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The Medical, Social and EthicalDimensions of Afinitor

Description of Afinitor

Afinitor, which is also known by itsgeneric name Everolimus is manufactured by a Swiss company calledNovartis International. It is categorized under the mTOR inhibitorcategory and it does little damage to normal cells in body whenattacking the cancerous ones.

Afinitor is categorized under theMammalian Target of rampamycin inhibitor category and it does littledamage to normal cells in body when attacking the cancerous ones. TheMammalian Target of rampamycin is a protein in our bodies thatcontrols proliferation of cells. These cells, in turn, becomespecialised and form tissues. Subsequently, the m-TOR also managesthe growth of new blood vessels to the newly formed tissues. Tumoursdevelop as a result of an out-of-control cell division. Afinitorbonds with FKBP-12,a type of protein in our bodies, to form a compound that is able toblock the Mammalian Target of rampamycin(m-TOR) .When Afinitorinhibits the m-TOR successfully, rapid dividing of tumour cells isstopped and also reduces the supply of blood to these cancerouscells. Consequently, these cells die due to lack of oxygen andnutrients. Afinitor has made management of cancerous cells easier dueto its mechanism of action.

Afinitor comes in two forms:Afinitor tablet and Afinitor Disperz. The tablet is simply swallowedwhile the Disperz tablets are dissolved in water to form an oralsuspension which is then drunk. For the Afinitor Disperz, handling ofthe tablet should be done after wearing gloves. One is advised to gofor a full body check before beginning the dose and the doctor shouldbe informed of any conditions the patient has. Afinitor should not beused by patients who are allergic to everolimus. Only after getting aqualified doctor’s approval and under his/ her supervision shouldone begin dosage. Ten milligrams every day is the recommended dosage.When one forgets to take his/her daily dosage, one should take itright away if it is within six hours from when he/she normally takesit. If the individual realizes that more than six hours are past fromhis/her usual time of taking the dose, he/she should forego the dosethat day. Afinitor could harm a foetus in the womb so the patient isadvised to take all measures to ensure that she does not becomeexpectant during that period. Vaccination during this period wouldnot be effective in the patient’s body. It is recommended to give agrace period of eight weeks after completing the Afinitor medication,before taking a vaccine.

Afinitor can be used by patientssuffering from a rare kidney cancer called Renal Cell Caricnoma.Drugs such as Sorafenib and Sunitinib had been previously used totreat advanced Renal Cell Carcinoma (RCC) but they weren’tsuccessful. Together with Exemestane, Afinitor tablets can also beused by women who are past menopause but have breast cancer that hasalready began spreading and whose cancer cells have hormone receptorson their surface. Previously, Anastozole and letrozole had beenpreferred for this rare type of breast cancer but they failed. Pancreaticneuroendocrine tumours could also be treated using Afinitor. Afinitortablets and Disperz can also be used by children and grown-ups withtuberous sclerosis complex and have subependymal giant cellastrocytome. Afinitor is used normally in advanced forms of cancer orwhen it is impossible to remove the cancerous cells surgically.

Comparison of Orphan Drug programsin the United States and in Australia

According to Norvatis officialwebsite, Afinitor has been approved inone hundred and sixcountries around the world including Australia. In those countries,Afinitor appears as either as a five milligram or ten milligramtablet. In March 2009, the Food and Drug Administration (FDA)approved Afinitor for treatment of Renal Cell Cancer and later in May2011, Afinitor got approval from FDA for treatment of pancreaticneuroendocrine tumours.

Therapeutic Goods Administration(TGA) is the body responsible for granting orphan drug status inAustralia. Thereafter, the approved companies are added into theAustralian Register of Therapeutic Goods (ARTG) and supply theirproducts lawfully, anywhere in Australia. With reference toTherapeutic Goods Regulation 1990, a drug categorized as orphan issupposed to prevent, treat or diagnose a rare condition or is notviable in commercial terms for production.

Norvatis International made anapplication to the Therapeutical Goods Administration and was laterfilled in the Australian Register of Therapeutic Goods (ARTG). On the17thday of July 2008, Afinitor was accredited orphan drug status used forcuring Renal Cell cancer. Two years later it was approved fortreating patients suffering from Tuberous Sclerosis Complex whichcaused growth of tumours in key organs such as the brain. This was abig move and later in 2012, Afinitor capsules were approved fortreatment of tumours in pancreas as a result of PancreaticNeuroendocrine Tumours. Norvatis believed people living in Australiawith rare types of cancer should have access to new medicine.Novartis is a member of the Innovative medicines industryassociation, Medicines Australia and Oncology Industry Taskforce.These organisations collaborate with the government among otherstakeholders to address the challenges facing ontological conditionsin Australia.

Parents and other family memberswith patients affected by these disorders face a lot of challenges.For example, in most cases where a child is affected, one parentalways stays at home to look after the child. This leaves one parentwith the burden of providing for the family and they cannot affordthese medications. The Pharmaceutical Benefits Scheme (PBS) offers asubsidized price for Afinitor tablets to patients who meet somecertain criteria and are normally given to those that have no otheralternative treatment available. PBS is part of the government’sNational Medicines Policy whose aim is to address scarcity ofnecessary medication and health related services, so that Australia’seconomic goals and health outcomes are attained. This helps theaffected Australians with some rare disease get medication. Afinitoris expensive to produce hence it would be out of reach for mostpeople who needed it but due to the government subsidy, it’s fairlyaccessible.

