- Rare Diseases
-
Patients' Opinion on the European Regulation on Orphan Medicinal Products
Organization for Rare Disorders
(EURORDIS)
DIA European Conference, March 1998
Patients suffering from rare diseases and the non-profit
organizations which represent them often feel like the orphans of the healthcare and
social systems. Because most healthcare professionals and the public at large do not know
anything about the diseases which affect them, they feel ignored, isolated and left to
their own fate in the shadow of society. Their needs are obvious: better training of
physicians, and especially General Practitioners, better diagnosis tools, better
treatments, increase in the funds spent researching the epidemiology, diagnosis and
therapy of these diseases, recognition of their specific social problems by authorities,
and means to end their isolation.
Today, persons suffering from rare diseases in various Member States do not enjoy equal
access to treatments. It is only through European legislation that each Member State's
patients will gain equal access to efficacious medicines against rare diseases.
In our eyes, the proposed Regulation on Orphan Medicinal
Products provides a series of major incentives and official guidance to convince
pharmaceutical companies to research and develop effective treatments against rare
diseases:
- Rare diseases are defined on the basis of their prevalence as
affecting less than five per ten thousand persons in the Community, thus relieving
sponsors from the burden of having to demonstrate lack of return on investment for each
product they may want to develop. However, we severely lack epidemiological knowledge on
most rare diseases, and the Committee on Orphan Medicinal Products will have to give
itself sufficient means to make informed decisions in the absence of prevalence data.
- Medicinal products which have been defined as orphan products
in the United States or in Japan will be allowed to apply for orphan status in Europe.
- A Committee on Orphan Medicinal Products will be established
to coordinate the implementation of this Regulation, on which patient organizations will
be granted three seats. As a patient organization, we consider as extremely important the
opportunity for affected persons to be able to express their needs and expectations at the
highest level. Patients and their representatives are bona fide experts, the most capable
of informing a professional committee about patients' needs, epidemiological data about
their diseases, and on the best way to enroll participants into the clinical trials which
are necessary to commercialize medicinal products.
- Sponsors will be guided in the process of clinical research,
application and review of efficacy data by experts from the European Agency for the
Evaluation of Medicinal Products and by the Committee on Orphan Medicinal Products.
- Funds allocated to the European Agency for the Evaluation of
Medicinal Products by the Community will be used to waive, in part or in total, the fees
payable by sponsors when applying for a marketing authorization. It is obvious that the
Parliament and the Council will have to provide sufficient funds to allow this Regulation
to function properly and to create the conditions necessary for the development of the
largest possible number of therapies against pathologies which are seriously neglected
today.
- Commercial exclusivity is granted to the sponsor for a period
of ten years for a defined therapeutic indication. Recently, the Commission introduced a
safeguard measure against eventual excessive profits, by allowing for the possibility of
withdrawing commercial exclusivity after six years if an unexpected increase in the
breadth of use of the product was demonstrated. However, the parameters used to evaluate
the use of such products will have to be defined precisely for the industry to feel
confident about investing in the development of orphan products.
The proposed Regulation defines orphan medicinal products as
intended for the diagnosis, prevention or treatment of rare diseases. This definition does
not include nutritional supplements and medical devices, despite the fact that these
products are germane to the care and treatment of numerous rare pathologies (such as
metabolic disorders). Measures will have to be taken to apply similar incentives to the
research and development of these therapeutic tools.
In addition, for us, it is essential that an independent Committee on Orphan Medicinal
Products be created, with tight links to the CPMP, hence within the EMEA, and that patient
representatives be members of it with full voting rights. These three conditions must be
met if we want to see an European orphan medicinal product policy which truly answers the
needs of patients, and works in the transparency necessary for the pharmaceutical industry
to engage itself optimally.
Original address of this page:
www.eurordis.org/gi/op-ang/gipatopeur-ang.htm
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