NDA Group AB

Achieving Market Access in Europe

Regulatory agencies are no longer at the forefront of deciding if patients get access to new medicines. A multitude of national agencies and organisations have risen over the past years with the purpose of evaluating the value of new medicines.

These systems are now a fact in the EU where different organisations are making individual decisions on whether pharmaceutical products will be allowed to pass through the public health care system or not in the region where they are responsible. Today it is ultimately these bodies that decide if a product will reach the patient.

Uncertainty demands more intelligence sooner
What consequences does this have on drug development and how should we act to ensure we take these changes into account in the development process?

The development of new medicines is now regulated by requirements from regulatory authorities as well as from HTA (Health Technology Assessment) bodies. The shift to increased payer power and the increased rate of change on the requirements affecting drug development because of this shift are making the process increasingly unpredictable. Therefore, there is a need for sound intelligence on how to optimize the development program to satisfy both the regulatory and the HTA requirements. This intelligence is hard to build up and maintain within companies, particularly for smaller and mid-sized companies. Consequently, companies experience a great need to seek external advice from both HTA/regulatory agencies, in the form of formal scientific advice, as well as from third party providers specialized in this field like NDA.

The successful companies are gradually building the combined intelligence into their decision making process, integrating it as early as possible. This is done with the understanding that if it is left for too long, until after design of the phase II and III studies, it could lead to a total rejection from HTA bodies with the medicine standing without reimbursement or with a price set at the level of a generic.

Growing need to understand national systems
As part of this process, it is very important to account for the differences between the national systems in judging the value of a medicinal product and to understand the consequences of these differences in terms of access, reimbursement and pricing. The national systems are currently under rapid development with the introduction of new legislation and changing organisational responsibilities, exemplified by the increased importance of the German, French and British payer/HTA organisations. The development of a market access strategy must, therefore, be based on a careful analysis of the individual national systems.

One of the more critical decisions in the development of a new drug is the choice of the comparator product and the endpoints in the clinical trials. This is well established by the regulators, where practice and therapeutic guidelines give good guidance in the choice of the comparator and relevant endpoints.

In the field of HTA, however, very different considerations decide what comparators and end-points may be accepted. In several cases we have seen how the German AMNOG has concluded that the wrong comparator was chosen. In one case, the preference would have been a non-medicinal therapeutic intervention. The lack of clinically relevant endpoints is also put forward as an argument by payers when rejecting applications for drugs previously approved by the regulators.

Consequently, these aspects will be of crucial importance when designing a clinical development program.

Continually measuring the value of medicines
Even in cases where all of these aspects have been considered in the pre-authorization phase, it may be impossible to demonstrate the real value of a new medicine in connection with the marketing approval. In many cases, real value can only be demonstrated once the product has been introduced into routine clinical use.

It is obvious that the maturity, mandate and willingness among payers to understand and take this into consideration vary when judging the value of new medicines. Accordingly, the market access strategy must also consider how to continually measure the value of the medicine in clinical practice. Different cost-sharing schemes between payers and companies are becoming relatively well established. However, there is a need to develop more comprehensive follow-up systems, including using registries and drug monitoring schemes, to continually measure the value of the medicinal product.

With the introduction of the new EU pharmacovigilance legislation it is now critical to maintain the regulatory / HTA alignment throughout the post-authorisation phase of the product. This new legislation has introduced requirements for companies to follow-up the benefit/risk profile of their medicines in clinical practice, and the types of studies that will have to be performed will be similar to those requested by payers. Therefore, there is a need to coordinate the design of post–authorization studies to satisfy both the regulatory and payer requirements for data from the real life use of medicines.

New organizational models needed
This new development, strongly influenced by the payers, will have consequences on the type of competence needed for the pharmaceutical company to manage this, but will also put new demands on how the internal organisation and decision making is set up within companies. The organisational models valid before the rise of the strong payer influence will not be able to respond to this new situation. The new external environment must be reflected in the internal organisation of successful pharmaceutical companies. Companies need to consider changing the internal decision making in the drug development process – working to integrate aspects from the whole life cycle of the drug. This will require creating multidisciplinary teams that are involved in critical go-no-go decisions during the development process. Individual companies naturally have to find the organisation that best suits them but a common theme is that vertical silos must be broken down in order to make room for more horizontal, multidisciplinary decision making.

How will the HTA area develop?
It took nearly twenty years for the EU drug regulatory system to be harmonised and stabilised. The payer/HTA community is just beginning to start a process similar to that of the regulatory EU alignment. Positive initiatives have been taken and the EU commission has been active in promoting this development, and the formation of EUnetHTA is a very promising move. There are already several draft guidelines produced that could be of help, with one covering the critical selection of a relevant comparator. A pharmaceutical company must carefully follow the work of this group and its output as well as provide feedback on guidance for consultation.

Companies should take advantage of the scientific advice that the EMA, national HTA bodies and EuNetHTA offer, in order to, as far as possible, increase the predictability of the outcome for their products.

Important projects involving both regulators and payers are now emerging that focus on new ways to develop drugs. The EMA’s concept of adaptive licensing and several projects within IMI are good examples of where the regulatory and payer playfields are converging. It is only through increased interaction and dialogue between the different stake holders that common mind-sets and models can be developed. These initiatives are therefore very important in the future development of market access in Europe.


How can NDA help?
At NDA we have developed a unique capability to integrate the regulatory and HTA requirements in our consultancy service. Our strategy is to help companies to minimize the uncertainties in drug development in order to save time to market and waste in development cost.

We support companies by providing objective advice and hands-on support to understand the payer/HTA landscape and to create strategies and evidence generation schemes that will satisfy the needs of payers as well as regulators.

Through our Advisory Boards, comprised of some of Europe’s most experienced regulators and payer experts, we provide high level strategic advice, second opinions on internal perspectives and assurance of the internal decision making process. Combined with our consulting capabilities we then support any necessary changes or programs in order to meet the identified requirements.

To learn more about how NDA can support your products entry into the European market, visit our web site and contact one of our representatives.


NDA Group AB
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