Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the com­pli­cat­ed U.S. health­care sys­tem, nav­i­gat­ing a se­ri­ous ill­ness such as can­cer or heart dis­ease can be re­mark­ably chal­leng­ing for pa­tients and care­givers. When that ill­ness is clas­si­fied as a rare dis­ease, those chal­lenges can be­come even more acute. And when that rare dis­ease oc­curs in a pop­u­la­tion that ex­pe­ri­ences health dis­par­i­ties, such as peo­ple with sick­le cell dis­ease (SCD) who are pri­mar­i­ly Black and Lati­no, chal­lenges can be­come al­most in­sur­mount­able.

Peo­ple with SCD of­ten have less ac­cess to com­pre­hen­sive care. A lack of treat­ment cen­ters and spe­cial­ists ded­i­cat­ed to the treat­ment of SCD is part of the prob­lem, as is the fact that a ma­jor­i­ty of pa­tients are in­sured through Med­ic­aid, fur­ther lim­it­ing care op­tions. In ad­di­tion, SCD has not his­tor­i­cal­ly re­ceived the re­search fund­ing and phar­ma­ceu­ti­cal in­vest­ment di­rect­ed to oth­er or­phan dis­eases. These dis­par­i­ties are thought to con­tribute to the dif­fer­ence in out­comes be­tween peo­ple with SCD and peo­ple with oth­er or­phan dis­eases.

What weighs heav­i­ly on me is that we lack a stan­dard­ized mod­el for tran­si­tion­ing from pe­di­atric to adult care in the U.S. There is a lack of in­for­ma­tion sur­round­ing this tran­si­tion, in part be­cause chil­dren with SCD were un­like­ly to sur­vive to adult­hood be­fore the in­tro­duc­tion of the use of hy­drox­yurea for SCD treat­ment in 1984. How­ev­er, ex­ist­ing da­ta now show that the mor­tal­i­ty rate for pa­tients tran­si­tion­ing from pe­di­atric to adult care more than dou­bles.i

There are far few­er com­pre­hen­sive cen­ters for adults with SCD than there are for chil­dren with the dis­ease.ii These cen­ters – which in­clude hema­tol­o­gists, emer­gency room physi­cians, so­cial work­ers, psy­chol­o­gists, ed­u­ca­tion spe­cial­ists and nu­tri­tion­ists, among oth­ers – have been avail­able for chil­dren with SCD since the ear­ly 1970s. There are on­ly 49 cen­ters for adults with SCD in the U.S., which is less than half of what’s avail­able to adults suf­fer­ing from he­mo­phil­ia or cys­tic fi­bro­sis.ii

Pe­ri­od­ic episodes of pain are a hall­mark symp­tom of sick­le cell dis­ease, yet there are al­so is­sues sur­round­ing how pain man­age­ment is han­dled in chil­dren ver­sus how it is han­dled in adults. In the adult pop­u­la­tion, there is a great deal of scruti­ny and stig­ma as­so­ci­at­ed with pain man­age­ment; adults with the dis­ease are of­ten per­ceived as drug seek­ing. This is dis­cour­ag­ing for pa­tients, but it al­so im­pacts the care they re­ceive. Once a pa­tient is per­ceived as drug-seek­ing, they may not be seen for hours, of­ten do not re­ceive ap­pro­pri­ate treat­ment and are sub­ject to fur­ther com­pli­ca­tions as a re­sult. One 17-year-old with SCD de­scribed her anx­i­ety sur­round­ing the tran­si­tion to adult care be­ing root­ed in the ex­pe­ri­ences of her old­er sis­ter who al­so has the ill­ness:

“I’m hon­est­ly re­al­ly, re­al­ly ner­vous. I re­al­ly hope I get a good doc­tor be­cause my sis­ter, who is al­ways in and out of the hos­pi­tal, told me that her doc­tors don’t be­lieve a word that she says. There are these stereo­types of sick­le cell pa­tients as drug ad­dicts… she’s nev­er had an en­counter with a good doc­tor as an adult.”

De­spite these lam­en­ta­ble ex­pe­ri­ences, there is hope and en­thu­si­asm sur­round­ing new ap­proach­es to tran­si­tion­ing adults with SCD. The very same changes that re­sult­ed in im­proved care for chil­dren can al­so be en­act­ed to ben­e­fit the adult pop­u­la­tion, in­clud­ing ad­di­tion­al fund­ing for com­pre­hen­sive cen­ters for adult care, as well as proac­tive­ly com­bat­ting the in­flu­ence of racism in our med­ical sys­tem. In fact, the state of Cal­i­for­nia just re­cent­ly se­cured $15 mil­lion from their leg­is­la­ture to fund sick­le cell cen­ters.

Fix­ing the tran­si­tion is­sue in SCD re­quires the en­tire health­care ecosys­tem – in­clud­ing those of us in bio­phar­ma – to in­no­vate in our ways of work­ing with the SCD com­mu­ni­ty. Draw­ing on the ideas of sick­le cell doc­tors, so­cial work­ers, care­givers and pa­tients, among oth­ers, call­ing for change, we need to cre­ate a new mod­el for pa­tient care, one that helps to over­come dis­trust and anx­i­ety, fo­cus­es on en­gage­ment and out­reach to the com­mu­ni­ty and seeks to over­come the spe­cif­ic ac­cess, eq­ui­ty, health­care lit­er­a­cy and qual­i­ty of life is­sues this pop­u­la­tion faces.

The Sci­ence of Giv­ing a Damn

At For­ma Ther­a­peu­tics, we are work­ing with lead­ers in the SCD com­mu­ni­ty to de­vel­op and share in­for­ma­tion on the chal­lenges and op­por­tu­ni­ties to im­prove care for pa­tients in tran­si­tion. We’ve de­vel­oped a pa­tient out­reach team that in­cludes SCD ad­vo­cates and ex­perts, and we are ded­i­cat­ed to work­ing along­side the com­mu­ni­ties we seek to serve. For us, keep­ing pa­tients at the cen­ter of all that we do means ap­proach­ing drug de­vel­op­ment dif­fer­ent­ly, not just cre­at­ing a drug that we hope will serve pa­tients well but ac­tu­al­ly chang­ing the care and treat­ment par­a­digm. We call this the sci­ence of giv­ing a damn.