tdr> Public/Private partnership - Visceral Leishmaniasis

[Cassy Workman]

Dear List,
I read with interest the reports of the trials for this important new 
drug.
This is especially important for patients who have failed all other 
options, however, it seems impossible to obtain any 
compassionate access for patients who are in this position..
In the HIV area we have been very successful in gaining access to 
drugs in phase III development for patients who are at imminent risk 
of disease progression and/ or death.
I fail to see why this should be any different for this drug and I 
would urge all readers of this list to pressure this company to offer 
the same.

We currently have one patient who is in desperate need of this drug 
and who is highly likely to die without access to it. 
That the company involved seems to not be concerned with such 
situations should temper our approval of their behaviour in general.

dr cassy workman 
_________________________________________


On 28 Oct 99, at 11:14, Jeffrey Shaw wrote:

Date sent:      	Thu, 28 Oct 1999 11:14:43 -0200 (EDT)
Send reply to:  	leish-l at bdt.org.br
From:           	Jeffrey Shaw <jshaw at tba.com.br>
To:             	Multiple recipients of list <leish-l at bdt.org.br>
Subject:        	tdr> Public/Private partnership - Visceral Leishmaniasis

> >Date: Thu, 28 Oct 1999 01:28:16 +0100 (MET)
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> >from: "Jocelyne Bruyere" <bruyerej at who.ch>
> >to: tdr-scientists at who.ch
> >Subject: tdr> Public/Private partnership - Visceral Leishmaniasis
> >Sender: owner-tdr-scientists at who.ch
> >
> >-------------------------------------------------------------------
> >-for tropical disease researchers & friends: "tdr-scientists list"
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> >
> >
> >PUBLIC/PRIVATE PARTNERSHIP: DEVELOPING AN ORAL TREATMENT FOR VISCERAL
> >LEISHMANIASIS (from TDRnews Oct 1999)
> >
> >A boy whimpers as the doctor searches for the vein in his skinny arm, in
> >which to insert the catheter. He knows what he's in for: six hours on a
> >drip with shivering and fever. But it's worth it and he will tolerate it.
> >Otherwise he will die.
> >
> >Twelve beds, each with a clean green sheet, are neatly arranged along the
> >length of a ward, each with a plastic bottle of golden-coloured fluid
> >suspended above, attached through a length of tube to the vein in a
> >patient's arm. Twelve patients lie, stoically biding the month of
> >treatment. There are children, men and women, old and young.
> >
> >A mass of patients crams a waiting room. Inside, more people. The doctor
> >is well known and patients, from the poorest sectors of society, are
> >referred to him from far away. A young child has received pentostam,
> >first-line treatment for kala-azar. His buttocks, the site for injection
> >of the drug, are sorely ulcerated. Is he one of the 40% who don't respond
> >to this treatment any more?
> >
> >A boy's eyes are earnestly fixed on the doctor's face. The doctor
> >pronounces him cured after six months of follow-up, and signs him off.
> >The boy looks eager. Now he knows he will live, he is full of hope.
> >
> >A man with a walrus moustache listens intently to the doctor's careful
> >explanation about the trial. He looks at the informed consent papers, now
> >translated into Hindi on advice from the local ethics committee, and
> >answers the doctor's questions. He knows he must receive treatment else
> >he will die. Finally he decides to refer to his family members before
> >consenting to enter the trial.
> >
> >In the laboratory where samples from the kala-azar patients are
> >analysed, the equipment is arranged systematically around the room.
> >There is a row of little tubes, each labelled clearly, the analyst
> >methodically testing each in turn. He codes, and carefully wraps, each
> >microscope slide made from samples taken from the patients' spleens. The
> >up-to-date reference books lining the walls already wear an aged look.
> >
> >This is the setting for imminent Phase III clinical trials of a new oral
> >treatment - miltefosine - for visceral leishmaniasis. The trial is being
> >run by TDR and ASTA Medica, a pharmaceutical company based in Frankfurt,
> >Germany. The place is Bihar, most poverty-stricken of Indian states and
