Fostering a research eco-system that can deliver that innovation rather than undermining it through challenges to IP, is the best way to protect citizens across Europe and around the world.
Every new treatment or cure starts with the spark of an idea. Protecting that spark can involve ensuring funding for early-stage development of a new therapy, it can be creating the right environment for collaboration between research partners, it can be evolving the regulatory framework to keep pace with rapidly advancing science and protecting the spark means having a strong and effective intellectual property framework.
Pharmaceutical intellectual property (IP) – incentives and rewards are the foundation on which innovation is built: they encourage and protect innovation, driving research and development investments into areas of unmet medical need.
INTELLECTUAL PROPERTY DELIVERS ACCESS TO TODAY’S MEDICINES AND INVESTMENT INTO TOMORROW’S CURES
The framework provides companies researching and developing new medicines the certainty that if a medicine makes it to the market, it will be protected from unfair competition for a limited period time. This is what they need to invest in the long, complex, risky and costly process of delivering new medicines to patients, to healthcare systems and to society.
It helps companies turn ideas into assets that address unmet medical needs, improve the lives of patients and their families, create value and also jobs. This, in turn, attracts investment which helps to protect the company’s ideas, retain the knowhow required to convert ideas into therapies, and support further research into the next generation of treatments to improve people’s lives.
With over 7000 medicines in development, the system of basic & overarching or targeted incentives is working: it enables a pipeline of this scale despite the high risk of failure.
With the number of new medicines approved every year, the system is working: it enables a pipeline of currently over 7000 medicines in development despite the high risk of failure.
protects inventions for a limited term of 20 years
introduced to offset some of the effective patent term lost during the development of medicine due to increasing regulatory approval requirements
protects data relating to preclinical and clinical trials of a medicine
supports research into the next generation of treatments for rare disease.
stimulates the development of new medicines for children
BASIC AND OVERARCHING INCENTIVES
Patents are the basic incentive supporting research into new medicines. It was designed to provide a limited period of exclusivity to all inventors to commercialise their innovations irrespective of the field of technology, thereby encouraging investment into the progress of science and technology. Patents are only granted to inventions & creations that are new, involve an inventive step and that are capable of industrial application.
A patent grants the right to exclude others from commercially exploiting their invention for a limited term of 20 years from filing date – i.e., as soon as research shows potential. As a quid pro quo for exclusivity, the inventor has to disclose the details of the invention, thereby contributing to the dissemination of scientific and technical knowledge, thus helping accelerate follow-on innovation.
SUPPLEMENTARY PROTECTION CERTIFICATES (SPCS)
Unlike in most other industries, around half of the standard 20-year patent term in the pharmaceutical product is spent on rigorous clinical trials to demonstrate the safety and efficacy of the medicine before it can be made available to patients. Recognising this disadvantage, the EU introduced supplementary protection certificates (SPCs) to offset part of the lost patent term and ensure sufficient protection is available to continue encouraging sustainable and appropriate funding for the next wave of biopharmaceutical research and development. In practice, the SPC restores part of the patent protection at a maximum of 5 years (to be determined case by case). The combined/total patent and SPC protection period from marketing authorisation, however, cannot exceed 15 years.
REGULATORY DATA PROTECTION (RDP)
In order to obtain a marketing authorisation, pharmaceutical companies need to submit extensive data relating to preclinical and clinical trials to demonstrate the quality, efficacy and safety of the medicine to be approved. Regulatory data protection (RDP) protects innovative companies’ investment in generating this extensive body of data through a limited period of exclusivity on the data, starting from marketing authorisation.
After the RDP period, generics are permitted to rely upon the originator’s data to obtain approval for their products through an “abbreviated” application. Such time limited protection is crucial to incentivize the significant investment necessary to demonstrate the safety and efficacy of new medicines, while nevertheless enabling other companies to eventually register their products on the basis of this data.
Specifically, the European legislator designed RDP in the following way:
- 8 years of data exclusivity during which the marketing-authorisation holder benefits from the exclusive rights to the data. This means that a generic or biosimilar applicant cannot cross-refer to this data in support of its own marketing authorization. After this period, anyone can rely on the innovator’s data in the “abbreviated” application for a marketing authorization. This is followed by 2 years of market protection during which a generic or biosimilar cannot be placed on the market. A generic or biosimilar company, can however rely on the data body to prepare its own marketing authorisation dossier.
- 1 year of additional market protection may be available in a case of a new therapeutic indication which brings significant clinical benefit in comparison with existing therapies. As per above, a marketing authorisation dossier can be prepared but the generic or biosimilar product cannot be placed on the market.
Did you know? A patent protects inventions, RDP protects data
TARGETED INCENTIVES FOR RARE AND PAEDIATRIC DISEASESThe EU has further sought to encourage the research and development of safe medicines for people living with rare diseases and children.
