Advancing rare disease treatments: patient groups and industry

Rare diseases have become a more interesting target to the pharmaceutical industry as a result of incentives offered by the USA Orphan Drug Act (1983) and EU/141/2000 legislation, the increasing development costs and failure rate of common disease drugs, and the potential for rare disease drugs to influence common disease treatment. It is predicted that revenue from rare disease drugs will continue to rise and that more pharmaceutical companies will divert resources towards their development (Orphan Drug Report 2014). However, the traditional drug development methods are not optimal for rare diseases as experts and patients tend to be sparse. Arguably the best way around this issue is to collaborate with a patient group – the concept of last week’s Findacure workshop in London.

Patient groups are experts in the disease, know the expert clinicians, the researchers and, more importantly, the patients. They can help design more accessible and suitable clinical trials (see our previous blog on clinical trials for rare disease patients), and can facilitate more long-term patient follow-up. Collaborations are also beneficial to the patient groups as there is a focus on their disease, increasing awareness and knowledge, and their members gain access new clinical trials.

However, such collaborations are often scrutinised by external groups and accusations of undue influence can be damaging to the patient group who must maintain the trust of their patients and partners. As collaborations have such scope for treatment development, it is important that they be protected from such attacks and that all parties are comfortable with continued interactions. For this reason it is essential that collaborations have predefined rules of engagement, maintain transparency for all interactions, especially those with a financial incentive, and welcome external scrutiny and assessment. In addition the majority of pharmaceutical companies will have strict codes of conduct, often including regulations regarding patient group contact. It is important however that both parties should be involved in drafting a collaborative agreement ensuring that both group agendas are fairly represented.

Patient groups should play an active role in these collaborations; acting as a voice for their patients, chasing unanswered questions and ensuring data dissemination. However, for patient groups to ask the right kinds of questions and have a meaningful input, industrial partners must ensure they provide understandable information about ongoing work and results. A recent survey found that only 22% of the general public in several European countries were knowledgeable about drug discovery and clinical trials (EUPATI Public views on Medicine Development). To increase understanding among the public, and importantly within patient groups, organisations such as EUPATI provide training, workshops, webinars and online resource libraries to help in the development of the skills required to form mutually beneficially collaborations.

The power of patient groups to impact the development of rare disease treatments is likely to increase following the recent launch of Rare Disease International (RDI) – a global organisation advocating for recognition of rare diseases as an international public health and research priority, thereby increasing the services and support available to patients and their families. RDI aim to facilitate networking, increase education of rare diseases on a national and international level, and increase rare diseases representation in the pharmaceutical industry. They will also be able to offer support and guidance to individual patient groups considering collaborations.

However, one of the largest issues associated with the development of treatments for rare diseases cannot be influenced by these groups alone: the cost of treatments. Pharmaceutical companies must recuperate the costs associated with the R&D and clinical trials for any new treatments. Unfortunately the limited number of target patients for any single rare disease most often results in these costs being extremely high per patient, severely limiting access. It is only through an overhaul of the pricing structures, and questioning profit after recuperation, that this issue can be addresses and newly developed treatments can be accessed by those most in need.

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