Access role

Introduction

Before discussing the access role itself, those less familiar with the function may wish to consider the terminology used in naming the activity, as well as how access differs from the regulatory affairs role.

A wide range of terms to describe the role

Over the years the role has borne a range of names, with every manufacturer seemingly using a different term. 'Reimbursement and pricing' was popular 20 years ago, while later terms such as 'value demonstration' emphasise the importance of communicating clinical and economic value. Also widely used is the concept of 'access', addressing the function's role in obtaining a geographic approval for use of a new treatment.

'Market access' acknowledges the necessity of obtaining an approval to sell the new treatment to the healthcare purchaser before it can be used to treat patients. More recently the central importance of the patient has encouraged use of the term 'patient access', although sensitivities of regions where regulations maintain a greater distance between manufacturers and patients have prevented universal use. 'Managed access' is a role most often carried out in the US, denoting responsibility for those parts of healthcare under greater governmental control.

Recognising the associations of these various modifiers, many use the unmodified 'access' term. This is the terminology that we have adopted.

Access is distinct from regulation

Those who don't work in the industry often refer to access activities as 'regulation', whereas within the industry 'regulation' refers to the activities of those bodies who grant the marketing authorisation following an assessment of the safety and efficacy of the medicine, with their function almost universally termed 'regulatory affairs'. 'Regulators' include the European Medicines Agency (EMA), the US Food and Drug Authority (FDA), the UK Medicines and Healthcare products Regulatory Agency (MHRA) etc.

The access role addresses the next stage in launching a new treatment and involves working with bodies that assess new treatments and decide whether they should be made available to a particular patient population for whom they are responsible, and at what price. Such bodies include the UK's National Institute for health and Care Excellence (NICE), or the German Gemeinsamer Bundesausschuss, or Federal Joint Committee of public health agencies, that advises on the benefit provided to patients by the new treatment.

What the access role involves

The access professional needs to demonstrate to the providers and payers the clinical benefit of the new treatment for the targeted patient group compared with the treatment options currently in use, and to locate it in the appropriate position in the therapeutic pathway to identify whether, and how, adoption of the new treatment will affect the delivery of care. It is also often necessary to address the economic impact of the new treatment, demonstrating the cost effectiveness of the treatment and assessing its impact on the healthcare and pharmaceutical budget.

Understanding what data the decision makers will accept and how and when they would like it presented is a core capability of the access professional, allowing the developer to incorporate these requirements into their evidence generation plans. The access role requires a good understanding of clinical evidence development, including trial methodology, real world evidence development, and derivative methods such as indirect treatment comparisons. Many decision makers need economic data and, while developing the models themselves is the responsibility of the specialist health economic role, the access professional needs to understand the methodology sufficiently well to be able to discuss requirements with the decision makers and guide the economic evidence development.

Addressing early access opportunities

When a treatment addresses a high unmet medical need, the access professional needs to understand what options are available to ensure that the therapy can be made available for patient treatment as soon as possible, while striking an appropriate balance with the need to demonstrate safety. It is on these occasions that a particularly good coordination is required between the access and regulatory teams to address the combined needs of the regulator and the payer.

A central role in pricing

In addition to these evidence generation and synthesis competencies, and interlinked with economic evidence development, the access role requires a good understanding of strategic and operational pricing. The access professional needs to understand how official and commercial prices are set within a country and more locally, how these prices affect what other countries are prepared to pay through international reference pricing, and how they are revised. A solid understanding of procurement practices, including tendering and negotiations, is needed in order to support local commercial staff.

The strategic and policy role

With increasing experience the access professional is well placed to develop competencies within the access function itself, to foster collaboration across functions within the company and to play a role in the development of corporate strategy. Externally this experience is valuable in working with policy developers, both independently and as a member of industry associations.