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www.eurordis.org

EFPIA Think Tank9 November 2007

Common and rare indications

Flaminia MacchiaEuropean Public Affairs Officer

Brussels, 9 November 2007

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A story from real life

9 November 1987: birth of twin girls Halla Ruth and Sillja BjorgHalla weak, tired, blue, breathing and swallowing problems.Nobody believed the mother. Stressed. Need restGrand mal seizure. Life less all kinds of tests. Hospitalisations.Different diagnosis among which acid reflux. Aluminium whichworsened Halla condition.Apnea alarm. Every night. Shifts night and day. Moved to Sweden .March 1989: mother started reading about endocrine diseases. Sherecognised the symptoms of Hypoparathyroidism: I had it all infront of me .April 1989: Halla was dying in the hospital. Mother desperatelybegging for a serum-calcium test W e dont do unnecessary testson Sundays . Finally, a night shift ! test was made. Halla had avery severe HPTH. The serum-calcium unbelievably low (1.23)

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A story from real life

In the hospital and started on D-vitamin treatment and calcium .August 1991: Halla diagnosed with nephrocalcinosis : her kidneyswere damaged and couldnt take more D Vitamin.She needed PTH hormone . Not produced because so few patientsthat drug companies would not benefit to produce it.In 1993: mother wrote 35 letters to authors of Medline articles askingif anyone in the world was treated with the PTH hormone.She learned about Dr. Karen W iner doing a study on the hPTH at theNIH (U.S.). Halla welcome to take part in the study J anuary 1994:first child in the world and now longest time - treated with hPTH.

In 1997 kidney damage was gone. Normal life. Not ill anymore.In 1998 Halla was told that the study would be cut down to 3 yearsand she would have to go back to the D Vitamin.Then, Swedish Orphan provided hPTH from 1999-2003 and thenForsteo off label since 2003.

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Definition and treatment

Hypoparathyroidism: condition in which the body produces too littleparathyroid hormone (PTH). The consequence, hypocalcemia , is aserious medical condition.

Some patients have severe hypocalcemia life-threateningcondition . The acute attacks are treated with IV calcium infusion(central venous catheter as calcium irritates peripheral veins andcauses phlebitis).

Conventional treatment of HPTH has been calcium and Vitamin Dsupplementation BUT treatment with vitamin D and calcium causeshypercalcemia and damages kidney function .

Teriparatide is a recombinant human DNA 1-34 parathyroid hormone,identical in structure to the active 1-34 portion of endogenous PTH.Brand name Forsteo/Forteo currently registered for osteoporosis.HPTH patients want this treatment no side-effects reported so far.

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Current situation

Patients with the rare condition for whom it is a life-savingtreatment do not get it (not marketed, not reimbursed).

Patients with the common condition - osteoporosis - get it (marketedand reimbursed).

According to Dr. Marlene Haffner, Director of the Office of RareDiseases for the FDA: "Sadly the company who developedTeriparatide only submitted data to the FDA for approval for osteoporosis."

398,87 for 28 injection 1 month!

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6 B russels 9 November 2007

Current situation

Problem in Europe. Yesterday nice email from Eli Lilly abouthPTH treatment in children.

BUT hPTH 1-34 has been studied for 14 years in Dr W inersstudies all data available. No side-effects reported on children.

New PO: HPTH Europe main goal is to get hPTH treatmentavailable (= marketed and reimbursed) for patients in Europe.

On the web: Teriparatide should only be used in patients for whom the potential benefit outweighs the potential risk. Isnt thewhole drug development based on a risk/benefit ratio?

In the US Nycomed is also marketing Preotact hPTH 1-84 for patients with osteoporosis, produced by NPS Pharmaceuticals.Studied for 3 years on HPTH patients (Dr Rubin study).

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Conclusions

A company may have its (good?) reasons to decide not todevelop a drug for the rare indication but cannot fail to its CSRand Public Health mission

There is a need for a transparent dialogue with patientrepresentatives and for answers to their legitimate questions. W ecan understand your reasons if we are explained.

The company holding the patent must do everything in its power to ensure the development of the drug for its rare indication,

either by itself or by another company.

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Thank you