To ensure that patients suffering from rare conditions have the same quality of treatment as any other patient in the EU, the EU Orphan Regulation was adopted in 2000. It introduced the definition of Orphan Medicinal Product (OMP), an orphan designation and a specific committee in charge of OMP at the EMA as well as a series of incentives to stimulate the development of treatments for rare diseases.
The Orphan Regulation is central to the EU’s strategy of promoting innovation that meets the needs of patients suffering from rare diseases. To qualify for orphan designation and thereby benefit from the framework of the Orphan Regulation, a medicine must meet a number of criteria:
- it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
- the prevalence of the condition in the EU must not be more than 5 in 10,000, OR it must be unlikely that without incentives marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
- no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.
Pharmaceutical companies investing in R&D for orphan medicines have to prove at two distinct points in time (at the time of application for orphan designation and at the time of marketing authorisation) that the medicine fulfils the strict requirements for obtaining an orphan designation.
Once a product has been approved for a specific indication, any new, additional indication or extension of the existing orphan indication, requires a separate assessment by the EMA and marketing authorisation decision by the European Commission (following the same strict criteria).
It is not a simple or quick process and in fact, only a few – around 11% – medicines have more than one orphan indication or treat a larger number of (rare disease) patients.1
The EU Orphan Regulation2 provides protocol assistance, reduced fees for regulatory activities, additional incentives for SMEs, and 10 years of market exclusivity. During that period, the European Medicines Agency (EMA) and the Member States shall not accept another application for an MA for a similar product. It is also worth noting that this exclusivity is not absolute as medicines showing clinical superiority can be approved and marketed during the 10-year period. As a result, there can be multiple treatment options for some rare diseases.
The introduction of the EU Orphan Regulation, along with scientific advances, led to a wave of new treatment options for rare disease patients. Since the adoption of the Orphan Medicinal Products regulation in 2000, more than 160 OMPs had been approved by the European Medicines Agency EMA by 2018.
Despite the considerable success so far, there is still high unmet medical need in rare diseases: only 5% of rare diseases are estimated to have an approved treatment and much work remains to be done. Maintaining the right regulatory, scientific and economic environment for orphan medicine development is therefore imperative if the public health challenge of rare diseases is to be met in future.
The Paediatric Regulation was adopted in 2007 to facilitate the development and availability of high-quality medicines suitable for children. The Regulation has contributed to make paediatric research an integral part of general medicines development, introducing obligations on pharmaceutical companies to research and develop their products in children as well as adults.
When a company completes a Paediatric Investigation Plan (PIP), the rewards can take two alternative forms:
- A 6-month extension to the supplementary protection certificate (SPC), which protects the product OR
- When the product which is the subject of the completed PIP is an orphan medicinal product, the 10 years of market exclusivity provided by the Orphan Regulation can be extended by a further 2 years.
Rewards are intended to support the industry by helping to offset the additional costs of conducting paediatric research. They are only available when a company has successfully completed its paediatric research as required by the EMA and updated the product labelling to reflect the results.3
The Regulation also provides for incentives for companies to develop off-patent compounds for paediatric indications, through the concept of Paediatric use marketing authorisations (PUMAs).4 A PUMA will only cover the paediatric indication(s) and will benefit from a separate 8+2 year period of data and market protection.
Rewards can be valuable but are far from certain to be achieved for all products for which PIPs are completed. According to the 2015 EMA report to the EC, out of the 113 PIPs completed, only 33 products had benefited from the 6-month SPC extension and 3 products obtained the 2-year orphan market exclusivity extension. Until July 2018; only 3 PUMAs have been authorized.
In the period 2007-2016, over 260 new medicines for use by children (new marketing authorisations and new indications) were authorised. Prior to the Regulation, 50% of medicines had not been tested and developed for children. In the past decade, the proportion of paediatric clinical trials has increased by 50% (from 8.25% to 12.4% of all trials conducted in the EU).5
- Future-proofing EU competitiveness by limiting the negative impact of the SPC manufacturing waiver get_app
- Review of CRA’s Report “Assessing the economic impacts of changing exemption provisions during patent and SPC protection in Europe" get_app
- Assessing the impact of proposals for a Supplementary Protection Certificate (SPC) Manufacturing Exemption in the EU get_app
- New indications & cross-label dispensing get_app
- Consultation on the respect of intellectual property in public procurement procedures get_app
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1 Dolon, data on file 2016
2 Regulation 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products https://eur-lex.europa.eu/LexUriServ/LexUriServ. do?uri=OJ:L:2000:018:0001:0005:en:PDF
3 Rewards are not available where a full waiver has been granted for the product in question nor when the paediatric investigation plan cannot be completed before expiry of the Supplementary Protection Certificate (SPC) term, or orphan market exclusivity period, as applicable.
4 A PUMA is granted to off-patent products which are usually already subject to generic competition. The 10-year period of data and market protection it provides is necessarily specific to the paediatric data/indication on which the PUMA is based, and therefore restrains generic applications for that particular indication and their reliance on that particular data only. In other words, generics may not then be authorised for the paediatric indication in question, during the exclusivity period.