Tuesday, January 21, 2020

The Freedom of Treatment Act: Empowering Terminally-ill Patients to Try Experimental Drugs and Therapies

Report by The Abigail Alliance Patient Advocacy Committee 
Rose Winkle, Chair | GBM Patient & Advocate | St. Louis, MO
Jeremy Van Tress, Vice-Chair | ALS Patient & Advocate | Corvallis, OR
Kyle Coddington | GBM Patient & Advocate | Boswell, PA
Corey Polen | ALS Patient & Advocate | Brownsburg, IN
Dr. Benjamin Freilich | Physician & GBM Patient Advocate | Englewood, NJ
Dr. Jim Neely | Physician & Colon Cancer Patient Advocate | Cameron, MO
Frank Burroughs | Head & Neck Cancer Patient Advocate | Lorton, VA
Steve Walker | Colon Cancer Patient Advocate, | Tampa Bay, FL

Summary

Patients facing a range of terminal-illnesses from Amyotrophic Lateral Sclerosis (ALS) to Glioblastoma Multiforme (GBM) have very few approved treatment options. Some investigational drugs offer a glimmer of hope, but less than 10% of patients are eligible to participate in clinical trials.

Although State and federal Right to Try laws provide important protections to physicians and drug companies that help terminally-ill people try investigational therapies outside of clinical trials, patient access has been limited up to this point due to ambiguities in the law.

Our committee sought to clarify the basic rights of people with terminal illnesses under Right to Try laws in order to facilitate wider access to new treatments.

We believe that terminally-ill patients should be allowed to exercise a basic freedom of treatment in their attempt to preserve their own lives. The burden that insurers, state bureaucracies, and researchers continue to impose on patients is a gross violation of personal autonomy.

Failure to facilitate access to treatment for terminally-ill patients who have run out of approved options is especially concerning in light of the growing number of states legalizing inexpensive assisted suicide drugs.

This report proposes that states enact “Freedom of Treatment” measures to protect the fundamental choice of people to try to save their own lives. This initiative would facilitate terminally-ill patients’ access to investigational drugs by dramatically reducing paperwork, wait times, and healthcare bureaucracy, potentially saving lives and reducing the number of patients who turn to assisted suicide due to lack of resources and treatment options.

Introduction


Less than two years after his father's death, Dr. Benjamin Freilich was working feverishly to save the life of another family member.

His brother's Glioblastoma Multiforme (GBM) — the most common and virulent form of brain cancer — had returned after surgery, radiation, and chemotherapy. He participated in a clinical trial, but the tumors persisted.

Since few GBM therapies are approved, patients ordinarily run out of treatment options shortly after their cancer returns.

But Dr. Freilich had done his research and assembled a team of forward-thinking physicians to help his brother get access to an experimental brain cancer treatment known as Gliovac (ERC1671). Epitopoietic Research Corporation (ERC), the Belgium-based manufacturer of Gliovac, uses a patient's own tumor cells as well as donor tumor cells to create a personalized immunotherapy vaccine.

Dr. Freilich's brother and 7 other glioblastoma patients have received Gliovac under Right to Try laws in the last year, but it has not happened without a fight from special interest groups who oppose expanding patient access to investigational treatments.

Despite the overwhelmingly bipartisan passage of state and federal Right to Try laws, critics have begun utilizing a new set of legal barriers to limit patient access to promising new therapies.


Insurers provide dark incentives for “Right to Die” instead of “Right to Try”


Less than a month after California legalized physician-assisted suicide in 2015, Stephanie Packer was informed that the experimental chemotherapy her doctors requested would not be covered.

But assisted suicide drugs were a cheap alternative. Packer’s insurer offered a $1.20 co-pay for life-ending prescription drugs.

In a growing number of states that have legalized assisted suicide, offering patients aid in dying rather than covering the costs of access to treatment could save insurance companies millions of dollars each year.

Stephanie Packer’s story is but one example.

