Clinical and Medical Research
Designing for Success
A Multi-stakeholder Approach to Clinical Development to Optimise Patient Access Developing new drug therapy is complex, taking many years to move from proof-of-concept to final data read out from the Phase III pivotal trial. After years of substantial investment, the end goal is to bring the therapy to patients in need. A significant number of pharmaceutical or biotech drug development companies are based in the USA, which is often the first market priority for commercialisation. Where companies are not commercially present in Europe, they have much to gain from a successful market entry into Europe, considering it generates the second largest global pharmaceutical sales revenue (approx. 20%) after the USA and Canada, which account for approx. 50%1. The European Union offers a certain degree of harmonisation; however, only with regard to the coordination of drug approval. When a drug is approved, it is valid for all countries within the European Union. Europe is a diverse and complex marketplace made up of more than forty distinct country-centred markets. Beside the more obvious differences in languages, each have their own specific methods to evaluate new drugs entering the market, and have differences in supply chains, patient demand, healthcare system setup and medical standards. Given these differences, bringing a new therapy to market in Europe takes time. According to the latest European Federation of Pharmaceutical Industries and Associations (EFPIA) data (Figure 1), the average time between marketing authorisation and patient access for a new drug varies from 119 days up to 925
days across Europe. Where a company has developed an orphan drug for a rare disease, time to market increases, and the average time between marketing authorisation and patient access increases, varying from 113 days up to 1141 days across Europe. In order to ensure patients in need can access new therapies as quickly as possible, it is critical that a multi-stakeholder approach is taken early on in the drug development process to maximise long-term success. Designing for Success - A Multi-stakeholder Approach to Clinical Development Planning for successful patient access for new drug therapies must start early in the life-cycle of the drug, right at the time of clinical development. Traditionally, drug development has operated in a siloed approach, whereby functions that are critical to the drugs success post-Phase III are missed from early consultations with research and development teams. Over the years, many companies have adopted a multi-stakeholder approach to drug development, involving other critical functions, such as regulatory, medical, commercial and patient access, and importantly, patient groups, early in the development process. For a new drug therapy to be commercially successful, it must satisfy the three critical external stakeholder needs; physicians want to prescribe it, patients see the benefits and individual market budget-holders are willing to pay for it. Research and development teams will engage with relevant clinical experts in the field to provide input into the acceptable
Figure 1: The average time between drug marketing authorisation and patient access.2 42 INTERNATIONAL PHARMACEUTICAL INDUSTRY
minimum efficacy outcomes, trial design and likely best patients to include into the trial. However, all other functions should be consulted and involved thought the whole development programme. Commercial and Medical Input and Opportunities Commercial and medical aspects can easily be missed at an early stage of drug development and can have severe consequences in the future. For example, working closely with the regulatory team to have a clear idea on the future label is critical. Small wording differences in the label such as “and”, “or” or “with” can have a significant impact on future positioning and promotion for commercial field force teams. Understanding the patient pathway and how physcians would intend to use and prescribe a new product is often a key medical input. However, understanding how the new drug may impact patients, and how they will use the product, should not be overlooked and should also be considered in early planning. Receiving input from patients early on can help inform R&D teams on aspects that may not have been considered before. For example, considerations regarding packaging, such as size and shape, especially if the drug requires cold storage in the patient’s home refrigerator, considerations to the type and size of syringe used, items included in ancillary sets or size of tablets and frequency of dosing. Patient Access Input and Engagement Opportunities Patient access (market access) input from an early stage is often missed. In cases where new drugs are developed in companies solely focusing on R&D, input is often overlooked and may not be considered until after Phase III initiation. Often the focus is just on pricing and reimbursement and the likely “achievable” price. However, patient access considerations should not just be focused on price, but also consider appropriate and relevant trial endpoints and evidence requirements from a budgetholder's perseptive that will allow them to clearly understand the value demonstrated by the new therapy. Winter 2020 Volume 12 Issue 4
