How the Saatchi Bill will help rare disease patients

Moved by the feeling that his wife’s ovarian cancer treatment was “medieval, degrading, and ineffective”, and the fact that gynaecological cancer mortality rates have not improved for 40 years, Lord Maurice Saatchi decided to put forward the Medical Innovation Bill. Also known informally as the Saatchi Bill, it aims to encourage doctors to develop innovative treatments for their patients without fear of reprisal.

Many of today’s standard treatments, such as the Halsted method to treat breast cancer, are based on innovations where doctors safely and successfully used previously untested treatments to help patients that were facing imminent death. However, although innovation is allowed in the NHS, an increasingly litigious society is causing a shift towards “defensive medicine”. The result is that doctors stick to standard practises to prevent being sued or stuck off, actively avoiding innovation or falling out of the habit of considering innovative treatments, and medical progress is stymied.

The Saatchi Bill seeks to reintroduce a culture of responsible innovation into the NHS. The Bill sets out parameters within which doctors can operate, so that they can use innovative therapies where standard treatment has failed, is not appropriate or does not exist. Doctors seeking to use innovative therapies will be legally protected so long as the patient gives their informed consent, and if other experts in the field agree that such treatment is necessary, appropriate, and in the patient’s best interests. Furthermore, the Bill will empower patients to ask for innovative therapies. The Bill will not protect doctors who act irresponsibly, negligently, without the agreement of their peers, or without the patient’s consent.

At the BHD Foundation we provide information to patients with Birt-Hogg-Dube Syndrome (BHD), and fund medical research into the disease. BHD is a rare genetic disorder that affects roughly 450 families worldwide. The symptoms of BHD are benign skin tumours of the face, neck and upper body (fibrofolliculomas); lung cysts and predisposition to lung collapse (pneumothorax); and kidney cancer. There is currently no cure.

Kidney cancer is the most life-threatening symptom of BHD, but due to the differing biology of BHD kidney tumours and regular kidney tumours, tyrosine kinase inhibitors – which are commonly used to treat kidney cancers – are not as effective for BHD patients. Several studies have shown that therapies currently approved for other diseases –mithramycinPaclitaxel and radiotherapy – may be effective treatments for BHD kidney tumours. However, in the current climate of defensive medicine, doctors are understandably unlikely to risk their careers by administering these treatments to BHD patients. Thus, we at the BHD Foundation and our sister organisation the Myrovlytis Trustwhole-heartedly support the Saatchi Bill.

Although the Saatchi Bill will be of benefit to all patients requiring innovative treatments regardless of what disease they have, the Bill particularly highlights the plight of patients with rare cancers. Indeed, patients with rare diseases – cancer or otherwise – are likely to derive the most benefit from the Bill, as there is often no standard treatment available; treatment is often limited to symptom management, rather than being curative. At the current rate of drug discovery, it would take hundreds of years to develop cures for all 6000-8000 rare diseases, thus innovation is sorely needed to get therapies to rare disease patients faster. Although individually rare, when considered collectively, rare cancers account for roughly 53% of all cancer cases, and 3.5 million people in the UK (roughly 1 in 17) have a rare disease, meaning that so-called “rare diseases” in fact affect a large proportion of the population.

Additionally, rare diseases are considered to be “fundamental diseases” as they are often extreme or pure forms of common complex disorders. For example, research into the rare disease Familial Hypercholesterolemia led to the development of statins, which are now widely used to treat high cholesterol – a major cause of heart disease, which affects millions of people in the UK. Thus, the Saatchi Bill has the potential to have a significant and positive impact on public health.

The Saatchi Bill is currently under public consultation and the Health Secretary, Jeremy Hunt, is willing to support the Bill, so long as the public, patients, charities and medical communities say they want it. Now is your chance to let your voice be heard on this important issue. If you support the Bill and would like it to be passed into law, please clickhere and vote “YES”. You will also have the opportunity to tell the Department of Health why you support the Bill, should you want to do so.

With your help, we really can make a difference to how medicine is practised in the UK, and give hope to millions of people living with diseases that are currently incurable.

If you would like more information, you can read the Bill here, visit the Saatchi Bill Tumblr feed, or follow @SaatchiBill on Twitter.

  • Lu X, Wei W, Fenton J, Nahorski MS, Rabai E, Reiman A, Seabra L, Nagy Z, Latif F, & Maher ER (2011). Therapeutic targeting the loss of the birt-hogg-dube suppressor gene. Molecular cancer therapeutics, 10 (1), 80-9 PMID: 21220493
  • Zhang Q, Si S, Schoen S, Chen J, Jin XB, & Wu G (2013). Suppression of autophagy enhances preferential toxicity of paclitaxel to folliculin-deficient renal cancer cells. Journal of experimental & clinical cancer research : CR, 32 (1) PMID: 24305604
  • Zhang Q, Si S, Schoen S, Jin XB, Chen J, & Wu G (2014). Folliculin-deficient renal cancer cells exhibit higher radiosensitivity through autophagic cell death. The Journal of urology PMID: 24434776

www.bhdsyndrome.org – the primary online resource for anyone interested in BHD Syndrome.

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