Insurers have also been quick to cover the cost of assisted suicide drugs in New Jersey, where one 46-year old father is being treated with Gliovac. His wife and children had to raise money to pay for treatment after his GBM recurred and his insurer declined to provide coverage.

The same was true for Dr. Freilich's brother, who is being treated in New York.

Both patients were ultimately able to receive treatment thanks to the generosity of donors and the vaccine's manufacturer, but access for most people with terminal illnesses is severely limited by a lack of resources.

According to reports from the Oregon Health Department, where assisted suicide has been legal since 1997, the percentage of deaths attributed to a patients’ reluctance to “burden” their families rose from 12% in 1998 to 40% by the 2010s.

The notion of assisted suicide as something individuals choose at the end of life to make their death more comfortable ignores the disturbing fact that without coverage of treatments that patients hope could extend or improve ther lives, “right-to-die” becomes the dangerous default for too many people facing terminal illnesses.

Under such grim circumstances, choosing to die is no choice at all.

If insurers begin providing basic coverage for the cost of treatment outside of clinical trials, manufacturers could quickly scale up existing Right to Try programs and new drugs would be provided to a larger demographic of terminally-ill patients. Additionally, reimbursing caregivers for their time in relation to terminally-ill patients’ participation in clinical trials or Right to Try programs could expand access to more patients who may otherwise opt for hospice care or assisted suicide due to lack of resources.

The Federal Right to Try law only allows drug manufacturers to recover the direct costs of providing access for patients, so the price per patient is negligible, especially in comparison to clinical trials.

Expenses associated with Right to Try programs do not ordinarily exceed the per patient cost of hospice and palliative care. Linking payment limits to the amount that would be typically paid for hospice and palliative care may be too low to cover all of the costs associated with personalized biological products like Gliovac, but providing any level of coverage would increase the number of terminally-ill patients able to access treatment.

Recommendation: States should require insurers to cover federally-eligible experimental therapies on a basis no less favorable than hospice and palliative care.

Bureaucrats Propose Regulation to Restrict Access to Cancer Treatment


An anti-competitive regulation proposed by the Michigan Certificate of Need (CON) Commission would have restricted access to a promising new form of cancer treatment. The rule was ultimately rejected, but its near-adoption stands as a reminder that hospitals have erected government barriers to protect profits at the expense of terminally-ill patients.

Under the rules passed by the CON commission, researchers would have been required to obtain additional third-party certifications in order to offer immunotherapy treatments. The additional certifications would have effectively prohibited many smaller hospitals and clinics from offering CAR T-cell treatments.

In 2017, the Food and Drug Administration (FDA) approved two CAR T-cell therapies for children with leukemia and adults with advanced lymphoma. In August of 2019, the Centers for Medicare and Medicare Services clarified that the type of certification proposed by the Michigan CON commission was not necessary for hospitals to safely offer CAR T-cell therapy. Instead, the agency recommended a different third-party certification which is already required for all hospitals in Michigan.

James Essell, cellular therapy chair of the U.S. Oncology Network, explained to the CON commission that many patients who do not have the resources to travel to major medical centers would be excluded from the potential benefits of these therapies if the rule was adopted.

Similar objections were raised by a variety of cancer research organizations, doctors, pharmaceutical companies, and patient advocates.

The American Cancer Society warned that the new regulations meant some patients "may not be offered CAR T-cell treatment or new cellular therapies in the future, or may experience significant health decline or death before they could identify a facility and gain access."

But the CON commission sided with the state's largest hospital systems in unanimously adopting the new rules.

Fortunately, on October 30, both chambers of the Michigan legislature approved a resolution scrapping the rules. A 2002 reform to the state's Certificate of Need laws let them block the regulations from taking effect.

Depending on the state, everything from the number of hospital beds used in a Right to Try program to the installation of a new MRI machine are subject to CON review. The boards themselves are often subject to regulatory capture and become subject to the influence of large hospital chains or other special interests.

