What Is Wrong with Orphan Drug Policies?

Abstract

The effects of orphan drug policies raise serious concerns among payer organizations and lead to often-tragic disappointment for patients who are denied much anticipated drug reimbursements. We evaluate the effects of orphan drug policies on the basis of this concern for real accessibility to drugs. We highlight two unforeseen effects of orphan drug policies: 1) they provide unique business opportunities for manufacturers and 2) drugs approved through these policies are often inaccessible because of their high price. We identify six causes of this emergence of effects. The first four are the direct result of incentives included in orphan drug policies. The fifth cause is the "off-label" use of orphan drugs. These emergent effects have several implications: 1) they raise doubts about the equity of access to drugs, 2) they highlight the limitations of the cohort paradigm in medicine, and c) they force third-party payers to make drugs accessible even when the prices of drugs are believed to be disproportionate to the clinical effects obtained.

Orphan drugs policies: a suitable case for treatment

“…Some researchers argue that the notion of 'social benefit' embodied in current reimbursement processes is too narrow [3]. Others argue, on a utilitarian basis, that the opportunity cost of treating rare diseases is too high [4], or point to the 'excessive' profits made by manufacturers and 'orphan drug creep', whereby some drugs with an orphan designation are also indicated for treatment of diseases with a much larger patient population [5].…”

Policymaking for Orphan Drugs and Its Challenges

“…As Cȏté and Keating state, fairness requires "a positive action by the state [or government] when the market does not provide a good match between investments and health [care] needs. Finally, fairness requires that the barriers to access should be morally justifiable" [23].…”

“…Several studies indicate that orphan drugs are very expensive and that their accessibility can be a huge concern [13]. For example, cerzyme was developed by Genzyme to treat Gaucher disease.…”

“…For example, cerzyme was developed by Genzyme to treat Gaucher disease. There are about 2,000 patients with Gaucher disease in the US [13], and the medication costs as much as $400,000 every year for an adult patient [14]. There is a concern that pharmaceutical companies can create a monopoly market [6], precluding payers' ability to negotiate prices, by "splitting up a disease into several sub-diseases that qualify as rare diseases (a practice called 'disease sub-setting,' 'salami-slicing' or disease stratification')" [15].…”

“…Some researchers also raise the question of whether, even when given incentives to focus on rare diseases, the pharmaceutical industry concentrates only on commercially lucrative areas. At least 95 of the aforementioned 400-plus orphan drugs were for cancer treatment; orphan drugs used to treat rare cancer are the most profitable [13,[18][19][20]. Haffner and colleagues ask, does the "development [of orphan drugs] actually take place for the truly rare diseases, or only for the more common ones" within the rare group, like the rare cancers [21]?…”