peer-group companies doing with regard to pre-approval access?
- What is the environment for clinical data capture?
- Are there any existing data sets in any country that could be used as historical controls for our pre-approval access program data?
Regardless of what happens with Right-to-Try, the environment in Europe for pre-approval access is well developed, clearly structured and well controlled for those companies who wish to make their innovative assets available to patients early.