rare disease

  1. List and describe 3 challenges for patients living with a . Do not list lack of a cure. For each challenge, provide a potential strategy to mitigate the challenge or provide a solution. In addition, be sure to identify the appropriate parties / stakeholders involved in the strategy or solution (30 points).

Patients living with the raredisease face several challenges. Firstly, it is difficult to obtainan accurate diagnosis since these diseases are rare (Dickson940).Consequently, patients end up facing increased economic, social, andmedical burdens. In addition, the patients feel they do not getadequate information, or they do not understand the explanation givenby the doctor. To deal with this challenge, medics should develop anunderstanding of the patient needs. For instance, CORD organisationin Canada advocates better recognition for such patients in thehealth system.

Secondly, it is difficult to findhealth centres or physicians with experience of the same raredisease. Inmost countries, there are few health centres and fewcomprehensive care services. Since these diseases are rare, doctorsdo not take the time to study them. In addition, these patients finddifficulties in accessing social, financial, and medical services.This is because most decision makers are neither familiar with thedisease nor its medication. Dickson(943) advocates the need for scientists to conduct innovativeresearch and in-depth study on the cause, diagnosis, and treatment ofthe disease. However, s are varied but they have similarcharacteristics. Nevertheless, researching on one disease increasesthe understanding of the other.

Thirdly, the requires expensive treatment compared to other common diseases. Dueto limited cases of s, their treatments are expensive andlimited due to limited or no research on the particular disease. Apatient may end up using varieties of medication in search of themost appropriate. As a result, lots of money is used. To deal withthis challenge, scientist needs to conduct intensive research to comeup with cheap and accurate treatment for s. CORD is agood example of an organisation deals with this challenge.

  1. List and describe 3 objectives of NORD. In addition, describe how NORD has addressed each of the objectives (30 points).

The three objective of NORD is toprovide advocacy, research and educational services, as well as offerpatient assistance programs.


NORD is working with otherpartners to achieve its advocacy goals. It acts as a link between thenational institutions and patient community to bring apositive impact on their health (NORD,n.p). Someof the organisations that collaborate with include the FederalGovernment, United States Congress, Broader Patients Advocate Groups,and Medical Societies. Its main objective is to offer advocacy onbehalf of patients. Indeed, NORD has written manyletters addressing to senators, congress, commissioners, governors,and to their partners to address the issues related to s.

Research and EducationalServices

NORD has a Research Grant Programwhose obligation is to offer seed money to academic scientistsstudying on diagnosis, medication, and treatment of s.Correspondingly, the clinical researcher provides preliminary data toindicate the best treatment that may be effective and safe forpatients. Additionally, NORD assists dedicated researchers of rarediseases by offering them opportunities to participate in theclinical trial on their websites. It seeks to enhance family-patientunderstanding through presentations and publications. This ensuresindividuals who take part in research understand the process hence,they participated as informed researchers.

Offer Patient AssistancePrograms

NORD achieve this objectivethrough NORD’s Patient Assistance Program, Patient informationcentres, Researcher stories, patient stories, online patientcommunities, NORD patients meeting, and clinical Trials. Since itsfoundation, NORD has used assistance program to assist the patientget life-sustaining and life-saving medication. At patientinformation centre, NORD has dedicated staffs that are willing toanswer any question related to clinical trials, patient resources,and any other question. Besides, NORD holds national and regionalmeetings for patients and their families. They also haveonline patient communities where they connect patients all over theworld. Here, patients can form a platform to give their stories,challenges, experiences, as well as give support each other.

  1. List and describe two challenges that manufacturers face when setting the price for an Orphan drug. Please be specific. In contrast, list and describe two reasons why some manufacturers may be attracted to Orphan drug development (40 points).

Today, manufacturers of Orphandrugs are facing many problems while setting the price of theirproducts. Firstly, they are facing distinct commercial problems.Orphan drugs present different commercial challenge compared totraditional biopharma drugs. Orphan drugs market is miniscule hence,it is difficult to gauge the value of the drug accurately. Inaddition, the commercial strategies used for orphan products arepoorly suited.

Secondly, market access alsoposes a challenge in pricing. The research cost of orphan drug ishigh due to few patients with s (Dickson,937). Asuccessful orphan drugs pricing strategy result in high-risk researchinvestment, the product must be justifiable by key stakeholders, andthe patient should access it in regardless of financial situation. Inmost cases, orphan products are injectable and require patient’ssupport to realise a positive outcome. Therefore, validate the priceof the drug become a challenge.

Although orphan products piecingfollows drug pricing policies, manufacturers are still attracted toOrphan Drug development. This is because the patients and thethird-party payers have limited or no negotiating power. Hence, themanufacturers have a chance to make more profits. Secondly,manufacturers have the ability to maximize orphan product price withreimbursement policies and domestic pricing. Haffner(30) urges that manufacturer can split the product into severalsub-drugs to qualify for similar s.

Work Cited

Dickson,D. W., et al. &quotOffice of Rare Diseases neuropathologic criteriafor corticobasal degeneration.&quot&nbspJournalof Neuropathology &amp Experimental Neurology&nbsp61.11(2002): 935-946.

Haffner,M. E. &quotOrphan Drug Development Update.&quot&nbspDrugInformation Journal&nbsp(1996):29-34. Print.

NORD.&quotWelcome to NORD.&quot&nbsp—National Organization for Rare Disorders.Web. 19 Mar. 2015.