Europe benefits from 50 years of pharma legislation, but it needs to evolve if we are to remain competitive
19.03.15
This is a painful portrayal of the Europe of yesteryear, a Europe without a cohesive form of legislation in the pharmaceutical sector. Half a century after the introduction of a more harmonised legislative framework, though, the picture is much improved.
It is hard to fully do justice to the benefits brought by EU medicines legislation over 50 years, but there are some significant points – inexhaustive though the list might be – worth mentioning.
A key element that has helped the European member states drive forward a more equitable provision of care with medicines has been the European Medicine Agency. Since 1995, the EMA’s main remit has been to assess medicines to rigorous scientific standards and provide partners and stakeholders with independent, science-based information on medicines.
Prior to its establishment, it took between seven months in some EU member states and up to six years in others to get marketing approvals for new products. Through the EMA, the approval process has been shortened considerably, and marketing approval can now, in general, be expected in less than a year. Similar progress has also been made in the mutual recognition and decentralised procedures.
Beyond the regulatory process, the European legislation has also sought to improve pricing and reimbursement processes to bring medicines faster to market for the benefit of patients. The Transparency Directive is an important tool to improve the openness, and therein the objectivity, of member states’ systems in this area.
It offers an additional legal basis to challenge a number of discriminatory and/or arbitrary practices, in the area of pricing and reimbursement of medicines. It sets time limits for the pricing and reimbursement decision-making process in Member States’ legislation. It requires Member States to base their pricing and reimbursement decisions on objective and verifiable criteria, to publish them and to provide proper justification for them. Finally, it requires Member States to set up appropriate appeal mechanisms and to inform the applicant of the remedies available to him.
The legislation has also benefited areas that to a large extent were overlooked. The Orphan Drug Regulation, for example, has had a significant positive impact. Drug approvals in this area had been limited, but since it came into force in 2000, over 100 new treatments for patients with rare diseases have been approved at European level. It is clear that the EU regulatory framework for orphan medicinal products provides clarity, predictability, while promoting innovation.
Similarly, the Paediatric Regulation, which came into force in January 2007 has facilitating the development and availability of medicines for children, ensuring that they are of high quality, ethically researched and authorised appropriately. It has to a large extent succeeded in achieving its aims without subjecting children to unnecessary trials or delaying the authorisation of medicines for use in adults.
So, over the last 50 years, laws and regulations governing medicinal products have evolved and, accordingly, the pharmaceutical industry has undergone a progressive globalisation process in the development, manufacture, marketing, and distribution of medicinal products. Nevertheless, a life sciences strategy is needed to ensure genuine coherence between research, health, and economic and fiscal policies.
Each aspect of a medicine’s journey, from discovery through development and beyond, to patient access, is intrinsically linked. Policy measures taken in the various areas of this journey need to be complementary and mutually reinforcing.
Bear in mind that Europe needs to compete on a global scale for attracting and retaining pharmaceutical R&D. A common effort between member states and the EU should strengthen this leadership and make Europe the best place to discover, develop and bring to patients medicines and medical technologies.
There is an increasing need to change the approval paradigm, moving away from the binary “yes/no” choice towards a process in which knowledge is collected and collated in routine care – so-called ‘real-world evidence’. This calls for a scenario in which silo-working is sidelined and industry, patients, health authorities, reimbursement bodies and prescribing physicians join together in speeding a drug from development to patient access.
What is required along this journey is a careful balancing of meaningful evidence of effectiveness at various stages with the need for safety. Ultimately there also need to be a weighing of innovation against affordability.
It is this very innovation in therapy that will provide major health benefits to patients and, indeed, to national health services. However, in order to encourage and safeguard innovation, the European regulatory system and therefore the legislative framework, will have to adapt to advances in science. The need to update is urgent if we are to remain competitive in the evolving global regulatory environment.
The EMA has already embarked upon a promising initiative to address this issue. Its adaptive pathway pilot project involving real medicines in development is a bold step towards improving the way innovative and needed new therapies reach patients. The ultimate aim is to achieve timely access for patients to treatments that promise to address serious conditions where there is an unmet medical need, especially when there are no satisfactory alternative therapies.
Within a “safe-harbour” environment, the pilot provides a framework for informal interactions between medicines manufacturers, regulators, patients and health technology bodies on ‘live assets’, that is medicines currently under development.
Industry itself is helping to propel MAPPs forward, within the framework of a public-private partnership, the Innovative Medicines Initiative. It envisages the creation of a platform to coordinate activities in this area and will be set up a co-ordination group in 2015 to evaluate requirements, to steer research and ensure comprehensive information gathering on MAPPS-related developments.