> >the home of kala-azar. Travelling north from Patna, the capital, to
> >Muzaffapur where there are two kala-azar clinics, one is struck by the
> >fertility of the land, the lush crops of all descriptions, and wonders w
> >hy the pov erty. Hand-in- hand with the poverty goes kala-azar, and north
> >of Muzaffapur, where poverty is perhaps even greater, kala-azar becomes
> >hyperendemic.
> >
> >The current drugs for kala-azar all have drawbacks. They are
> >administered by injection or infusion and require the patient to be
> >hospitalized - miltefosine will be the first oral treatment. Nearly 40%
> >of cases in Bihar, where the disease is anthroponotic, are resistant to
> >treatment with pentostam. The drug causes serious side-effects -
> >mortality in 2-5% of patients and toxicity in 10-15%. The second-line
> >treatment, pentamidine, also has serious side-effects, causing 7 -9%
> >mortality and 60% toxicity with irre versible damage such as diabetes.
> >
> >Amphotericin B is most effective, but is also toxic, causing severe
> >rigor and fever and sometimes anaphylaxis. It requires infusion every
> >other day for 15-20 days, and besides is so expensive as to be
> >unaffordable by 95% of patients. In fact, cost is a problem with all the
> >treatments available; miltefosine will certainly be cheaper, should it
> >reach the market, although the exact price is yet to be determined.
> >Affordable cost for target populations is a key feature of all TDR
> >product profiles.
> >
> >In preparation for the trial, a workshop was held in Bihar in early
> >July. This was a follow-up to earlier workshops, mentioned in TDRnews
> >Nos. 58 and 59, where clinical monitors were trained. Four teams from
> >three kala-azar clinics and a laboratory in Bihar, who constitute the
> >investigators in the trial, and an international team of three from
> >India, Thailand and Viet Nam, who constitute the monitoring team,
> >discussed the finer details of the protocol with representatives of ASTA
> >Medica. The role of the mon itors is to oversee the trial at the three
> >centres to ensure that the rights of the patients are protected and that
> >details of all treatments of all patients, including all adverse events,
> >etc., are recorded accurately, and that each and every miltefosine
> >capsule is accounted for. Between them, a monitor will be on site for
> >almost the entire duration of the trial; it is hoped their work will
> >hasten the drug's passage through regulatory affairs .
> >
> >How do the doctors view the new oral treatment? "Compliance is good, and
> >earlier trials have shown it to have an overall cure rate greater than
> >90%." It is specific, and on the whole not too toxic. "On a
> >toxicity:benefit ratio it scores well and on all parameters should be an
> >ideal first-line drug" said one investigator. "The major drawback is its
> >effect on the foetus, and for this reason it cannot be used as mass
> >outpatient treatment - it will need to be monitored all the time.
> >However, women constitute on ly 25% of cases (more men are infected owing
> >to their habit of sleeping outside), and among female patients, only 30%
> >are of child-bearing age. Thus a relatively small number of patients will
> >be excluded from treatment on these grounds."
> >
> >What do TDR and ASTA gain by working together? TDR provides scientific
> >and organizational input, and tropical diseases know-how, while ASTA
> >provides the full backing and experience of industry. Costs are shared
> >50:50 between the two partners, and when finally the drug is ready to
> >proceed to the regulatory authorities, the application will be more
> >powerful for being backed by both partners. ASTA feels that WHO, through
> >its relationship with governments, will help bring the drug to the market
> >more quickly, an d views its relationship with WHO as an asset and mark
> >of high quality. "Its an example of a relationship that's really working"
> >said an ASTA representative.
> >
> >--
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> >
> 


Cassy Workman MBBS
Associate Director
AIDS Research Initiative
48 Little Oxford Street
Darlinghurst  NSW  2010
Australia
Telephone 61+2+93312688
Fax 61+2+93311833
Email: drcw at rainbow.net.au