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Developing Medical Devices for use in Rare Diseases

Authored by James Sheppard

The global medical device market is highly competitive and currently valued over $300bn in 2011. Only a very small fraction of this market is attributable to rare diseases. Medical devices are of crucial and often vital importance for patients living with a rare disease. In most cases, medical devices provide a major contribution to life expectancy and quality of life in both children and adults. Despite this, for patients suffering from rare diseases, medical devices are often not considered for use or specific medical devices have not yet been developed.

Medical devices and rare diseases are far less frequently mentioned together in articles or discussions than therapeutic drugs. Although, frequently medical devices are discussed in connection with treatment of paediatric diseases, which is often the population most affected by rare diseases too. There are several diseases or conditions that can only be treated with the help of medical devices. One such device is The Vertical Expandable Prosthetic Titanium Rib (VEPTR) that has saved over 300 infants who otherwise would have suffered fatality from lack of breath from thoracic insufficiency syndrome. The inventor of the product, Dr Robert Campbell, took 13 years to gain regulatory approval from the FDA as they could not get enough patients to sufficiently test the device. This is not an isolated case and it has occurred across a number of different specialised medical devices. Many of the devices currently used are delivery systems for drugs or biologics and improve the treatment regime for patients with very challenging conditions

Regulators have begun to put in place steps to ease the approval of medical devices for rare diseases. Unlike pharmaceuticals and biologics, medical device regulations are not as clear cut when it comes to orphan diseases. In 1990, the US FDA began to develop specific guidelines for the use of medical devices in rare diseases. The first device was approved in 1997 for the treatment of foetal urinary tract decompression. The Humanitarian Device Exemption (HDE) program designates a device that is intended to benefit patients by treating or diagnosing a disease or condition which affects fewer than 4,000 individuals in the United States per year.

At time of writing, the FDA list 56 approved devices. As the medical device market has grown over the last decade the number of HDE approvals has also grown. One of the latest approved devices is the NeuRx DPS, a diaphragm stimulator indicated for patients living with Amyotrophic Lateral Sclerosis (ALS). The device was developed by SYNAPSE Biomedical a USA based medical device company which was founded in order to commercialise the NeuRx DPS system and delays the need for full-time ventilation. The devices approved by the FDA have been developed by a range of companies including innovative companies such as SYNAPSE and global medical device companies such as Medtronic. The main benefit to developers is that it offers the chance to commercialise devices faster and for the patients the chance to have access to potentially life-saving devices sooner.

Devices approved under the HDE program are treated as experimental. An area that has benefited from the HDE program is diagnostics and specifically laboratory tests. An example of a laboratory test exempted under the HDE is Mesomark, manufactured by Fujirebio Diagnostics which is used in the detection of a rare cancer called Mesothelioma. The regulation creates an alternative pathway to market for devices that may help people with rare diseases. In 2011 the FDA approved six medical devices for rare diseases. It is hoped that having a similar policy in Europe may allow for more devices to reach the market.

In 2008 the European Commission held a public consultation entitled ‘Recast of the Medical Device Directive.’ EURORDIS, the Rare Disease patient group organisation in Europe, stated that it ‘does not support the proposed recast of the current legislative framework, including the proposal to create a MDs Committee within the EMEA.’ The results of the consultation is that EURORDIS believe it will take longer and cost more to get potentially life-saving devices to market for patients with rare diseases. EURODIS believe that the only way forward is to create European wide HDE program.

It is fundamental to remember the specific needs of rare disease patients. For most rare disease patients there is currently no therapeutic cure and the one way of alleviating their suffering maybe the use of specific medical devices. By definition there are very few people suffering from each rare disease and in the same way that drugs are specific for each disease, medical devices too are highly specific. Therefore patients need medical devices that are produced for a small patient population. This creates challenges for companies and patients alike. By only producing small numbers of highly specialised devices means that the development/production costs will be very high. Typically medical device companies rely heavily on economies of scale in order to reduce development and manufacturing production costs.   Now though, some medical device companies are adapting currently available, mass produced devices for rare disease patients. An example of this is the BSD-2000 (BSD Medical Corporation) which is indicated for the treatment of severe cervical carcinoma under the US HDE program. Normally the BSD-2000 is used in general solid tumour radiotherapy. Of course, similar to therapeutic drugs, the main problem with the majority of devices lies in the cost and many payers are unwilling to offer reimbursement for these devices. For the foreseeable future, reimbursement looks set to remain a considerable challenge for the European rare disease industry during a period of pronounced austerity.

Although the global medical device market has delivered considerable innovation and improved many fields of healthcare, the rare diseases aspect of the market has been somewhat neglected. The US has taken significant steps forward in creating a framework to get rare disease medical device products to market but Europe is lagging. Over the coming years consultations are set to take place in order to try and establish European wide regulation for medical devices in rare diseases. Until this lofty objective is achieved the prospect of increasing use for medical devices in rare disease is very limited.

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