State lawmakers could repeal CON laws entirely to prevent other needless regulations in the future. At the very least, pre-approval access programs being conducted for patients with terminal illnesses should be exempt from state CON laws.

Recommendation: States should exempt Right to Try programs from CON requirements.

Researchers Slow to Adopt New Informed Consent Procedures


Since passage of the Federal Right to Try law, many physicians and research centers have begun adopting a new set of best practices in their informed consent procedures for terminally-ill patients.

The Chao Family Comprehensive Cancer Center at University of California, Irvine already ensures that patients are informed of the possibility of receiving Gliovac outside of the clinical trial being conducted.


Even bioethicists who ardently opposed Right to Try are beginning to acknowledge that physicians have a duty to inform terminally-ill patients if another physician is willing to provide access outside of clinical trials.

"A doctor cannot be made to seek non-trial access for a patient, but if one is unwilling to do so, patients may find another doctor," said Dr. Alison Bateman-House, a bioethicist at NYU. "Particularly if a physician knows her colleagues are willing to seek access, she may have a moral duty to refer patients."

Unfortunately, some research centers have been slow to adopt the practice.

At the federal level, regulators are looking to upgrade the clinicaltrials.gov website, which is a crucial resource for patients interested in clinical trials, but inclusion of Right to Try programs on the website is unlikely due to the agency's preference for its own outdated "expanded access" program.

Establishing state-level clinicaltrials.gov websites would help ensure that terminally-ill research subjects know all of their treatment options.

Recommendation: Establish state-level clinicaltrials.gov websites to ensure that terminally-ill research subjects know all of their treatment options prior to providing informed consent for clinical trials.

Conclusion


The burden that insurers, state CON laws, and researchers continue to impose on people facing life-threatening illnesses needs to be eliminated. Our committee has proposed “The Freedom of Treatment Act”, which contains three major provisions to help patients overcome barriers to treatment.

The legislation:

  • Allows patient participation in pre-approval access programs without regard to state certificate of need requirements
  • Establishes a state-level clinicaltrials.gov website to ensure that terminally-ill research subjects know all of their treatment options
  • Requires insurers to provide coverage of federally-eligible experimental therapies on a basis no less favorable than hospice and palliative care

Model Legislation


To repeal section ____.____, and to enact in lieu thereof one new section relating to investigational treatments.

Be it enacted by the General Assembly of the state of _______, as follows:

Section A. Sections ____.____ is repealed and one new sections enacted in lieu thereof, to be known as section ____.____, to read as follows:

____.____

1. The provisions of this section may be cited as “The Freedom of Treatment Act".

2. For purposes of this section, the following terms shall mean:

(1) "Eligible patient", a person who meets all of the following:

(a) Has a terminal illness;

(b) Has considered all other treatment options currently approved by the federal Food and Drug Administration and all relevant clinical trials conducted in this state;

(c) Has received a prescription or recommendation from the person's physician for an investigational drug, biological product, or device;

(d) Has given written informed consent which shall be at least as comprehensive as the consent used in clinical trials for the use of the investigational drug, biological product, or device or, if the patient is a minor or lacks the mental capacity to provide informed consent, a parent or legal guardian has given written informed consent on the patient's behalf; and

(e) Has documentation from the person's physician that the person has met the requirements of this subdivision;

(2) "Investigational drug, biological product, or device", a drug, biological product, or device that has successfully completed at least phase one of a clinical trial but has not been approved for general use by the federal Food and Drug Administration and remains under investigation in a clinical trial. The term shall not include state or federal Schedule I controlled substances;

(3) "Terminal illness", a disease that without life sustaining procedures will result in death in the near future or a state of permanent unconsciousness from which recovery is unlikely.

3. A manufacturer of an investigational drug, biological product, or device may make available an investigational drug, biological product, or device to eligible patients under this section. This section does not require that a manufacturer make available an investigational drug, biological product, or device to an eligible patient. A manufacturer may:

(1) Provide an investigational drug, biological product, or device to an eligible patient without receiving compensation; or

(2) Require an eligible patient to pay the costs of or associated with the manufacture of the investigational drug, biological product, or device.

