Muscular Dystrophy: Drugs

Department of Health written question – answered on 28th November 2014.

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Photo of Julian Huppert Julian Huppert Liberal Democrat, Cambridge

To ask the Secretary of State for Health, what steps he is taking to ensure that emerging treatments for Duchenne muscular dystrophy which receive conditional approval are available through the Early Access to Medicines Scheme as early as possible; what steps he is taking to encourage flexible approaches to the licensing process for potential new treatments for rare conditions; what progress NHS England has made on development of a commissioning policy for the Translarna treatment for Duchenne muscular dystrophy; and if he will make a statement.

Photo of George Freeman George Freeman The Parliamentary Under-Secretary of State for Business, Innovation and Skills, The Parliamentary Under-Secretary of State for Health

The Early Access to Medicines Scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need. The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for the scientific aspects of the scheme and the scientific opinion will be provided after a two-step evaluation process:

- step I, the promising innovative medicine (PIM) designation

- step II, the early access to medicines scientific opinion

The PIM designation will give an indication that a product may be eligible for the EAMS (based on early clinical data) and that the development programme is on track. The PIM designation will be issued after an MHRA scientific meeting and could be given several years before the product is licensed.

The scheme is voluntary and the opinion from MHRA does not replace the normal licensing procedures for medicines.

There have been no applications from companies with products for Duchene Muscular Dystrophy.

The opinion will support the prescriber and patient to make a decision on whether to use the medicine before its licence is approved. The EAMS scientific opinion is valid for one year in the first instance and lapses at this time or at the time of the grant of a marketing authorisation e.g. conditional marketing authorisation.

For certain categories of medicines going through the centralised marketing authorisation procedure (European procedure), in order to meet unmet medical needs of patients and in the interest of public health, it may be necessary to grant marketing authorisations on the basis of less complete data than is normally required. In such cases, it is possible to recommend the granting of a marketing authorisation subject to certain specific obligations to be reviewed annually, a conditional approval. The granting of a conditional marketing authorisation will allow medicines to reach patients with unmet medical needs earlier than might otherwise be the case.

A conditional marketing authorisation for the first in class medicinal product Translarna (ataluren) was granted this year. Translarna is an orphan medicinal product that is used to treat patients aged five years and older with Duchenne muscular dystrophy (DMD) who are able to walk. Translarna is expected to slow down the loss of walking ability in DMD patients. As part of the conditional marketing authorisation, the company will be required to provide comprehensive data on the efficacy of Translarna from an ongoing confirmatory study.

Rare diseases are classified as conditions affecting no more than 5 in 10,000 people in European Union and patients with rare conditions deserve the same quality, safety and efficacy in medicines as other patients with more common conditions. Since the pharmaceutical industry has little interest, under normal market conditions, in developing and marketing medicines intended for small numbers of patients (orphan medicinal products), the European Union offers a range of incentives to encourage the development of these medicines in order to address the unmet clinical need (orphan drug legislation, Regulation (EC) No 141/2000). These incentives include a period of 10 years market exclusivity, the provision of Protocol Assistance (scientific advice specifically tailored for orphan medicinal products) and fee reductions and waivers for regulatory procedures. Products intended for treatment of Duchenne muscular dystrophy qualify for incentives in the orphan drug legislation.

Applications for the designation of orphan medicines are reviewed by the European Medicines Agency through the Committee for Orphan Medicinal Products (COMP). For orphan designation, the following criteria must be fulfilled. The medicinal product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the European Union or without incentives it is unlikely that expected sales of the medicinal product would cover the investment in its development and no satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorised, or, if such method exists, the medicinal product will be of significant benefit to those affected by the condition. Via the MHRA, the United Kingdom takes an active role in the decision making process at the COMP, ensuring applications for Orphan Drug designation of potential drug candidates for rare diseases are appropriately recognised, encouraging companies to develop their products further. For licensing, it is compulsory for designated orphan medicinal products to use the centralised procedure to gain a marketing authorisation.

The UK is fully represented at the Committee on Human Medicinal Products, ensuring that applications for a Marketing Authorisation (MA) for an Orphan Drug are thoroughly and rapidly evaluated for quality, safety and efficacy and a MA is granted without undue delay for the treatment of rare diseases.

In some circumstances, marketing authorisations may undergo a more rapid regulatory review called ‘accelerated assessment’. This occurs where the Applicant can demonstrate that the medicinal product is expected to be of major public health interest (particularly from the point of view of therapeutic innovation). For drugs for rare diseases, marketing authorisation applications may be granted as a conditional authorisation or an authorisation under exceptional circumstances. The granting of a conditional marketing authorisation allow medicines to reach patients with unmet medical needs earlier than might otherwise be the case, and ensures that additional data on a product are generated, submitted, assessed and acted upon. Under exceptional circumstances, the MA Applicant must demonstrate that he is unable to provide comprehensive data on the efficacy and safety under normal conditions of use, because, for example, the indications for which the product in question is intended are encountered so rarely that the Applicant cannot reasonably be expected to provide comprehensive evidence.

NHS England can confirm that a draft clinical commissioning policy for Translarna treatment for Duchenne muscular dystrophy has been developed and is being considered as part of the annual funding prioritisation process for 2015-16.

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