Page 1 of 1

Hard to get prescribed and paid for besides of the clinical trials

Posted: Sun Oct 28, 2018 12:29 am
by Olga
It is October 2018 and the clinical trials with ICI drugs - Keytruda, Tecentriq etc. are still ongoing and far from being done with and the results published. There are numerous published cases of success in ASPS plus reports from our patients, there are some interim reports from the clinical trials too. But for the insurance to pay for the drug, it needs to be prescribed off label at the moment meaning there is no published approved indication for ASPS on any ICI drug label at the moment despite many oncologists openly admitting its unprecedented activity in ASPS.
To get it prescribed and paid for, you would need to fight for it. There is a sample letter we wrote when trying to get it for Ivan ere in BC, Canada. ASPS being a registered rare disease helps too as there are often special considerations/rules for rare diseases not requiring the full clinical trials procedure to be done but midterm results and case reports are taken into account too.
The sample letter is here:
http://www.cureasps.org/forum/viewtopic.php?f=76&t=1482
it is only good specifically for ASPS. Other rare cancers or sarcoma would need to do the similar research and compile their own list like this.

Re: Hard to get prescribed and paid for besides of the clinical trials

Posted: Sun Oct 28, 2018 12:30 am
by Olga
The sample review to get Keytruda or Opdivo:


Canadian ASPS Patients on PD-1 / PD-L1 inhibitors

ASPS patient case 1 from Toronto area, Dx May 2015 with the large unresectable primary tumor in the (paralumbar mass 7*8 cm) and very multiple bilateral lung metastases largest 10-15 mm. Several metastases in skull, largest being 1.5 cm.
Cediranib NCT01391962 May 22, 2015 to Sep 8, 2015. Received 25 rounds of IMRT (radiotherapy) to primary Nov 2, 2015 to Dec 7 2015. 25 rounds. Radiation was recommended by his oncologist, Dr. Albiruni Razak immediately before the trial. On Atezolizumab (PD-L1 inhibitor) NCT02458638 Dec 31, 2015 to July 2017. Completed ~21 rounds.
His last dose was in May 2017 with the almost complete resolution of the primary tumor and metastases. He had to be taken off the trial due to the colitis he developed, but the response continued with some additional shrinkage in mm range (Oct.2017)


ASPS patient Case 2 from Montreal. Got Keytruda off label/expanded access in Montreal, starting in September 2016. The patient is responding well presently (Nov 2017) with a partial response in many lung metastases and the complete resolution of some of them, plus with the primary tumor responding by PET, size and manual evaluation (reduced density). Elisa’s oncologist is Dr. Palumbo at the Jewish General Hospital in Montreal. Dr. Razak, sarcoma oncologist from PMH recommended adding radiation therapy to intensify the response after the first evaluation has shown stability, which was done after the second dose (IMRT to the primary tumor). Merck’s Patient Assistance Program is paying for the drug.

There are other numerous cases of the durable, long term responses to Keytruda, Opdivo and Tecentriq in ASPS patients residing in other countries (mostly US and China), in the clinical trials setting and off label prescription, the information re. Clinical cases with the contact information of the treating oncologists might be provided if needed.


ASPS & PD-1 publications reporting clinical benefit:

CTOS 2017 presentation.

The interim results of the clinical trial Axitinib and Pembrolizumab in Subjects With Advanced Alveolar Soft Part Sarcoma and Other Soft Tissue Sarcomas NCT02636725 confirmed an activity and a clinical benefit in ASPS and were presented on the CTOS 2017.
In the subset of nine patients with alveolar soft part sarcoma, four (44%) achieved partial response and three (33%) achieved stable disease, equating to a 78% clinical benefit rate.
Wilky BA, et al. Abstract 2762964. Presented at: Connective Tissue Oncology Society Annual Meeting; Nov. 8-11, 2017; Maui.

Publications.

We researched both, pembrolizumab (Keytruda) and Nivolumab (Opdivo) as they appear to be interchangeable:
Nivolumab and pembrolizumab: Monoclonal antibodies against programmed cell death-1 (PD-1) that are interchangeable http://dx.doi.org/10.1053/j.seminoncol.2017.06.007
It appears likely that any effective PD-1 blocking agent will have similar clinical outcomes (subject to trial design) and the evidence or lack thereof of efficacy can be combined.

1. Anti-PD1 therapy with nivolumab in sarcoma https://doi.org/10.1093/annonc/mdw388.06
2. PD-1 blockade using pembrolizumab in adolescent and young adult patients with advanced bone and soft tissue sarcoma.
http://ascopubs.org/doi/abs/10.1200/JCO ... suppl.3060
3. Positive Tumor Response to Combined Checkpoint Inhibitors in a Patient With Refractory Alveolar Soft Part Sarcoma: A Case Report http://ascopubs.org/doi/full/10.1200/JGO.2017.009993
4. PD-1 blockade using pembrolizumab in adolescent and young adult patients with advanced bone and soft tissue sarcoma
https://doi.org/10.1186/s13569-016-0064-0
5. Immunoprofiling in alveolar soft part sarcoma http://ascopubs.org/doi/abs/10.1200/JCO ... uppl.11059
6. A phase II trial of axitinib plus pembrolizumab for patients with advanced alveolar soft part sarcoma (ASPS) and other soft tissue sarcomas (STS).

Presented on ASCO 2018 on Saturday, June 2, 2018
https://meetinglibrary.asco.org/record/162020/abstract
" PFS3mo in ASPS pts was 90.9% [95% CI 50.8-98.7], with best ORR of 45.5% [95% CI 18.1-75.4] and CBR of 72.7% [95% CI 39.3-92.7]... Conclusions: Combination Ax/P is well-tolerated with promising activity in ASPS pts. "

Re: Hard to get prescribed and paid for besides of the clinical trials

Posted: Sun Oct 28, 2018 5:00 am
by D.ap
Olga
This is wonderful information !
Thank you.
Number 5 on the above post link isn’t working

http://ascopubs.org/doi/abs/10.1200/JCO ... uppl.11059

Re: Hard to get prescribed and paid for besides of the clinical trials

Posted: Sun Oct 28, 2018 10:06 am
by Olga
Thanks for proof reading, corrected the broken link. If you find any other supporting articles for Keytruda or Opdivo in ASPS, please post them here.