4. This section does not require a health care insurer to provide coverage for the cost of any investigational drug, biological product, or device. A health care insurer may provide coverage for an investigational drug, biological product, or device. Any company or health benefit plan that provides coverage or benefits for hospice or palliative care shall provide coverage and benefits for investigational drugs, biological products, and devices under this section or 21 USC 360bbb-0a on a basis no less favorable than hospice and palliative care.

5. Coverage of investigational drugs, biological products, and devices under this section or 21 USC 360bbb-0a shall not be subject to any prior authorization, dollar limit, co-payment, deductible, or other out-of pocket expense that does not apply to hospice and palliative care, regardless of benefit category determination by the company administering the health benefit plan.

6. For a health benefit plan that meets the definition of "high deductible health plan" as defined by 26 U.S.C. Section 223(c)(2), the provisions of subsection 5 of this section shall only apply after a covered person's deductible has been satisfied for the year.

7. This section does not require the department of corrections to provide coverage for the cost of any investigational drug, biological product, or device.

8. The Department of Health shall publish a website at the URL “clinicaltrials.STATE.gov”. The website shall include a list of all drug developers, physicians, and hospitals that have voluntarily provided the Department of Health with a list of diseases and conditions for which they provide investigational drugs, biological products,or devices under this section or 21 USC 360bbb-0a.

9. Notwithstanding any other provision of law to the contrary, no state agency or regulatory board shall revoke, fail to renew, or take any other action against a physician's license issued under chapter 334 based solely on the physician's recommendation to an eligible patient regarding prescription for or treatment with an investigational drug, biological product, or device. Action against a health care provider's Medicare certification based solely on the health care provider's recommendation that a patient have access to an investigational drug, biological product, or device is prohibited.

10. Notwithstanding the provisions of [State Certificate of Need Law] or any other law to the contrary, any person or entity may develop, offer, and provide new institutional health services within the state to provide investigational drugs, biological products, and devices to eligible patients under this section or 21 USC 360bbb-0a.

11. If the clinical trial is closed due to lack of efficacy or toxicity, the drug shall not be offered. If notice is given on a drug, product, or device taken by a patient outside of a clinical trial, the pharmaceutical company or patient's physician shall notify the patient of the information from the safety committee of the clinical trial. Any sponsor of a clinical trial for patients with terminal illnesses conducted in the state shall provide prospective trial participants, as part of the informed consent proceedings conducted under 21 C.F.R. 50.25 or any other law, with a list of all drug developers, hospitals, and physicians that treat prospective trial participants’ disease or condition as indicated by clinicaltrials.STATE.gov.

12. Except in the case of gross negligence or willful misconduct, any person who manufactures, imports, distributes, prescribes, recommends, dispenses, or administers an investigational drug, biological product, or device to an eligible patient with a terminal illness in accordance with this section shall not be liable in any action under state law for any loss, damage, or injury arising out of, relating to, or resulting from:

(1) The design, development, clinical testing and investigation, manufacturing, labeling, distribution, sale, purchase, donation, dispensing, prescription, administration, or use of the drug or device; or

(2) The safety or effectiveness of the drug or device.

13. If any provision of this section or its application to any person or circumstance is held invalid, such determination shall not affect the provisions or applications of this section which may be given effect without the invalid provision or application, and to that end, the provisions of this section are severable.

14. The director of the department of health and senior services shall promulgate all necessary rules and regulations for the administration of this section. Any rule or portion of a rule, as that term is defined in section ___.___, that is created under the authority delegated in this section shall become effective only if it complies with and is subject to all of the provisions of chapter ____ and, if applicable, section ___.____. This section and chapter ____ are nonseverable, and if any of the powers vested with the general assembly pursuant to chapter ____ to review, to delay the effective date, or to disapprove and annul a rule are subsequently held unconstitutional, then the grant of rulemaking authority and any rule proposed or adopted after __________, shall be invalid and void.