Patients suffering from a raredisease undergo several challenges which make their life unbearableand difficult to cope. Rare disease needs a lot of financial supportdue to the scarcity of medical attention. It leads to an individualusing most of his income to attend to his medical bills (Dickson842). Even those using insurance coverage have an annual limit, whichmakes them end up using their finances to meet their care attention.Most of the insurance covers have a higher installment yet they donot cover for the rare diseases because few people have this illness,and their medical bills are very expensive to cater.
Lack of the right medication forthese conditions has become so devastating for the patients who aresuffering the disease. Challenge in drug development for most of therare diseases is that, little known about the pathophysiology(Dickson940). Best example is with a disease like cancer, which a patientjust goes through therapy which determines his survival. Whenundertaking the treatment, it affects the patient psychologicallybecause one is not in his or her normal statue of mind. Furthermore,it deteriorates the physical appearances of the casualty, which makesone have a low self-esteem about them. Some of the patients with rarediseases are financially unstable they can’t meet even theirclinical finance, which has lead to death of some patients with thisdisease.
Symptoms of the disease start toshow at quite late stage of the illness. Most of these rare diseasestend to hide in the body of a casualty, and the symptoms begin toshow when the disease is at its full blown stage. These makestreatment of the disease somehow impossible, leading to an individualsurviving on drug and therapy alone which only stabilizes thecondition of the patient. The patient keeps taking the medicationuntil the point of death or cure.
NORD has three goals in which ithas helped patients with rare diseases. These targets are financialsupport, medication assistance resources and education resources.
i) Medication assistance support, NORD can assist a patient obtain life-sustaining medication all over the world no matter the place onelives. It has helped patients get assistance on the rare diseasetreatment at affordable prices and also on other different types ofdisease. It has a comprehensive database of patients programs thatenables professional medics that are willing to help the neededpatients without any charges can be able to offer their support. Theyhave also cited Tools, new and articles of equipments rarely foundaround medical localities for the rare disease, which making itpossible for a patient to know where to find the medical assistancefaster.
ii) Financial support is another sector that NORD has helped out inpatients that cannot meet their financial needs. NORD has facilitatedcommunication and collaboration among the organization that are ready pay medical bills for those patients who can`t afford theirtreatment or even buy drug for themselves. It`s able to link thefunding organization and the patients with the financial burden thiseven improves the living standard of the patient (NORD, n.p).
iii) Education assistances, NORD has a research grant program whoseobligation is to provide scientific study on medication, treatmentand diagnosis of different type of disease and which are thesymptoms. It also funds the production of the drug for the illness.Helps researches of this rare disease gain access to the patients ofthe required disease so as to close examine them.
Manufacturers of orphan drug arefacing a significant challenge in setting price for their product dueto several unavoidable challenges, which is brought about by
Costof R&D and market access
Orphan drugs are expensive in some way due to the cost of the R&Dprocess of market access procedures. Carrying out the R&D processis an important step in the manufacturing of the drug, which in someorphan drugs it’s unavoidable thus making the productionexpensive.( Stahl,Elizabeth.Miroslav 34) Due to the high expense of production,makes pricing harder for the producers because they still want tomake the price affordable to the patient. The R&D cost needs tobe recouped from a small number of orphan drugs patients, thusresulting in high acquisition cost per patient. Difficulty in pricingalso comes about where some of the orphan drugs use historical basis,where manufacture are not required to produce new evidence so as togain market for the drug. It makes some of the orphan drugs easy toproduce and other difficult to manufacture, so the market access forthe drugs becomes an issue.
Economic evaluation of orphan drugs.
Pharmaceutical pricing derivedfrom the evidence from the economic evaluation in differentcountries. Unreliable and insufficient clinical data of patientdepending on orphan drug dosage has inhibited economic evaluation ofthe drug. So for manufacturers they are uncertain about the amount toinvest on so as to produce the drug, making pricing a major problemtoo.( Stahl, Elizabeth, Miroslav40) In contrast to the challenge of pricing, somemanufacturers are attracted to orphan drug development due to someadvantages in the production process. The new business of orphan drugmodel, offers an incorporated healthcare solution that has enabledpharmaceutical companies to develop new areas of taking care of thepatient, and also being able to monitor them. Incentives for drugdevelopment provided by the government and other corporationrepresent a further boost for companies developing orphan drug, andtherefore expenses get reduced giving the companies an advantage.Some of this Corporation has announced a more profitable process tohelp regulate, identify and share information with the manufacturersto be able to make development process of orphan drugs lessexpensive.
Although orphan drug pricing followsdrug policies, manufacturers are still paying attention to theproducts development. It is because the payer and the patient havelimited negation power. Hence, the manufacturer has a chance to makemore income. Manufacturers have the ability to maximize the orphanproduct price according to the policy and domestic pricing. Monopolist power gives them the advantage of the fact that noalternative health technology exist for many orphan drugs. Additionally, marketing can further boost market power of the productso leading to more demand for the medication of the rare diseases.
Dickson,D. W., et al. "Office of Rare Diseases neuropathologic criteriafor corticobasal degeneration." Journalof Neuropathology & Experimental Neurology 61.11(2002):
NORD."Welcome to NORD." —National Organization for Rare Disorders.Web. 19 Mar. 2015.
Stahl, Elizabeth,and MiroslavReljanovic. Orphan Drugs understanding the raredisease market and its dynamics.Cambridge, UK: Biohealthcare Pub, 2012. Print.