Nature of disease, number ofaffected individuals and prevalence

In the US, according to the OrphanDrug Act (1983), an orphan disease is a condition that affects fewerthan 200,000 US citizens. However the meaning in Australia differsfrom that in the US. Referencing from the Therapeutic GoodsRegulations (TGR) 1990, an Orphan Drug is regarded as that which isused in treating rare diseases or that which is unprofitable if itwas produced to cure another disease. Paraphrasing from the TGR 1990,an orphan drug should be used by less than 2000 people in each yearfor it to maintain its Orphan Drug Designation. According to theAustralian Bureau of Statistics, the total population is about 23.8million people. Deductively, a rare disease in Australia is one thataffects one out of 11900 people. The current total US populationestimates are about 318.9 million people. Therefore, a rare diseasein US is one affecting about six out of 10000 people in US. TheAustralian figure is quite low considering less than one person outof 10000 Australians is said to have a rare disease. Therefore, itcan be concluded that the US Orphan Drug Program covers more peoplethan the one in Australia.

Renal Cell Carcinoma is a rarecancerous condition affecting the kidney. Three percent of highlyinfectious abnormal growths is caused Renal Cell Carcinoma (RCC) andis regarded as the sixth leading cause of death in the US (Bukowskiet al, 2015).

Advanced hormone receptor-positiveher2-negative breast cancer occurs especially among some women whoare past menopause. This breast cancer is rare and occurs when theHer2 genes are fewer than normal in the breast. Breast Cancer in manywomen is caused by high levels of Her2 genes but low levels of Her2are rare but and cause lumps in the breast.

Tuberous Sclerosis Complex (TSC) isa rare genetic condition leading to abnormal growths in the brain andother important organs. This causes learning disorders amongschool-going children. According to the Food and Drug Administration2012 release, 40,000 patients are affected by TSC in the U.S.A.alone.

PancreaticNeuroendocrine Tumours is a type of cancer that affects thepancreatic cells. The pancreas is a gland near the stomach region andhas two types of cells namely: endocrine and exocrine pancreaticcells. When this cancer advances, it becomes difficult to remove thiscells surgically since the pancreas is delicate hence Afinitor ispreferred.

Unfortunately, affected families areprone to social stigma. The parents have to be around the home tolook after the child with rare disease. For example, the mothercannot socialize with other friends because she has to take care ofher child. In most cases, the parent does not allow other people tolook after the child on the basis that they won’t know how tohandle the child properly. Therefore, it is important for affectedpeople to get help as early as they can.

Program Incentives

Sponsor companies have to do a lotof research and clinical trials on these rare oncological conditionswhich are expensive. The target populations are spread out far andwide making the clinical trials difficult. A substantial number oftests have to be done with different patients to determine the sideeffects of the drug. Therefore, development of new drugs such asAfinitor is not commercially sustainable hence little research isdone. More funds are directed towards the diseases that affect themasses leaving out people with rare conditions. This is unethicalsince everyone has a right to quality healthcare. Production oforphan drug and research into rare diseases should not be based ontheir commercial viability that is outright discrimination againstpeople with rare diseases. Therefore the governments in US andAustralia have come up with incentives which may be offered duringthe clinical testing when experiments are being done and/or after thedrug is in the market.

i) Sponsors own the marketexclusively for a period.

Incentives granted by the OrphanDrug Act offer protection from competition by the manufacturers ofthe drug. The law restricts, for a period of seven years, any otherindividual or company from entering the market with the same drug forthe same use. This is after the sponsor has got FDA marketingapproval. Unfortunately, this isn’t the case in Australia.

ii) Tax Credit

Norvatis in the US also got taxcredit for not more than 50% for the costs involved in theexperiments before the drug is released to the market. This helps thesponsoring company recoup money lost in the research and encouragemore research about the rare disease.

iii) Grant funding.

The sponsors, on request, may getgrants and funds to enable them do extensive research about rarediseases. In the US this is catered for by Orphan Product GrantsProgram.

iv) Waiver Fees

Norvatis enjoys benefits like notpaying some fees unlike other drug manufacturers in the mainstreamindustry. Likewise, in Australia, it is free to apply for an orphandrug designation.

v) Immediate review of new drugspurported to treat rare diseases.

These drug manufacturers get anin-depth review as soon as they come up with a new drug. The standardtime for reviewing new drugs is 10 months but for any drug believedto treat some rare diseases, the time is reduced to 6 months.Companies in Australia believed to be manufacturing drugs to helppeople living with rare diseases are also given a priority when beingreviewed by the Therapeutic Goods Administration.

vi) Protocol assistance

Food and Drug Administration reviewssome protocols in one and a half months and issues relating to theprotocols to assess whether they are adequate to meet scientific andregulatory requirements identified by the sponsor.

Works Cited

Bukowski, Ronald M, Robert A. Figlinand Ronald Motzer. RenalCell Carcinoma:Molecular Targets and Clinical Applications. P.6

Mantus, David and Douglas J. Isano,FDARegulatory Affairs,3rdEdition p. 191-197.

Australian Therapeutic GoodsAdministration. TheTherapeutic Goods Administration in Australia, n.d 13 Mar. 2015

The Novartis International.TheNorvatis International company, n.d. 13 Mar. 2015.